Carcer Investigation, 9(1) 85-92 (1991)
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PUBLIC ISSUES
Moving Ahead in Cancer Research: Who Pays for Patient Supportive Care for Participation in
Experimental/Investigational Trials? Grace Powers Monaco, J. D. Candlelighters Childhood Cancer Foundation 1312 18th Street NW Washington, DC 20036
INTRODUCTION
payment through stringent application of contract exclusions relating to experimental and investigational care. This “tightening” of reimbursement has occurred at the state medicaid level as well as in private and nonprofit insurance plans, and for the older cancer patient population, extends to medicare reimbursement through the federal government. I will review the background of the current policies of insurers on payment for patient supportive care costs associated with participation in investigational clinical trials, the interpretation of these policies by the courts, and options for crafting insurance benefits that purposefully and prudently cover investigational therapies with scientific merit. The premise of this report is that, except for compassionate and emergency use and NCI category C drugs, patients should receive experimental and investigational therapies only within the ambit of well-controlled clinical trials that are designed to collect data that will answer the question of patient benefit. I mention off label use of approved drugs only peripherally. I consider it abusive of insurers to refuse to pay for indicationsnot approved by the Food and Drug Administration when it is clear, for example, through
The Candlelighters Childhood Cancer Foundation (CCCF) (1) links and facilitates networking, advocacy, and communication among an international volunteer coalition begun more than 18 years ago which now involves 300 plus mutual support groups for parents whose children currently have or had cancer and their medical care teams in the United States, Canada, and around the world. One of the dominant advocacy roles of CCCF involves monitoring the responses of insurers to requests for payment for aggressive, potentially curative care for the young cancer patient and working with families and the medical care team to understand insurancecontract terms, maximize payment under existing policies and, where necessary, take action to change payment policies that threaten access to what our physicians have indicated is needed care. Most pediatric cancer patients participate in clinical protocols and, until recently, insurers have paid for most care prescribed for young cancer patients even if it wore the label of experimental and investigational. In the last two years there has been a consistent tightening of insurer 85 Copyright 0 1991 by Marcel Dekker, Inc.
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reference to the United State Pharmacopeia’s Drug Information for Physicians that certain unlabeled uses have become standard usage. I do not address the issue of when innovation in medical care crosses the line to experimental and investigational, but consider this a subject which needs to be addressed by the medical community. It is my opinion that participation in clinical trials is usually the best treatment available for refractory patients or patients with a conditionnot yet curable or controllable, and that payment for patient supportive care costs associated with participation in wellconceived clinical trials such as those listed on the National Cancer Institute’s Physician Data Query Program is a public benefit and social cost that should be borne by the federal government. However, unless the medical and patient communities are willing to march enmasse to Washington to demand appropriations to cover this cost, we will need to engage in a collaborative, collegial dialogue with insurers, employers, and unions to formulate mechanisms for private sector coverage.
THE HIRORY BEHIND INSURANCE POLICY EXCLUSIONS Most insurance policies cover reasonable and necessary medical care. This term is traditionally defined to mean care that is generally recognized as effective or of proven value in the management of a specific disease and to exclude treatments that have not met that standard, namely, that are still experimental, investigational, or constitute participation in research (2). This standard provision for coverage mirrors the criteria for drug approval by the Food and Drug Administration; generally recognized as effective as demonstrated by objective evidence produced in well-conceived clinical trials (3). It is also in accord with federal reimbursement policy (4). Until the mid-197Os, courts generally interpreted the term reasonable and necessary medical care as excluding therapies that were not generally recognized as effective (i.e., experimentalhnvestigational) (5). There followed a period of legal challenge in which courts took the position that absent express exclusion of experimentalhvestigational treatments or a specific listing of excluded therapies or definition of terms in a way that would support exclusion-treatments would be covered (6). These exclusions were not intended to control costs. Rather, they were intended to counter the potential for abuse of the insurance payment system by demanding patients and oversolicitousphysicians (7) and to support the appropriate use of premium dollars by providing a
mechanism for refusing payment for worthless therapies (quackery) (8) and therapies that were in controlled trials but had not yet demonstrated efficacy (9). Before health care dollars began to tighten up in the late 1970s and early 1980s, the experimental agent and at least some patient supportivecare costs, were covered under NIH grants (10). During that time frame, it appears that most experimentalprotocols were outpatient, involved hospitalization only for complications or tests associated with the research, and the costs of the drug being tested were, then as now, usually covered by the pharmaceutical company sponsoring the trial. Except for W a t r i c cancer treatments, the insurers weren’t seeing all that many claims for inpatient in-hospital supportive care costs associated with implementation of an experimental protocol (11). In pediatric cancer care involving aggressive therapy requiring in-hospital support, until recently, reimbursement was not usually a problem. Whether this is because of the track record of participation in clinical research protocols by most pediatric cancer patients and their acknowledged good track record or for some other reason is unclear (12). It is clear that until the last two years, in pediatric oncology, once the insurer was given a logical and demonstrable explanation of the need for aggressive treatment and provided with documentation to support the reasonable expectations for patient benefit and improved survival, reimbursement was forthcoming (13). In those cases in which insurer resistance is encountered, usually for predeterminationof coverage, a dialogue among the insurer, family and pediatric medical care team is initiated. If this dialogue reveals that the therapy is illfounded or a “crap shoot” for families (rare), challenge to refusal to pay rarely goes further. If, on the other hand, insurer resistance is arbitrary and denial of benefits is contested, childhood cancer families usually prevail (14). However, the times they are a changing! The components of cancer care in the adult population are changing to match pediatric cancer in aggression! The therapies being developed and tested to expand cure or control, primarily in adult cancers, such as autologous bone marrow transplantation, interleukins, and various LAK and TIL cell therapies, and so on, all may require extensive hospitalizations and supportive care. Insurers have indicated to me that lower level claims personnel have inadvertently paid for some of these therapies without realizing they were experimental or investigational. It is these therapies that they have now targeted for identificationat the lower claims level to have passed up for review at the management and technology
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Moving Ahead in Cancer Research
assessment levels for a determinationof whether they are excluded by contract language. In short, from the files of CCCF and my communications with insurers, the insurer position appears to be that (i) they have never agreed to pay for investigationaland experimental therapies whether delivered inside or outside a clinical trial or research setting, (ii) they have not made actuarial provision for coverage and their present profit margin could not absorb broadcast payments for these therapies without substantial premium adjustments, and (iii) inadvertentor erroneouspayment should not compel them to keep on paying. There is at present very little hard data that would permit us to understand the extent of insurer exposure and price out the real costs of coverage should insurers agree to pay for patient supportive care costs associated with participation in clinical trials. It has been indicated that Blue Cross and Blue Shield are looking into doing a prospectivestudy to delineatecosts for one of their programs (15). As far as I know, no insurer has taken a systematic retrospective look at the extent to which they are now, inadvertently, meeting the patient care costs associated with clinical trials.
WHO PAYS NOW? The federal government and other granting authorities should pay for the patient supportive care costs associated with research progress. But do they? Antmann and colleages acknowledgethat until recently NIH: provided important financial support for the care of patients in selected clinical research trials through grants to individual projects or clinical centers. Recently, however, funding for the costs of patient care has largely been deleted from research budgets, and these costs have been assumed by the patients, their health insurers, or both (10). The inclusion of patient care costs in grants to support clinical investigationis in accord with the historical ethic surrounding the conduct of clinical research. For example, Sidney Wolfe, Director of the Public Citizen Health Research Group, stated the generally accepted position on the issue of payment for participation in clinical investigation (16).
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One aspect of experimentaldrugs that many lay people (and, unfortunately, some physicians) do not seem to understand is that they are not legitimate articles of commerce, although the FDA of late has been acting as though they should be. Human medical experimentationof all kinds-emphatically including new drug investigations-are legally subject to rigid ethical standards, one of which is that human guinea pigs should not have to pay for the privilege of being experimented upon (2-4).
To the same effect see Lind (17): Whether patients can be asked to pay for research designed for personal therapeutic gain or for experimental treatment is a difficult question. It might be argued that patients should not be asked to pay for an unproved treatment, regardless of the severity of their disease or the ease with which they can affort to pay.
* * * Since miracles are rare and the first patient to receive a new treatment is unlikely to be cured, the burden of being the first to try a treatment should not be compounded by the need to pay for it (3, pp 314, 315). Traditionally, when actuarially determining risks and premiums, insurers have worked from the premise that therapies that have not been established through clinical trials as needed for patient benefit or palliation will be covered by another mechanism (18). There is no question that many insurers have paid. However, in the cases I have observed, payment has been: Due to the mistaken belief of lower level claims processors that the therapy was generally accepted or standard. Upon request for amwaiver, although tcchuidy experimental was established by studies and articles furnished by the treating physician to have shown sufficient expectation of patient benefit in survival or palliation that it warranted coverage (19). Payment for a class of therapy on an interim basis pending completion of clinical trials-this would include payment for NCI Category C drugs. Payment for unlabeled use8 listed in the USPDI.
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WHEN CAN WE CONSIDER THERAPIES READY FOR COVERAGE UNDER STANDARD POLICY LANGUAGE? 1. ’zheneed for prognostic factors to be &lined before patient benefit uin be implied and coverage extended under standard policy language. Singer and Goldstone in their report Clinical Stzufies of ABMT in Non-Hodgkin ’s Lymphoma well state the types of criteria that insurers want to see before they will classify a therapy as “standard” and appropriate for coverage under general contract language:
...reliable prognostic features must be established to identify the patients for whom this treatment is necessary to achieve durable complete remissions and cure. At present the picture is blurred by the new regimes and uncertainty about improvements obtained.
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patients, any therapy of theoretical value no matter how remote the chance of benefit should be attempted. However, should we expect insurers or patients to pay for that attempt? The experience in the development of AZT, the FDA treatment IND proposal, and NCI’s category C have shown that outstanding drugs can get into the patient pipeline earlier. The current proposal by the Food and Drug Administration to expedite commercial approval for drugs needed for refractory patients or conditions without currently curative regimens, provided the drugs promise is sufficient to permit randomized controlled trials in Phase 11,would also carry an economic payoff-nce drugs are approved for a specific indication by the FDA, it is not likely that an insurer will refuse to cover costs for a patient, even if postmarketing surveillance looking suspiciously like Phase III trials is required (23).
WHEN ARE EXPERIMENTAL
THERAPIES COVERED UNDER THE STANDARD POLICY LANGUAGE?
The low numbers of truly refractory patients who have achieved durable complete remissions with megatherapy and ABMT suggest that this is not the optimal stage for the treatment (20). 2. Response rate is not generally equivalentto patient benefit. Applebaum and Buckner, in their 1986 survey article, Overview of the Clinical Relevance of Autologous Bone Marrow Transplantation,are very clear that response rates should not be equated with benefit to patients (21). These response rates, however, should not be interpreted to mean clinical benefit to the patient. In general, the duration of the response has been short with median duration of responses in most studies being less than 6 months and frequently only 2-4 months. Given the toxicities of these therapies and the amount of time required in hospital, it is difficult to conclude that these short-lived responses are worthwhile, and, in fact, few investigators working in this area make that claim (5, p. 5). Duration of response or disease-& survival and quality of life are the accepted indicia or endpoints of benefit to a patient (22). Certainly it is attractive to argue that if no therapy currently holds the promise of cure for a particular class of
Most insurance contracts define what they cover or exclude through some variation of the following:
To be reasonably necessary, a service or supply must be ordered by a doctor and be commonly and customarily recognized throughout the doctor’s profession as appropriate in the treatment of the diagnosed sickness or injury. It must be neither educational, experimental or investigational in nature, nor provided primarily for research.
HOW ARE THESE STANDARD CLAUSES APPLIED IN PRACTICE? 1. Erroneous or inadvertant payment does not compel continued coverage? Erroneous or inadvertent payments do not compel continued payment. It has been held that erroneous payments for therapies based upon the misconceptionthat they are standard therapies and not investigational will not compel insurers to continue paying, this “weapon” to compel payment would not be helpful in the majority of individually litigated cases. A case in point is Vincent B. Odenwaller v. Aem fife Insurance Company (24). In that case, local claims processors paid several claims for controversial experimentaltreatments without referring those
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Moving Ahead in Cancer Research
claims to the home office for review. The judge held that these erroneous payments did not render Aetna arbitrary and capricious if it denied other claims, and pointed out that claims of like nature referred to and reviewed at the home office level were steadfastly denied. 2. Is there an ambiguity in denial of coverage for participation in clinical trials as not medically necessary when (a) standard treatments have been exhausted or do not exist and (b) reference to appropriate clinical trials is in those circumstancesis considered “reasonable”and “standard” oncologic practice? An insurance contract is enforced according to its contract language unless there is an ambiguity in the contract language (25). (a) When there is no express exclusion relating to participation in experimentalhnvestigation therapies or research, coverage should generally be available. Case law precedent on this point is sparse. However, in the unpublished case of Petrovich v. Califontia WesternStates Life Insurance (26), the court was asked to consider whether a bone marrow transplant was “experimental” for treatment of childhood leukemia. From review of data showing that objective benefit in terms of disease-freesurvival from the use of this therapy had been achieved in a significant number of other pediatric cancer patients, the court held that the treatment was not experimental because it was a treatment of last resort, had the approval of the oncology community, and was necessary to save the life of the patient. (b) What if the contract expressly excludes experimentallinvestigationaltreatmentsand participation in research? Can that express exclusion be undone and rendered useless by a statement by the oncologic community that it is appropriate for patients who have exhausted or are refractory to available therapies to be referred to and to participate in investigational protocols? (i) Pructicul perspective. I anticipate, that insurers will meet any attempt to finesse their contract language through this mechanism by expanding their contract definitionof the exclusionto specificauy reference Phase I, Phase II and perhaps Phase IU trials. (ii) Legal Perspective. (a) Courts mcry nor rewire the insurance catnvrct d r the guise of commaion. It is a fundamental prhiple that a court cannot, by constructim of a contract, disregard words used by the parties or relieve one of the parties from t e r n which he voluntarily consented to (27). Stated differently, a court may not remake a contract in the guise of interpretation to implement an unexpected intention (28). Courts which force payment for experimental therapies under a “last resort” rationale where exptimental therapies are expressly exempted from coverage, are in effect rewiting the contract and rendering the exclusion meaningless.
89 Standard policies are meant to cover therapies which are proven and established as providing life extension or meaningful palliation. They are not intended to cover experimental therapies which use patients to collect data for research unless it can be demonstrated that enough is known about this therapy that this profile of patient can be expected to benefit in some meaningful way from the therapy. See Petrovich and Guilot at note 14. Clues in Point: Aurologous Bone Marrow Transplantationfor Breast Cancer. In ’Ihomas v. Guy Henlth Plan er al. (29), Judge Hand acknowledged sympathy for the patient and the financial burden she would bear if insurance did not pay for her treatment. However, he stated that “the administrator would not be fulfilling his fiduciary duty to expand the terms of coverageprovided by the plan” which excluded experimentallinvestigational therapies.
Judge Hand held that the insurer was not arbitrary in rejecting a claim for autologousbone marrow transplantation for refractory breast cancer under a contract that provided that medically necessary services do not include those that are “experimental or investigative,” even though: (a) the claimant’s treating physician had advised the claimant that conventional chemotherapy offers no real hope for major benefits and recommended either discontinuing treatment or pmeeding with an experimental treatment, (b) bone marrow harvesting was requested and precertified two years prior and claimant argued that this procedure was part of a treatment plan approved in case chemotherapyproved less effective for this breast cancer patient, (c) the insurer had inadvertently paid for this procedure in another case, and (d) some plans have considered ABMT payable for some subsets of cancer (30). 2. IL-2 for Thyroid Cancer [The Gaffney Compromise]. Claimant sought an injunction requiring the Kaiser Foundation Health Plan to pay for interleukin-2 for his thyroid cancer under a policy explicitly excluding experimental treatments from coverage (31). Kaiser had previously paid for his use of the then experimental drug interferon which may limit the usefulness of this decision in cases which do not involve inconsistent application of contract language. The court requiredthat Gaffney post a bond for $lO,OOO and that the IL2treatments be reimbursed by Kaiser pending a trial and final determination on the merits.
...it is evident that Gaffney will suffer irreparable harm either in the form of accelerated death or grave illness if he is not treated with one of the very few drugs which hold out any hope to plaintiff of respite from his rare cancer.
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The fact that Intezleukin II may not, in fact, cure G@ey’s cancer is only part of the story. The drug may offer him some hop, in the face of what is otherwise a hopeless situation.
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In mntmt to the %mas case, supra, the Judge permiued sentiment, expredy the treatments offer of ‘‘hope”
rather than the confumed possibility of “help” to invalidate the contract language excluding experimental therapies. 3. Isn’t a patient free to rely on getting reimbursement for any breabnentthat is prescribed by his or her onoologist regardless of the label placed on it? A patient’s bbunfettered right” to chose a physician and to follow his advice does not create a corresponding responsibility in the insurer to pay for those treatments. In Group Hospiitalizatm, Inc. v. Levin (32), the Court found g d y that it was: Not unmi&ul of the rising costs of medical insurance and recognizes that unless the term “necessary medical services” is given a literal meaaing, the availability of insurance...could be abusedby portieatS and oversolicitous physicians to the point of destroying the actuarial basis of current premium rates. In a case specfidy relating to an alleged “experbnen-
tal treatment,” Free v. Travelers Znsurance Co. (33), the court rejectad a claim for reimbursementfor the then unproven and dubious remedy laetrile (since disproved) and in so doing, w e d the criteria which usually would be applied to evaluation of experimentaVinvestigational therapbforpurposes. T h e m observed that: it is simply not enough to show that some people, even experts, have a belief in the safety and effectiveness of a particular drug. A reasonable number of people will attest to the worth of almost any product or idea (33, p 560).
The court concluded that to require insurers to pay for every remedy prescribed by a physician could invalidate the actuarial basis of current premium rates. (See notes 32, 33, and Anuuo, note 1, supra.) SOLUTIONS 1. Litigation is a crup shoot und no! a preferred way of solving cancer patients problems.
From the cases discussed and referenced, it is apparent that recourse to case by case litigation is a “crap shoot ” that does not solve the problem of reimbursementforparticipation in well-conceived research. Depending upon the basic documentationyou can bring to show the potential benefit of the proposed treatment, the “bent” of the judge and jury and the passion and attractiveness of your patient and the physician advocate for treatment, you might or you might notbe able to wrest payment from an insurer. This highly emotionally charged, time- and resourceconsuming, case by case contest approach to reimbursement is certainly neither an attractive solution or one available to most parties. If an ERISA (34) contract is involved, insurers only have to show that they were not arbitrary in refusing coverage. In non-ERISA contracts, insurers are usually able to demonstrate that their conclusion that treatment X is experimental is not arbitrary. Arbitrary or bad faith conduct in denial of a claim are the prerequisites for punitive damages. The possibility of collecting punitive damages is what makes a case attractive to a lawyer behg asked to take a case on a contingency fee basis instead of an up front hours and cost basis. Contingency litigation is the only avenue that patient finances would usually support; therefore, only rich patients will usually be able to interest an attorney in taking their case. 2. Government payment is the ethical and “right approach. ” The discussions by Lind and others, cited previously, support the payment of the patient supportive care costs for participation in research by the government. These patients usually cannot expect to benefit from a Phase I or Phase II trial themselves. Their participation in research is to help define the therapy for the benefit of those who will come after them. Their sacrifice moves the research and treatment effort forward to the public and social good (2,3,35). With a new administration in place and a Secretary of HHS with a special sensitivity to cancer treatment issues and the needs of minority patients, this may be the time for the oncology and patient communities to join hands and initiate a concerted, vocal lobbying effort to compel the government to shoulder this responsibility. If we do not believe that we can marshal1 sufficientforces to assert and advance our priorities over the zero based budget mindset, then we need to proceed to the next step. 3. Working with insurers, employers and unions to price out, gain acceptance for and implement inclusion of payment of the supportive patient care costs in standard policies.
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Moving Ahead in Cancer Research The taxpayingpublic subsidizese x p e b n t a l trial costs under either the government funding or insurer funding scenario. Insurers have inadvertentlybeen paying for a good deal of experimental therapies over the years. Tacitly, they have made actuarial provision for some of these costs. We need to help them get a handle on the rest of these costs and work with them to either rewrite policies to include clinical trial participation under stringent controls with suitable raises in premiums or work with them to offer a separate rider which would specifically cover participation in clinical trials at all phases. The benefit should expressly exclude picking up costs that have been traditionally borne by the pharmaceutical industry in seeking commercial approval of a product for market or seeking to add indications of the labeling. However, an unlabeled use of an approved drug should also be picked up by an insurer for a clinical trial which is not sponsored by a drug company.
A SUMMARY OF SOLUTIONS Curing cancers that have been resistant to standard approaches appears to require trial of more intensive, aggressive treatments that may increasingly require hospital monitoring to minimize side effects. The preferablemethod of accommodating these new aggressive research therapies is through provisions for payment of patient supportive care costs by the federal governmentagain under the p u b l i c l d benefit rationale. In the alternative the oncology community needs to work with insurers to: (a) determine the actuarial costs of changing their coverage to include participation in research including unlabeled drug use of approved products in clinical trials; (b) support in reducing costs where possible by agreeing on outmoded, ineffective cancer therapies and diagnostic procedures that are no longer “recommended”practice should not be paid for; and (c) obtaining premium increases where necessary. Why? The margin of profit on health insurance is very small (36) and insurers indicatethat they have not made actuarial provision for payment for investigational therapies. If forced to cover investigationaland experimental therapies without proper monetary underpinning, insurers may not have the reserves to pay for these therapies and with the return on the health insurance segment of the insurance business falling to 1% or less, they would be encouraged to exclude these therapies by name or to stop writing health benefits entirely if forced to pay before they can
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make fmncial provision for payment (36). There is a pattern established for this in the number of major carriers that by name excludehigh cost high technology treatments such as autologous bone marrow transplantation, heart or heart and lung transplants, liver transplants, etc. For the benefit of patients for whom.participation in clinical trials using investigational and experimental modalities may be the best and only option available(37), the medical care community and patients need to make common cause and work with insurers to educate unions, employers, the federal government, and the major selfinsurers such as the Fortune 500 companies to pick up this benefit. We also need to determine a mechanism whereby we cao determine outmoded and ineffectivetherapies and list them so that insurers wiU have some backup to stop covering them. The United States Pharmacopeial Convention panel process determines what drug through its cousages have become generally recognized as ready for incorporation into standard practice. May I suggest that the oncology community through one of its professional organizations such as the American Society of Clinical Oncology devise a similar consensus panel approach to assist in cost reduction. It we do not take these steps we can expect that: (a) Insurers will specifically amend their policies to exclude the high cost technologies in transition from coverage. (b) Insurers will probably amend their policies to expressly exclude participation in Phase I, II, or Ill trials. (c) Insurers will establish their own groups to determine what is appropriate versus unnecessary or excessive therapy. (d) Patients will be forced to forego participation in clinical trials that involve &$-cost care or mortgage the homestead to get it. Not a happy thought.
Address reprint requests to: Grace Powers Monaco,Candlelighters CCF, 1312 18th Street NW,Washington, DC 20036.
NOTES AND REFERENCES Rimarily supported since 1980 under an affiliationagreement betweenccm &the n a t i d officeof the Americancancer sociery. Sce, e.g., 2kbberg v. Blue Cross and Blue Sieki @Greater New Yo&, 487 NY2d 595 (NY App. 1985), Miskofsky v. Ohio Casualty Ins. CO., 497 A.2d 223 (N.J. Super. 1984), Wehmcycr v. Prudential, N.J. Sup. Ct. Civ. No. L-18383-81; RA., et a1 v. Prudential, Docket No. -3-79 N.J.Super. Ct. Law Div. August 6, 1982) bench slip opinion; Cf., WiLFm v. Travelers Ins. CO., 605 P.2d 1327, 1329 n5 (Okia. 1980). Weinberger v. Hjwon, Wesco%t& hmning, 412 U.S. 609 (1973). See 45 Fed. Reg. 37858,37860 (June 5,1980) at which the Health Care Financing Administration agreed to pay for drugs listed and made available for the treatment of certaintypes of cancerthrough
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5.
6.
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7.
8.
9.
10.
11. 12. 13.
14.
15.
the National Cancer Institute’s Cancer Therapy Evaluation Program since listing by NCI was believed to demonstrate efficacy. See discussion generally, Cline LS, Rosten, KA; 1985. “The Effect of Policy Language on the Containmentof Health Care Cost. ” Tort & Insurance L.J., 21:120, 122. See, e.g., M c h g M n v. Connectcut GeneraiLp Insurance Company, 565 F. Supp 434 (N.D. Cal., 1983), Wilson v. Travelers, 605 P.2d 329 ( m a . 1980), Zuckerberg v. Blue Cross and Blue Shield of Greater New York, 119 Misc. 2d 834,464 NYS 2d 678 (N.Y. Sup. 1983) reversed, 487 NY2d595 (App. 1985); Shwnoke v. Travelers Insurance Co., Docket No. 81-5769-CK, slip opinion (Mich. Co. Cir. Ct., Jan. 6, 1984). Group HospitalizoTion, Inc. v. Levin, 305 A.2d 248 (D.C. App., 1973): “p3e Caurt is] Not unmindful of the rising costs of medical insurance and recognizes that unless the term “necessary medical services” is given a literal meaning, the availability of insurance ... could be abused by patients and oversolicitous physicians to the point of destroying the actuarial basis of current p m i u m rates.” Zuckerberg v. Blue Cross and Blue Shield of Greater New York, 487 NYS 2d 595 (April 8, 1985): “...the denial of coverage by defendant Blue Cross for the Gerson therapy Mr. Zuckerberg received at La Gloria will not discourage the developmentand acceptance of legitimate innovative methods of treating cancer. It will, rather have the desirable effect of affording greater protection to the general public and, in palticular, cancer patients who are especially vulnerable to unfounded claims of miraculous cures. Specifically, our decision will insure that the treatment rendered on behalf of patients is administered in facilities comporting with certain minimal standards and that its effectivenesshas been adequately demonstrated by studies conducted in accordance with a p propriate scientific methodology before the resources of a major health insurer are utilized to support it.” Zuckerberg v. Blue Cross and Blue Shield of Greater New York, 487 NYS 2d 595 (“...ourdecision will insure that the treatment rendered on behalf of ptients.. .effectivenesshas been adequately demonstrated by studies conducted in accordance with appropriate scientific methodology before the resources of a major health insurer are utlized to support it.“); Misbjiky v. Ohio casualry Ins. Co., 497 A.2d 223 (N.J.Super. 1984). Antmann K, Schnipper LE,Frei E; 1988. Sounding Board. “Thirdparty insurance and investigation therapy. N Engl J Med 319(1): 46-48, 1988. Personal notes; Reimbursement Working Group Meetings, National Cancer Institute, 1988. CCCF insurance case file. See, e.g., Mr. Snai Hospital v. Zo& er al., 271 N.Y.S. 2d Series 1012, 1016, 1018 (NY,1966); Fcrssio v. MonraM Phys. Service, 553 P. 2d 998, 1OOO-1001 (Mont. 1976); Doe v. Srare of Minnesoria, 257 NW 2d 816, 821 @inn. 1977). See, e.g., Perrovich v. California Western Srates Life Insurance, Docket No. 3106-85, Sup. Ct. of Calif., SacramentoCounty, May 17, 19&1and Guilor v. Cherry, Docket No. 794371, U S . District Court for the Eastern District of Louisiana. Personal communication from Dr. Karen Antman and Dr. Robert Wines. The Cancer Therapy Evaluation Program has been collecting some data on the exposure of insurers to use in a dialogue with insurers on picking up costs.
16. Health Letter 3(10):1-4, 1987. 17. Lind SE: Soundingboard: Fee for service research. N Engl J Med 3 14:312-315, 1986. 18. See, e.g., notes 1,3,7,8 supra. 19. For example, aerosolized pentamidine, prophylactically for AIDS, is paid for under waiver by many companies on this basis or on the basis that it reduces costs by obviating the need for frequent hospitalizations. 20. Singer CR, Goldstone AH: Report on clinical studies of ABMT in non-Hodgkin’s lymphoma. Clin Haematol 15: 105-150, 1986. 21. Applebaum FR, Buckner CB: Overview of clinical relevance of autologous bone marrow transplantation. Clin Hematol 15: 1-18, 1986. 22. DeVita V: 1985. Cancer Principles and Practices of Oncology, 2nd ed. Lippincott, Philadelphia, 1985, p. 331. 23. Investigational new drug, antibiotic and biological drug product regulations; Procedures for drugs intended to treat life-treatening and severely debilitating illnesses; interim rule, Food and Drug Administration. 54:41516-41524, 1988. 24. See Vincent B. Odenwaller v. Aema fife Insurance Company, Docket No. K86283CA4. Opinion of May 27, 1988. U.S. District Court for the Western District of Michigan. 25. Yeng Sue Chow v. Levi Srrauss & Company, 122 Cal. Rptr. 816 (1975). 26. Perrovich v. California Western Srares Life Insurance, Docket No. 3106-85, Sup.Ct. of Calif., Sacramento County, May 17, 1984. S e e also, Guilor v. Cherry, Docket No. 79-4371,U.S. District Court for the Eastern District of Louisiana. 27. 17 Am. Jr. 2d Section 242. 28. Ross v. Special0 Insularion Mfg. Co., 410 NYSZd 28 (1978). 29. %mas v. Gulf Health Plan et al., Docket Civ. A. No. 884265-BH-M, Opinion of June 15, 1988. 30. According to Judge Hand, in February of 1988, the Blue Cross Medical review committee recommended that ABMT be classified as no longer experimental or investigational for some stages of Hodgkin’s and for neuroblastoma, ALL and ANLL. 31. &$hey v. Kaiser et al., No. 86-5367 (S.D., N.Y.) 32. Group Hospitalization, Inc. v. Levin, 305 A.2d 248 (D.C. App., 1973). 33. Free v. Travelers Insurance Company, 551 F. Supp. 554,560 (D. Ct. Md., 1982). 34. The Employee and Retirement Income Security Act (ERISA) of 1974 was intended to provide uniform federal regulation of employee benefit plans. As a result, ERISA exempts self-insured employee benefit plans, including health benefit plans, from state regulation. 35. The Belmont Report. DHEW Publication No. (05)78-0012, 1978, P 3‘ 36. Smith CE: Life insurance today: An actuaries view. J Insur Med 20:7-11, 1988. 37. Goodwin PJ, Feld R, Evans WK, Pater J: Cost-effectiveness of cancer chemotherapy: An economic evaluation of a randomized trial in small-cell lung cancer. J Clin Oncol61537-1547, 1988. Chemotherapy extended life significantly and was cheaper than supportive care alone for this class of unresectable cancer.
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PUBLIC ISSUES
Moving Ahead in Cancer Research: Who Pays for Patient Supportive Care for Participation in
Experimental/Investigational Trials? Grace Powers Monaco, J. D. Candlelighters Childhood Cancer Foundation 1312 18th Street NW Washington, DC 20036
INTRODUCTION
payment through stringent application of contract exclusions relating to experimental and investigational care. This “tightening” of reimbursement has occurred at the state medicaid level as well as in private and nonprofit insurance plans, and for the older cancer patient population, extends to medicare reimbursement through the federal government. I will review the background of the current policies of insurers on payment for patient supportive care costs associated with participation in investigational clinical trials, the interpretation of these policies by the courts, and options for crafting insurance benefits that purposefully and prudently cover investigational therapies with scientific merit. The premise of this report is that, except for compassionate and emergency use and NCI category C drugs, patients should receive experimental and investigational therapies only within the ambit of well-controlled clinical trials that are designed to collect data that will answer the question of patient benefit. I mention off label use of approved drugs only peripherally. I consider it abusive of insurers to refuse to pay for indicationsnot approved by the Food and Drug Administration when it is clear, for example, through
The Candlelighters Childhood Cancer Foundation (CCCF) (1) links and facilitates networking, advocacy, and communication among an international volunteer coalition begun more than 18 years ago which now involves 300 plus mutual support groups for parents whose children currently have or had cancer and their medical care teams in the United States, Canada, and around the world. One of the dominant advocacy roles of CCCF involves monitoring the responses of insurers to requests for payment for aggressive, potentially curative care for the young cancer patient and working with families and the medical care team to understand insurancecontract terms, maximize payment under existing policies and, where necessary, take action to change payment policies that threaten access to what our physicians have indicated is needed care. Most pediatric cancer patients participate in clinical protocols and, until recently, insurers have paid for most care prescribed for young cancer patients even if it wore the label of experimental and investigational. In the last two years there has been a consistent tightening of insurer 85 Copyright 0 1991 by Marcel Dekker, Inc.
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reference to the United State Pharmacopeia’s Drug Information for Physicians that certain unlabeled uses have become standard usage. I do not address the issue of when innovation in medical care crosses the line to experimental and investigational, but consider this a subject which needs to be addressed by the medical community. It is my opinion that participation in clinical trials is usually the best treatment available for refractory patients or patients with a conditionnot yet curable or controllable, and that payment for patient supportive care costs associated with participation in wellconceived clinical trials such as those listed on the National Cancer Institute’s Physician Data Query Program is a public benefit and social cost that should be borne by the federal government. However, unless the medical and patient communities are willing to march enmasse to Washington to demand appropriations to cover this cost, we will need to engage in a collaborative, collegial dialogue with insurers, employers, and unions to formulate mechanisms for private sector coverage.
THE HIRORY BEHIND INSURANCE POLICY EXCLUSIONS Most insurance policies cover reasonable and necessary medical care. This term is traditionally defined to mean care that is generally recognized as effective or of proven value in the management of a specific disease and to exclude treatments that have not met that standard, namely, that are still experimental, investigational, or constitute participation in research (2). This standard provision for coverage mirrors the criteria for drug approval by the Food and Drug Administration; generally recognized as effective as demonstrated by objective evidence produced in well-conceived clinical trials (3). It is also in accord with federal reimbursement policy (4). Until the mid-197Os, courts generally interpreted the term reasonable and necessary medical care as excluding therapies that were not generally recognized as effective (i.e., experimentalhnvestigational) (5). There followed a period of legal challenge in which courts took the position that absent express exclusion of experimentalhvestigational treatments or a specific listing of excluded therapies or definition of terms in a way that would support exclusion-treatments would be covered (6). These exclusions were not intended to control costs. Rather, they were intended to counter the potential for abuse of the insurance payment system by demanding patients and oversolicitousphysicians (7) and to support the appropriate use of premium dollars by providing a
mechanism for refusing payment for worthless therapies (quackery) (8) and therapies that were in controlled trials but had not yet demonstrated efficacy (9). Before health care dollars began to tighten up in the late 1970s and early 1980s, the experimental agent and at least some patient supportivecare costs, were covered under NIH grants (10). During that time frame, it appears that most experimentalprotocols were outpatient, involved hospitalization only for complications or tests associated with the research, and the costs of the drug being tested were, then as now, usually covered by the pharmaceutical company sponsoring the trial. Except for W a t r i c cancer treatments, the insurers weren’t seeing all that many claims for inpatient in-hospital supportive care costs associated with implementation of an experimental protocol (11). In pediatric cancer care involving aggressive therapy requiring in-hospital support, until recently, reimbursement was not usually a problem. Whether this is because of the track record of participation in clinical research protocols by most pediatric cancer patients and their acknowledged good track record or for some other reason is unclear (12). It is clear that until the last two years, in pediatric oncology, once the insurer was given a logical and demonstrable explanation of the need for aggressive treatment and provided with documentation to support the reasonable expectations for patient benefit and improved survival, reimbursement was forthcoming (13). In those cases in which insurer resistance is encountered, usually for predeterminationof coverage, a dialogue among the insurer, family and pediatric medical care team is initiated. If this dialogue reveals that the therapy is illfounded or a “crap shoot” for families (rare), challenge to refusal to pay rarely goes further. If, on the other hand, insurer resistance is arbitrary and denial of benefits is contested, childhood cancer families usually prevail (14). However, the times they are a changing! The components of cancer care in the adult population are changing to match pediatric cancer in aggression! The therapies being developed and tested to expand cure or control, primarily in adult cancers, such as autologous bone marrow transplantation, interleukins, and various LAK and TIL cell therapies, and so on, all may require extensive hospitalizations and supportive care. Insurers have indicated to me that lower level claims personnel have inadvertently paid for some of these therapies without realizing they were experimental or investigational. It is these therapies that they have now targeted for identificationat the lower claims level to have passed up for review at the management and technology
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Moving Ahead in Cancer Research
assessment levels for a determinationof whether they are excluded by contract language. In short, from the files of CCCF and my communications with insurers, the insurer position appears to be that (i) they have never agreed to pay for investigationaland experimental therapies whether delivered inside or outside a clinical trial or research setting, (ii) they have not made actuarial provision for coverage and their present profit margin could not absorb broadcast payments for these therapies without substantial premium adjustments, and (iii) inadvertentor erroneouspayment should not compel them to keep on paying. There is at present very little hard data that would permit us to understand the extent of insurer exposure and price out the real costs of coverage should insurers agree to pay for patient supportive care costs associated with participation in clinical trials. It has been indicated that Blue Cross and Blue Shield are looking into doing a prospectivestudy to delineatecosts for one of their programs (15). As far as I know, no insurer has taken a systematic retrospective look at the extent to which they are now, inadvertently, meeting the patient care costs associated with clinical trials.
WHO PAYS NOW? The federal government and other granting authorities should pay for the patient supportive care costs associated with research progress. But do they? Antmann and colleages acknowledgethat until recently NIH: provided important financial support for the care of patients in selected clinical research trials through grants to individual projects or clinical centers. Recently, however, funding for the costs of patient care has largely been deleted from research budgets, and these costs have been assumed by the patients, their health insurers, or both (10). The inclusion of patient care costs in grants to support clinical investigationis in accord with the historical ethic surrounding the conduct of clinical research. For example, Sidney Wolfe, Director of the Public Citizen Health Research Group, stated the generally accepted position on the issue of payment for participation in clinical investigation (16).
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One aspect of experimentaldrugs that many lay people (and, unfortunately, some physicians) do not seem to understand is that they are not legitimate articles of commerce, although the FDA of late has been acting as though they should be. Human medical experimentationof all kinds-emphatically including new drug investigations-are legally subject to rigid ethical standards, one of which is that human guinea pigs should not have to pay for the privilege of being experimented upon (2-4).
To the same effect see Lind (17): Whether patients can be asked to pay for research designed for personal therapeutic gain or for experimental treatment is a difficult question. It might be argued that patients should not be asked to pay for an unproved treatment, regardless of the severity of their disease or the ease with which they can affort to pay.
* * * Since miracles are rare and the first patient to receive a new treatment is unlikely to be cured, the burden of being the first to try a treatment should not be compounded by the need to pay for it (3, pp 314, 315). Traditionally, when actuarially determining risks and premiums, insurers have worked from the premise that therapies that have not been established through clinical trials as needed for patient benefit or palliation will be covered by another mechanism (18). There is no question that many insurers have paid. However, in the cases I have observed, payment has been: Due to the mistaken belief of lower level claims processors that the therapy was generally accepted or standard. Upon request for amwaiver, although tcchuidy experimental was established by studies and articles furnished by the treating physician to have shown sufficient expectation of patient benefit in survival or palliation that it warranted coverage (19). Payment for a class of therapy on an interim basis pending completion of clinical trials-this would include payment for NCI Category C drugs. Payment for unlabeled use8 listed in the USPDI.
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WHEN CAN WE CONSIDER THERAPIES READY FOR COVERAGE UNDER STANDARD POLICY LANGUAGE? 1. ’zheneed for prognostic factors to be &lined before patient benefit uin be implied and coverage extended under standard policy language. Singer and Goldstone in their report Clinical Stzufies of ABMT in Non-Hodgkin ’s Lymphoma well state the types of criteria that insurers want to see before they will classify a therapy as “standard” and appropriate for coverage under general contract language:
...reliable prognostic features must be established to identify the patients for whom this treatment is necessary to achieve durable complete remissions and cure. At present the picture is blurred by the new regimes and uncertainty about improvements obtained.
* * *
patients, any therapy of theoretical value no matter how remote the chance of benefit should be attempted. However, should we expect insurers or patients to pay for that attempt? The experience in the development of AZT, the FDA treatment IND proposal, and NCI’s category C have shown that outstanding drugs can get into the patient pipeline earlier. The current proposal by the Food and Drug Administration to expedite commercial approval for drugs needed for refractory patients or conditions without currently curative regimens, provided the drugs promise is sufficient to permit randomized controlled trials in Phase 11,would also carry an economic payoff-nce drugs are approved for a specific indication by the FDA, it is not likely that an insurer will refuse to cover costs for a patient, even if postmarketing surveillance looking suspiciously like Phase III trials is required (23).
WHEN ARE EXPERIMENTAL
THERAPIES COVERED UNDER THE STANDARD POLICY LANGUAGE?
The low numbers of truly refractory patients who have achieved durable complete remissions with megatherapy and ABMT suggest that this is not the optimal stage for the treatment (20). 2. Response rate is not generally equivalentto patient benefit. Applebaum and Buckner, in their 1986 survey article, Overview of the Clinical Relevance of Autologous Bone Marrow Transplantation,are very clear that response rates should not be equated with benefit to patients (21). These response rates, however, should not be interpreted to mean clinical benefit to the patient. In general, the duration of the response has been short with median duration of responses in most studies being less than 6 months and frequently only 2-4 months. Given the toxicities of these therapies and the amount of time required in hospital, it is difficult to conclude that these short-lived responses are worthwhile, and, in fact, few investigators working in this area make that claim (5, p. 5). Duration of response or disease-& survival and quality of life are the accepted indicia or endpoints of benefit to a patient (22). Certainly it is attractive to argue that if no therapy currently holds the promise of cure for a particular class of
Most insurance contracts define what they cover or exclude through some variation of the following:
To be reasonably necessary, a service or supply must be ordered by a doctor and be commonly and customarily recognized throughout the doctor’s profession as appropriate in the treatment of the diagnosed sickness or injury. It must be neither educational, experimental or investigational in nature, nor provided primarily for research.
HOW ARE THESE STANDARD CLAUSES APPLIED IN PRACTICE? 1. Erroneous or inadvertant payment does not compel continued coverage? Erroneous or inadvertent payments do not compel continued payment. It has been held that erroneous payments for therapies based upon the misconceptionthat they are standard therapies and not investigational will not compel insurers to continue paying, this “weapon” to compel payment would not be helpful in the majority of individually litigated cases. A case in point is Vincent B. Odenwaller v. Aem fife Insurance Company (24). In that case, local claims processors paid several claims for controversial experimentaltreatments without referring those
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Moving Ahead in Cancer Research
claims to the home office for review. The judge held that these erroneous payments did not render Aetna arbitrary and capricious if it denied other claims, and pointed out that claims of like nature referred to and reviewed at the home office level were steadfastly denied. 2. Is there an ambiguity in denial of coverage for participation in clinical trials as not medically necessary when (a) standard treatments have been exhausted or do not exist and (b) reference to appropriate clinical trials is in those circumstancesis considered “reasonable”and “standard” oncologic practice? An insurance contract is enforced according to its contract language unless there is an ambiguity in the contract language (25). (a) When there is no express exclusion relating to participation in experimentalhnvestigation therapies or research, coverage should generally be available. Case law precedent on this point is sparse. However, in the unpublished case of Petrovich v. Califontia WesternStates Life Insurance (26), the court was asked to consider whether a bone marrow transplant was “experimental” for treatment of childhood leukemia. From review of data showing that objective benefit in terms of disease-freesurvival from the use of this therapy had been achieved in a significant number of other pediatric cancer patients, the court held that the treatment was not experimental because it was a treatment of last resort, had the approval of the oncology community, and was necessary to save the life of the patient. (b) What if the contract expressly excludes experimentallinvestigationaltreatmentsand participation in research? Can that express exclusion be undone and rendered useless by a statement by the oncologic community that it is appropriate for patients who have exhausted or are refractory to available therapies to be referred to and to participate in investigational protocols? (i) Pructicul perspective. I anticipate, that insurers will meet any attempt to finesse their contract language through this mechanism by expanding their contract definitionof the exclusionto specificauy reference Phase I, Phase II and perhaps Phase IU trials. (ii) Legal Perspective. (a) Courts mcry nor rewire the insurance catnvrct d r the guise of commaion. It is a fundamental prhiple that a court cannot, by constructim of a contract, disregard words used by the parties or relieve one of the parties from t e r n which he voluntarily consented to (27). Stated differently, a court may not remake a contract in the guise of interpretation to implement an unexpected intention (28). Courts which force payment for experimental therapies under a “last resort” rationale where exptimental therapies are expressly exempted from coverage, are in effect rewiting the contract and rendering the exclusion meaningless.
89 Standard policies are meant to cover therapies which are proven and established as providing life extension or meaningful palliation. They are not intended to cover experimental therapies which use patients to collect data for research unless it can be demonstrated that enough is known about this therapy that this profile of patient can be expected to benefit in some meaningful way from the therapy. See Petrovich and Guilot at note 14. Clues in Point: Aurologous Bone Marrow Transplantationfor Breast Cancer. In ’Ihomas v. Guy Henlth Plan er al. (29), Judge Hand acknowledged sympathy for the patient and the financial burden she would bear if insurance did not pay for her treatment. However, he stated that “the administrator would not be fulfilling his fiduciary duty to expand the terms of coverageprovided by the plan” which excluded experimentallinvestigational therapies.
Judge Hand held that the insurer was not arbitrary in rejecting a claim for autologousbone marrow transplantation for refractory breast cancer under a contract that provided that medically necessary services do not include those that are “experimental or investigative,” even though: (a) the claimant’s treating physician had advised the claimant that conventional chemotherapy offers no real hope for major benefits and recommended either discontinuing treatment or pmeeding with an experimental treatment, (b) bone marrow harvesting was requested and precertified two years prior and claimant argued that this procedure was part of a treatment plan approved in case chemotherapyproved less effective for this breast cancer patient, (c) the insurer had inadvertently paid for this procedure in another case, and (d) some plans have considered ABMT payable for some subsets of cancer (30). 2. IL-2 for Thyroid Cancer [The Gaffney Compromise]. Claimant sought an injunction requiring the Kaiser Foundation Health Plan to pay for interleukin-2 for his thyroid cancer under a policy explicitly excluding experimental treatments from coverage (31). Kaiser had previously paid for his use of the then experimental drug interferon which may limit the usefulness of this decision in cases which do not involve inconsistent application of contract language. The court requiredthat Gaffney post a bond for $lO,OOO and that the IL2treatments be reimbursed by Kaiser pending a trial and final determination on the merits.
...it is evident that Gaffney will suffer irreparable harm either in the form of accelerated death or grave illness if he is not treated with one of the very few drugs which hold out any hope to plaintiff of respite from his rare cancer.
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The fact that Intezleukin II may not, in fact, cure G@ey’s cancer is only part of the story. The drug may offer him some hop, in the face of what is otherwise a hopeless situation.
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In mntmt to the %mas case, supra, the Judge permiued sentiment, expredy the treatments offer of ‘‘hope”
rather than the confumed possibility of “help” to invalidate the contract language excluding experimental therapies. 3. Isn’t a patient free to rely on getting reimbursement for any breabnentthat is prescribed by his or her onoologist regardless of the label placed on it? A patient’s bbunfettered right” to chose a physician and to follow his advice does not create a corresponding responsibility in the insurer to pay for those treatments. In Group Hospiitalizatm, Inc. v. Levin (32), the Court found g d y that it was: Not unmi&ul of the rising costs of medical insurance and recognizes that unless the term “necessary medical services” is given a literal meaaing, the availability of insurance...could be abusedby portieatS and oversolicitous physicians to the point of destroying the actuarial basis of current premium rates. In a case specfidy relating to an alleged “experbnen-
tal treatment,” Free v. Travelers Znsurance Co. (33), the court rejectad a claim for reimbursementfor the then unproven and dubious remedy laetrile (since disproved) and in so doing, w e d the criteria which usually would be applied to evaluation of experimentaVinvestigational therapbforpurposes. T h e m observed that: it is simply not enough to show that some people, even experts, have a belief in the safety and effectiveness of a particular drug. A reasonable number of people will attest to the worth of almost any product or idea (33, p 560).
The court concluded that to require insurers to pay for every remedy prescribed by a physician could invalidate the actuarial basis of current premium rates. (See notes 32, 33, and Anuuo, note 1, supra.) SOLUTIONS 1. Litigation is a crup shoot und no! a preferred way of solving cancer patients problems.
From the cases discussed and referenced, it is apparent that recourse to case by case litigation is a “crap shoot ” that does not solve the problem of reimbursementforparticipation in well-conceived research. Depending upon the basic documentationyou can bring to show the potential benefit of the proposed treatment, the “bent” of the judge and jury and the passion and attractiveness of your patient and the physician advocate for treatment, you might or you might notbe able to wrest payment from an insurer. This highly emotionally charged, time- and resourceconsuming, case by case contest approach to reimbursement is certainly neither an attractive solution or one available to most parties. If an ERISA (34) contract is involved, insurers only have to show that they were not arbitrary in refusing coverage. In non-ERISA contracts, insurers are usually able to demonstrate that their conclusion that treatment X is experimental is not arbitrary. Arbitrary or bad faith conduct in denial of a claim are the prerequisites for punitive damages. The possibility of collecting punitive damages is what makes a case attractive to a lawyer behg asked to take a case on a contingency fee basis instead of an up front hours and cost basis. Contingency litigation is the only avenue that patient finances would usually support; therefore, only rich patients will usually be able to interest an attorney in taking their case. 2. Government payment is the ethical and “right approach. ” The discussions by Lind and others, cited previously, support the payment of the patient supportive care costs for participation in research by the government. These patients usually cannot expect to benefit from a Phase I or Phase II trial themselves. Their participation in research is to help define the therapy for the benefit of those who will come after them. Their sacrifice moves the research and treatment effort forward to the public and social good (2,3,35). With a new administration in place and a Secretary of HHS with a special sensitivity to cancer treatment issues and the needs of minority patients, this may be the time for the oncology and patient communities to join hands and initiate a concerted, vocal lobbying effort to compel the government to shoulder this responsibility. If we do not believe that we can marshal1 sufficientforces to assert and advance our priorities over the zero based budget mindset, then we need to proceed to the next step. 3. Working with insurers, employers and unions to price out, gain acceptance for and implement inclusion of payment of the supportive patient care costs in standard policies.
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Moving Ahead in Cancer Research The taxpayingpublic subsidizese x p e b n t a l trial costs under either the government funding or insurer funding scenario. Insurers have inadvertentlybeen paying for a good deal of experimental therapies over the years. Tacitly, they have made actuarial provision for some of these costs. We need to help them get a handle on the rest of these costs and work with them to either rewrite policies to include clinical trial participation under stringent controls with suitable raises in premiums or work with them to offer a separate rider which would specifically cover participation in clinical trials at all phases. The benefit should expressly exclude picking up costs that have been traditionally borne by the pharmaceutical industry in seeking commercial approval of a product for market or seeking to add indications of the labeling. However, an unlabeled use of an approved drug should also be picked up by an insurer for a clinical trial which is not sponsored by a drug company.
A SUMMARY OF SOLUTIONS Curing cancers that have been resistant to standard approaches appears to require trial of more intensive, aggressive treatments that may increasingly require hospital monitoring to minimize side effects. The preferablemethod of accommodating these new aggressive research therapies is through provisions for payment of patient supportive care costs by the federal governmentagain under the p u b l i c l d benefit rationale. In the alternative the oncology community needs to work with insurers to: (a) determine the actuarial costs of changing their coverage to include participation in research including unlabeled drug use of approved products in clinical trials; (b) support in reducing costs where possible by agreeing on outmoded, ineffective cancer therapies and diagnostic procedures that are no longer “recommended”practice should not be paid for; and (c) obtaining premium increases where necessary. Why? The margin of profit on health insurance is very small (36) and insurers indicatethat they have not made actuarial provision for payment for investigational therapies. If forced to cover investigationaland experimental therapies without proper monetary underpinning, insurers may not have the reserves to pay for these therapies and with the return on the health insurance segment of the insurance business falling to 1% or less, they would be encouraged to exclude these therapies by name or to stop writing health benefits entirely if forced to pay before they can
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make fmncial provision for payment (36). There is a pattern established for this in the number of major carriers that by name excludehigh cost high technology treatments such as autologous bone marrow transplantation, heart or heart and lung transplants, liver transplants, etc. For the benefit of patients for whom.participation in clinical trials using investigational and experimental modalities may be the best and only option available(37), the medical care community and patients need to make common cause and work with insurers to educate unions, employers, the federal government, and the major selfinsurers such as the Fortune 500 companies to pick up this benefit. We also need to determine a mechanism whereby we cao determine outmoded and ineffectivetherapies and list them so that insurers wiU have some backup to stop covering them. The United States Pharmacopeial Convention panel process determines what drug through its cousages have become generally recognized as ready for incorporation into standard practice. May I suggest that the oncology community through one of its professional organizations such as the American Society of Clinical Oncology devise a similar consensus panel approach to assist in cost reduction. It we do not take these steps we can expect that: (a) Insurers will specifically amend their policies to exclude the high cost technologies in transition from coverage. (b) Insurers will probably amend their policies to expressly exclude participation in Phase I, II, or Ill trials. (c) Insurers will establish their own groups to determine what is appropriate versus unnecessary or excessive therapy. (d) Patients will be forced to forego participation in clinical trials that involve &$-cost care or mortgage the homestead to get it. Not a happy thought.
Address reprint requests to: Grace Powers Monaco,Candlelighters CCF, 1312 18th Street NW,Washington, DC 20036.
NOTES AND REFERENCES Rimarily supported since 1980 under an affiliationagreement betweenccm &the n a t i d officeof the Americancancer sociery. Sce, e.g., 2kbberg v. Blue Cross and Blue Sieki @Greater New Yo&, 487 NY2d 595 (NY App. 1985), Miskofsky v. Ohio Casualty Ins. CO., 497 A.2d 223 (N.J. Super. 1984), Wehmcycr v. Prudential, N.J. Sup. Ct. Civ. No. L-18383-81; RA., et a1 v. Prudential, Docket No. -3-79 N.J.Super. Ct. Law Div. August 6, 1982) bench slip opinion; Cf., WiLFm v. Travelers Ins. CO., 605 P.2d 1327, 1329 n5 (Okia. 1980). Weinberger v. Hjwon, Wesco%t& hmning, 412 U.S. 609 (1973). See 45 Fed. Reg. 37858,37860 (June 5,1980) at which the Health Care Financing Administration agreed to pay for drugs listed and made available for the treatment of certaintypes of cancerthrough
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5.
6.
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7.
8.
9.
10.
11. 12. 13.
14.
15.
the National Cancer Institute’s Cancer Therapy Evaluation Program since listing by NCI was believed to demonstrate efficacy. See discussion generally, Cline LS, Rosten, KA; 1985. “The Effect of Policy Language on the Containmentof Health Care Cost. ” Tort & Insurance L.J., 21:120, 122. See, e.g., M c h g M n v. Connectcut GeneraiLp Insurance Company, 565 F. Supp 434 (N.D. Cal., 1983), Wilson v. Travelers, 605 P.2d 329 ( m a . 1980), Zuckerberg v. Blue Cross and Blue Shield of Greater New York, 119 Misc. 2d 834,464 NYS 2d 678 (N.Y. Sup. 1983) reversed, 487 NY2d595 (App. 1985); Shwnoke v. Travelers Insurance Co., Docket No. 81-5769-CK, slip opinion (Mich. Co. Cir. Ct., Jan. 6, 1984). Group HospitalizoTion, Inc. v. Levin, 305 A.2d 248 (D.C. App., 1973): “p3e Caurt is] Not unmindful of the rising costs of medical insurance and recognizes that unless the term “necessary medical services” is given a literal meaning, the availability of insurance ... could be abused by patients and oversolicitous physicians to the point of destroying the actuarial basis of current p m i u m rates.” Zuckerberg v. Blue Cross and Blue Shield of Greater New York, 487 NYS 2d 595 (April 8, 1985): “...the denial of coverage by defendant Blue Cross for the Gerson therapy Mr. Zuckerberg received at La Gloria will not discourage the developmentand acceptance of legitimate innovative methods of treating cancer. It will, rather have the desirable effect of affording greater protection to the general public and, in palticular, cancer patients who are especially vulnerable to unfounded claims of miraculous cures. Specifically, our decision will insure that the treatment rendered on behalf of patients is administered in facilities comporting with certain minimal standards and that its effectivenesshas been adequately demonstrated by studies conducted in accordance with a p propriate scientific methodology before the resources of a major health insurer are utilized to support it.” Zuckerberg v. Blue Cross and Blue Shield of Greater New York, 487 NYS 2d 595 (“...ourdecision will insure that the treatment rendered on behalf of ptients.. .effectivenesshas been adequately demonstrated by studies conducted in accordance with appropriate scientific methodology before the resources of a major health insurer are utlized to support it.“); Misbjiky v. Ohio casualry Ins. Co., 497 A.2d 223 (N.J.Super. 1984). Antmann K, Schnipper LE,Frei E; 1988. Sounding Board. “Thirdparty insurance and investigation therapy. N Engl J Med 319(1): 46-48, 1988. Personal notes; Reimbursement Working Group Meetings, National Cancer Institute, 1988. CCCF insurance case file. See, e.g., Mr. Snai Hospital v. Zo& er al., 271 N.Y.S. 2d Series 1012, 1016, 1018 (NY,1966); Fcrssio v. MonraM Phys. Service, 553 P. 2d 998, 1OOO-1001 (Mont. 1976); Doe v. Srare of Minnesoria, 257 NW 2d 816, 821 @inn. 1977). See, e.g., Perrovich v. California Western Srates Life Insurance, Docket No. 3106-85, Sup. Ct. of Calif., SacramentoCounty, May 17, 19&1and Guilor v. Cherry, Docket No. 794371, U S . District Court for the Eastern District of Louisiana. Personal communication from Dr. Karen Antman and Dr. Robert Wines. The Cancer Therapy Evaluation Program has been collecting some data on the exposure of insurers to use in a dialogue with insurers on picking up costs.
16. Health Letter 3(10):1-4, 1987. 17. Lind SE: Soundingboard: Fee for service research. N Engl J Med 3 14:312-315, 1986. 18. See, e.g., notes 1,3,7,8 supra. 19. For example, aerosolized pentamidine, prophylactically for AIDS, is paid for under waiver by many companies on this basis or on the basis that it reduces costs by obviating the need for frequent hospitalizations. 20. Singer CR, Goldstone AH: Report on clinical studies of ABMT in non-Hodgkin’s lymphoma. Clin Haematol 15: 105-150, 1986. 21. Applebaum FR, Buckner CB: Overview of clinical relevance of autologous bone marrow transplantation. Clin Hematol 15: 1-18, 1986. 22. DeVita V: 1985. Cancer Principles and Practices of Oncology, 2nd ed. Lippincott, Philadelphia, 1985, p. 331. 23. Investigational new drug, antibiotic and biological drug product regulations; Procedures for drugs intended to treat life-treatening and severely debilitating illnesses; interim rule, Food and Drug Administration. 54:41516-41524, 1988. 24. See Vincent B. Odenwaller v. Aema fife Insurance Company, Docket No. K86283CA4. Opinion of May 27, 1988. U.S. District Court for the Western District of Michigan. 25. Yeng Sue Chow v. Levi Srrauss & Company, 122 Cal. Rptr. 816 (1975). 26. Perrovich v. California Western Srares Life Insurance, Docket No. 3106-85, Sup.Ct. of Calif., Sacramento County, May 17, 1984. S e e also, Guilor v. Cherry, Docket No. 79-4371,U.S. District Court for the Eastern District of Louisiana. 27. 17 Am. Jr. 2d Section 242. 28. Ross v. Special0 Insularion Mfg. Co., 410 NYSZd 28 (1978). 29. %mas v. Gulf Health Plan et al., Docket Civ. A. No. 884265-BH-M, Opinion of June 15, 1988. 30. According to Judge Hand, in February of 1988, the Blue Cross Medical review committee recommended that ABMT be classified as no longer experimental or investigational for some stages of Hodgkin’s and for neuroblastoma, ALL and ANLL. 31. &$hey v. Kaiser et al., No. 86-5367 (S.D., N.Y.) 32. Group Hospitalization, Inc. v. Levin, 305 A.2d 248 (D.C. App., 1973). 33. Free v. Travelers Insurance Company, 551 F. Supp. 554,560 (D. Ct. Md., 1982). 34. The Employee and Retirement Income Security Act (ERISA) of 1974 was intended to provide uniform federal regulation of employee benefit plans. As a result, ERISA exempts self-insured employee benefit plans, including health benefit plans, from state regulation. 35. The Belmont Report. DHEW Publication No. (05)78-0012, 1978, P 3‘ 36. Smith CE: Life insurance today: An actuaries view. J Insur Med 20:7-11, 1988. 37. Goodwin PJ, Feld R, Evans WK, Pater J: Cost-effectiveness of cancer chemotherapy: An economic evaluation of a randomized trial in small-cell lung cancer. J Clin Oncol61537-1547, 1988. Chemotherapy extended life significantly and was cheaper than supportive care alone for this class of unresectable cancer.
