Reimbursement of Biotherapy: Present Status, Future Directions -Perspectives of the Hospital-Based Oncology Nurse Mary S. McCabe
2 (IL-2). All of these agents are currently benefiting cancer patients in a variety of circumstances and clinical settings. In addition, many promising biotherapies are in development. Examples include X-6, which affects platelets and exerts antitumor effects, and all-trans retinoic acid, which is active in forms of leukemia and appears useful in other malignancies. ’ Investigational agents such as these represent important, if not the best, clinical options for many patients, given the severe, life-threatening nature of malignant disease and the less than successful state of many current treatments. Investigational therapy, therefore, is important to oncology nurses, especially those providing care in cancer centers, outpatient departments, and oncology clinical research departments. Nurses should be informed about the drug development process, the therapeutic potential of investigational therapy, and the financial impediments patients face in obtaining access to these promising new treatments.
Cancer is a set of diseases that affects one in four Americans and which is too often fatal. For many types of cancer, there is no fully satisfactory treatment. Therefore, investigational therapy provided in a formal, peerreviewed clinical trial can present the best available treatment for patients with life-threatening malignancies. At a time when clinical cancer research is at a particularly promising point and multiple new biologics are on the horizon, the traditional partnership of the federal government, the pharmaceutical industry, private institutions, and insurers is threatened. The issue of increasing concern is the impact of changes in third-party reimbursement policy on cancer clinical investigation. Although its incidence, magnitude, and geographic distribution have not been specifically defined, this issue is generating serious concern and intense national debate. The growing denials of patient-care costs related to clinical trials serve to threaten patient access to potentially efficacious therapy. The implications of this reimbursement issue are tremendous for both clinical investigation and patient care. The refusal to pay for patient-care costs related to investigational therapy may adversely influence clinical trial design and will certainly affect the speed with which new therapies are developed. When insurers refuse to cover these dinical costs, only affluent individuals have access to the most promising treatments. Nursing actions can positively affect the reimbursement debate. Nurses must develop cost-efficient plans of care and the means to evaluate their effectiveness. Educating patients about and assisting them with reimbursement issues maximizes their prospects for receiving appropriate investigational therapy. Good communication and ongoing discussions with local insurers are essential. Nurses should be aware of their potential contribution in this important issue and should participate in this debate both locally, in the clinical setting, and nationally, through professional organizations. This is a US government work Them are no restrictions on its use.
CLINICAL TRIALS
The National Cancer Institute (NCI) clinical trials program is important because of the commitment of investigators on a national level and the impact it has on the development of more effective cancer treatments. The total number of participants in NC1 trials is small-less than 30,000 patients per year-compared with the 1,250,000 Americans diagnosed with cancer each year.2 However, thousands of nurses and physicians in more than 1,300 hospitals and institutions across the country conduct NC1 trials, and the benefit to the patients involved can be tre-
ECENT ADVANCES in biotechnology have led to the development of important new biologic cancer agents. Beginning with interferon alfa, these drugs have been identified, formulated, tested, and marketed at an ever-accelerating pace. The future holds possibilities for increasingly successful cancer therapies, as evidenced by recently approved biologics such as erythropoietin (EPG), granulocyte and granulocyte-macrophage colony-stimulating factors (G-CSF and GM-CSF, respectively), and, as of May 5, 1992, interleukin-
R
~e~ham
in
Onmlogy Nursing.
Vol8,
No 4. Suppl
1 (November),
PROGRAM
From the Division of Cancer Treatment, National Cancer Institute, Bethesda, MD. Address reprint requests to Mary S. McCabe, RN, BA, BS, Clinical Trials Specialist, National Cancer Institute, Division of Cancer Treatment, Bethesda, MD 20892. This is a US government work. There are no restrictions on its use. 0749-2081/92/0804-IOO4%05.00/0 1992:
pp 3-7
3
MARY
mendous. Any disruption of this research program can potentially impede improvements in cancer treatment and thereby affect all future cancer patients. INVESTIGATIONAL
THERAPY
It is important that scientifically valid clinical research with investigational agents be distinguished from unscientific, unscrupulous use of cancer treatments that may be wrongly labeled “cancer research.” The term “investigational therapy” is intended to include treatment administered as part of a formal, peer-reviewed clinical trial. New drugs proceed through an established sequence of clinical trials in which knowledge builds from one stage to another, and continued evaluation depends on meaningful positive results. The clinical trials process comprises three phases, each offering therapeutic potential to participating patients: (1) phase I-identification of the maximal safe dose; (2) phase II-evaluation of efficacy; and (3) phase III-randomized comparison against the standard therapy. This process is designed to assure that clinical cancer investigation proceeds in an orderly sequence. However, because the clinical trials process yields cumulative information, important data about the benefits of a new therapy may become available before completion of the process or Food and Drug Administration (FDA) approval of the therapy. Because of this continuum of knowledge, the distinction between investigational or experimental and “standard” therapy is often blurred.3 Thus, this distinction, although often defined and codified, may not be the best way to determine insurance coverage for life-threatening disease. GROUP C ANTICANCER
types. There is an appreciable time period before these drugs, sponsored by the pharmaceutical industry, receive NDA [New Drug Application] approval. In order to allow patients with these diseases to have the benefit of this drug therapy, a Group C drug classification has been developed. The purpose of this distribution is to allow accessto beneficial therapy and to acquire information on safety in the context in which the drug is likely to be used in clinical practice after marketing.“”
For a drug to qualify for Group C status, a review of abstracts, papers, and reports must show that it has reproducible efficacy against a specific tumor type, that it alters the pattern of care of the disease, and that it may be administered safely by properly trained physicians without the need for special supportive care facilities.’ The one exception to this last requirement is IL-2/lymphokine-activated killer cells (LAK) which, because of the additional laboratory and safety monitoring it entails, was placed in a Modified Group C category. Drugs included in this modified category meet all other Group C criteria, including efficacy. Early in the history of the Group C program, drugs remained in this category for extended periods, in part because of the lack of a commercial sponsor. But more recently, the Group C category has been operating as a brief bridge between the identification of activity and FDA approval. Table 1 lists current Group C drugs and their indications. Recently approved drugs formerly in Group C include ifosfamide/mesna, deoxycoformycin (pentostatin), levamisole. fludarabine phosphate, and IL-2. Despite the formal review and strong demonstration of benefit accompanying drugs placed in this Group C category, there are no assurances that patient care costs associated with such ther-
DRUGS
Recognizing that important knowledge about the activity and benefit of a drug becomes evident even while a drug continues through the evaluation process, the NC1 and the FDA in 1976 established a category of drugs called Group C. The purpose of this category is to make active agents more rapidly and widely available before FDA approval. In the words of the NCI: “During the evaluation of investigational drugs by the Division of Cancer Treatment, NCI, certain drugs are found to have a role in the treatment of specific tumor
S. MCCABE
Table
1.
Grow
C Druas
and
Their
Group C Drug
Indications Indication
Asparaginase-erwmia
Acute lymphocytlc patients sensitive
Amsacrine
Refractory adult myelogenous
Azactldine
Refractory
Taxol Teniposide
leukemia Refractory ovarian carcinoma Relpased or refractory acute lymphoblastic leukemia
2Xhlorodeoxyadenosine
Active
asparaginase
(Escherichia
acute
hairy
leukemia to acute leukemia myelogenous
cell leukemia
co/i)
PERSPECTIVES
OF THE HOSPITAL
ONCOLOGY
apy will be reimbursed. The Health Care Financing Administration (HCFA), which administers the federal Medicare program, published formal guidelines in 1982, which recently have been restated, that “a Group C drug and the related hospital stay are covered if all other applicable coverage requirements are satisfied.“6 However, this statement of coverage is not necessarily transferable to other insurers, and many choose not to cover this important group of agents. Even the threat of non-reimbursement is a strong disincentive to the use of these drugs, and such discouragement is indefensible. Only the treatment-related costs are, in fact, at issue, since the drugs themselves are distributed free of charge by the NCI, and there is no expectation that this policy will change. EVOLVING
TRENDS
AND ISSUES
At this scientifically exciting juncture, debate is increasing over the cost of the United States health care system, estimated at $700 billion per annum.7 Concern is mounting as to how the current system will address the rapid advance of expensive technologies, the growing number of elderly, and the increasing number of uninsured and underinsured citizens, while at the same time stabilizing expenditures. The American health care system is in crisis because of escalating costs. Daily, the popular press and professional journals report the mounting problems. In the current health care climate, economic considerations are an integral part of any planning of research and care. They cross all subspecialty borders, but are particularly important in oncology. At no other time have financial issues and decisions so influenced the responsibilities of oncology nurses. REIMBURSEMENT
5
NURSE
OF INVESTIGATIONAL THERAPY
Third-party reimbursement for patients receiving investigational agents in clinical trials is a key element of the cancer economics discussion because such research is an essential and wellestablished health care priority. Historically, there has been a partnership of payers including the federal government, private institutions such as universities, the pharmaceutical industry, and third-party insurers.8 This coalition is changing, threatening reimbursement for both the research and patient care components of clinical trials.
There is a growing perception that lack of reimbursement is having a serious impact on current cancer patients and has implications for drug development, and thus for patients, now and in the future. Volgelzang and Richards at the University of Chicago have documented delayed completion and early closure of studies because of reimbursement problems with biologics such as IL-2.9 Such difficulties slow the development of new therapies. Reimbursement restrictions also could decrease the cost effectiveness of drug development, if the design of studies were determined by insurance coverage rather than by the pursuit of doses, schedules, and delivery systems designed to produce the best possible clinical outcome. Moreover, reimbursement restrictions could lead to reduced access to promising new therapies and to an elitist system of health care, with only patients who could afford the full cost entering clinical trials. Including only affluent patients may also skew clinical results, since socioeconomic factors may affect patients’ general health and response to therapy. Presently, a serious reimbursement problem is occurring with taxol, which, although not a biologic, is one of the most important cancer agents currently in development. Nationwide, patients are having difficulty securing insurance coverage for treatment through the NC1 Treatment Referral Center, despite strong data supporting its efficacy in refractory ovarian carcinoma.” Further problems in the reimbursement of clinical trials stem from the decentralized nature of the health insurance industry, which results in inconsistent coverage policies. Because insurers are regulated at the state rather than the national level, reimbursement policies can differ from state to state. A patient covered for a therapy in one state may lose this coverage on moving to another state. Even Medicare, although a national program, suffers from inconsistent reimbursement policies, because unless HCFA has developed a national policy for a therapy, local intermediaries are allowed to determine coverage. CONSENSUS FOR REIMBURSEMENT OF INVESTIGATIONAL THERAPY
Growing concern over the lack of reimbursement for investigational therapy has prompted a number of national organizations and coalitions
6
MARY
to study this issue and publicly present statements and recommendations to address it. For example, in 1988, the Institute of Medicine issued a report declaring that: “Third-party payers (government and non-govemment) should pay the necessary and appropriate patient care costs for beneficiaries enrolled in approved clinical investigational protocols. This requires a clarification in current Medicare regulations involving definitions of medically necessarycare. State regulatory agencies should require conforming changes by all other third party payer policies.” “There are diseasesfor which appropriate and required care involves investigational protocols. Such diseases include certain types of cancer, genetic diseases, and possibly other severe, life threatening diseases.In these cases, third party payers (government and nongovernment) should pay the standard patient care costs while costs related to the investigational conclusions should be borne by the sponsoring agency.“”
The American Society of Clinical Oncology (ASCO), which represents oncologists, released a similar position statement in 1990: “Both Medicare and private insurers should accept the views of experts to the effect that treatment under a clinical protocol represents the best available therapy for critically ill cancer patients and that patient care costs, e.g., the cost of hospitalization-should be borne by insurers.“‘*
A group of health care professionals, attorneys, and administrators convened by the NC1 developed a consensus statement advocating that: ‘6 . third-party coverage be allowed for patient-care costs of all nationally approved (NC1 or FDA) cancer treatment research protocols and that third party coverage should also be allowed for all cancer treatment research protocols not subject to national approval, provided these protocols have been approved by established peer review mechanisms .‘r8
An important group convened by then Vice President Bush, the National Committee to Review Current Procedures for Approval of New Drugs for Cancer and AIDS (the Lasagna Committee), also supported this position, recommending that: “The cost of investigational drugs, and marketed drugs prescribed for unlabeled indications, as well as all ancillary medical care, should be covered by Medicare, Medicaid, and private insurance, if the use has been approved by expert government agencies, in authoritative medical compendia, or by a committee established by the Secretary of Health and Human Services to deal with this matter.“13
S. MCCABE
The Association of Community Cancer Centers (ACCC), another important oncology organization, also has supported reimbursement for clinical trials. I4 However, despite these strong, consistent recommendations of national health care leaders and formal scholarly groups, reimbursement problems continue to go unresolved. Too often, the courts determine reimbursement for investigational therapy. All groups of health care providers, patients, and insurers seeking to resolve this serious issue acknowledge that such a pattern is inappropriate and adversarial. RECOMMENDATIONS
FOR NURSING ACTION
To be informed is one of the principal responsibilities nurses have when interacting with patients seeking information about clinical trials or when caring for those receiving investigational therapy. The economic issues related to such treatment are too integral to oncology nurses’ professional roles to be ignored. First, it is important to know the process of working with insurers. For nurses in the hospital setting, this may mean assisting and educating patients to negotiate with representatives of their insurance plans. For nurses in the outpatient setting, this may involve communicating directly with insurance companies about a clinical trial. It often is helpful to (1) assemble packets of appropriate data for review by the insurance company’s medical director; (2) update local plans about important studies at the institution; and (3) provide data demonstrating the standards of excellence of both clinical investigators and ongoing clinical trials. Many cancer centers have developed good working relationships with insurers in their area. Insurers see them as “centers of excellence” because of the quality of patient care and their use of appropriate, scientifically sound therapies that offer patients potential for real benefit. In addition to involving themselves in these institutional reimbursement issues, nurses can pursue individual activities that address the need to be economically efficient. Most important is the development of a care plan that takes into consideration (1) potential self-care options for patients, such as self-injection or the use of ambulatory infusion pumps; (2) the choice of an economical, but safe and comfortable, treatment
PERSPECTIVES
OF THE
HOSPITAL
ONCOLOGY
NURSE
7
setting for the patient; and (3) discharge planning strategies to minimize hospital stays and provide for continuity of care at home. Patients themselves have a need and responsibility to be informed of the financial issues related to the investigational therapy being considered. They generally assume that their insurance covers such therapy, and frequently are frightened and angry to discover that this is not the case. Thus, nurses should view patient education on financial issues as part of the informed consent process. Pharmaceutical companies provide reimbursement information services and indigent patient programs. A listing appears in the article by Xistris in this supplement. Instituting change in state and national policies on reimbursement for investigational therapy can and should involve nurses. Oncology nurses should join community cancer groups working to educate and influence insurers and officials. Membership in national organizations, such as the American Cancer Society, that promote the
development of new cancer therapies, can increase support for reimbursement. The Board of Directors of the Oncology Nursing Society has identified reimbursement for clinical trials as a formal Priority Issue. Reimbursement-related activities and legislation are tracked and evaluated for potential nursing input. The opportunity and need for nursing involvement in addressing this issue is growing, but first the commitment must be made to become involved. The issue of reimbursement for the patient care costs of investigational therapy is not easy to resolve. It involves assurances that ethically and scientifically important questions are being addressed, that patients are receiving quality care, and that outcome information is being obtained from trials so that the level of benefit can be determined. These are mutual goals of health professionals and insurers alike. Only by communicating and working together can these problems be resolved and progress continue to be made in the treatment of cancer.
REFERENCES 1. Smith MA, Parkinson DR, Cheson BD, et al: Retinoids in cancer therapy. J Clin Oncol 10839-864, 1992 2. Data from the Cancer Therapy Evaluation Program, Division of Cancer Treatment, National Cancer Institute, Bethesda, MD 3. Friedman MA, McCabe MS: Assigning care costs associated with therapeutic oncology research: A modest proposal. J Nat1 Cancer Inst 84:760-763, 1992 4. Drug Master File 2803, Division of Cancer Treatment, National Cancer Institute, Bethesda, MD 5. Investigators Handbook. Bethesda, MD, Cancer Therapy Evaluation Program, National Cancer Institute, 1986 6. Health Care Financing Administration: Certain drugs distributed by the National Cancer Institute, in Medicare Coverage Issues Manual, reprinted in Medicare and Medicaid Guide (CCH) 27:201, 1989 7. Hilts PJ: Demands to fix U.S. health care reach a crescendo. NY Times, May 19, 1989
8. McCabe MS, Friedman MA: Impact of third party reimbursement on cancer clinical investigation: A consensus statement coordinated by the National Cancer Institute. J Nat1 Cancer Inst 81:1585-1586, 1989 9. Vogelzang NJ, Richards JM: Third-party reimbursement issues during a phase I trial of interleukin-2. J Nat1 Cancer Inst 81:544-545, 1989 10. Treatment Referral Center Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD 11. Resources for Clinical Investigation-Report of a Study. Washington, DC, Institute of Medicine, 1988 12. Position statement: American Society for Clinical Oncology, Government Relations Office, Washington, DC, 1990 13. National Committee to Review Current Procedures for Approval of New Drugs for Cancer and AIDS: Final Report, August 15, 1990 14. Association of Community Cancer Centers: Cancer Treatments Your Insurance Should Cover. Rockville, MD, ACCC, March 1991
2 (IL-2). All of these agents are currently benefiting cancer patients in a variety of circumstances and clinical settings. In addition, many promising biotherapies are in development. Examples include X-6, which affects platelets and exerts antitumor effects, and all-trans retinoic acid, which is active in forms of leukemia and appears useful in other malignancies. ’ Investigational agents such as these represent important, if not the best, clinical options for many patients, given the severe, life-threatening nature of malignant disease and the less than successful state of many current treatments. Investigational therapy, therefore, is important to oncology nurses, especially those providing care in cancer centers, outpatient departments, and oncology clinical research departments. Nurses should be informed about the drug development process, the therapeutic potential of investigational therapy, and the financial impediments patients face in obtaining access to these promising new treatments.
Cancer is a set of diseases that affects one in four Americans and which is too often fatal. For many types of cancer, there is no fully satisfactory treatment. Therefore, investigational therapy provided in a formal, peerreviewed clinical trial can present the best available treatment for patients with life-threatening malignancies. At a time when clinical cancer research is at a particularly promising point and multiple new biologics are on the horizon, the traditional partnership of the federal government, the pharmaceutical industry, private institutions, and insurers is threatened. The issue of increasing concern is the impact of changes in third-party reimbursement policy on cancer clinical investigation. Although its incidence, magnitude, and geographic distribution have not been specifically defined, this issue is generating serious concern and intense national debate. The growing denials of patient-care costs related to clinical trials serve to threaten patient access to potentially efficacious therapy. The implications of this reimbursement issue are tremendous for both clinical investigation and patient care. The refusal to pay for patient-care costs related to investigational therapy may adversely influence clinical trial design and will certainly affect the speed with which new therapies are developed. When insurers refuse to cover these dinical costs, only affluent individuals have access to the most promising treatments. Nursing actions can positively affect the reimbursement debate. Nurses must develop cost-efficient plans of care and the means to evaluate their effectiveness. Educating patients about and assisting them with reimbursement issues maximizes their prospects for receiving appropriate investigational therapy. Good communication and ongoing discussions with local insurers are essential. Nurses should be aware of their potential contribution in this important issue and should participate in this debate both locally, in the clinical setting, and nationally, through professional organizations. This is a US government work Them are no restrictions on its use.
CLINICAL TRIALS
The National Cancer Institute (NCI) clinical trials program is important because of the commitment of investigators on a national level and the impact it has on the development of more effective cancer treatments. The total number of participants in NC1 trials is small-less than 30,000 patients per year-compared with the 1,250,000 Americans diagnosed with cancer each year.2 However, thousands of nurses and physicians in more than 1,300 hospitals and institutions across the country conduct NC1 trials, and the benefit to the patients involved can be tre-
ECENT ADVANCES in biotechnology have led to the development of important new biologic cancer agents. Beginning with interferon alfa, these drugs have been identified, formulated, tested, and marketed at an ever-accelerating pace. The future holds possibilities for increasingly successful cancer therapies, as evidenced by recently approved biologics such as erythropoietin (EPG), granulocyte and granulocyte-macrophage colony-stimulating factors (G-CSF and GM-CSF, respectively), and, as of May 5, 1992, interleukin-
R
~e~ham
in
Onmlogy Nursing.
Vol8,
No 4. Suppl
1 (November),
PROGRAM
From the Division of Cancer Treatment, National Cancer Institute, Bethesda, MD. Address reprint requests to Mary S. McCabe, RN, BA, BS, Clinical Trials Specialist, National Cancer Institute, Division of Cancer Treatment, Bethesda, MD 20892. This is a US government work. There are no restrictions on its use. 0749-2081/92/0804-IOO4%05.00/0 1992:
pp 3-7
3
MARY
mendous. Any disruption of this research program can potentially impede improvements in cancer treatment and thereby affect all future cancer patients. INVESTIGATIONAL
THERAPY
It is important that scientifically valid clinical research with investigational agents be distinguished from unscientific, unscrupulous use of cancer treatments that may be wrongly labeled “cancer research.” The term “investigational therapy” is intended to include treatment administered as part of a formal, peer-reviewed clinical trial. New drugs proceed through an established sequence of clinical trials in which knowledge builds from one stage to another, and continued evaluation depends on meaningful positive results. The clinical trials process comprises three phases, each offering therapeutic potential to participating patients: (1) phase I-identification of the maximal safe dose; (2) phase II-evaluation of efficacy; and (3) phase III-randomized comparison against the standard therapy. This process is designed to assure that clinical cancer investigation proceeds in an orderly sequence. However, because the clinical trials process yields cumulative information, important data about the benefits of a new therapy may become available before completion of the process or Food and Drug Administration (FDA) approval of the therapy. Because of this continuum of knowledge, the distinction between investigational or experimental and “standard” therapy is often blurred.3 Thus, this distinction, although often defined and codified, may not be the best way to determine insurance coverage for life-threatening disease. GROUP C ANTICANCER
types. There is an appreciable time period before these drugs, sponsored by the pharmaceutical industry, receive NDA [New Drug Application] approval. In order to allow patients with these diseases to have the benefit of this drug therapy, a Group C drug classification has been developed. The purpose of this distribution is to allow accessto beneficial therapy and to acquire information on safety in the context in which the drug is likely to be used in clinical practice after marketing.“”
For a drug to qualify for Group C status, a review of abstracts, papers, and reports must show that it has reproducible efficacy against a specific tumor type, that it alters the pattern of care of the disease, and that it may be administered safely by properly trained physicians without the need for special supportive care facilities.’ The one exception to this last requirement is IL-2/lymphokine-activated killer cells (LAK) which, because of the additional laboratory and safety monitoring it entails, was placed in a Modified Group C category. Drugs included in this modified category meet all other Group C criteria, including efficacy. Early in the history of the Group C program, drugs remained in this category for extended periods, in part because of the lack of a commercial sponsor. But more recently, the Group C category has been operating as a brief bridge between the identification of activity and FDA approval. Table 1 lists current Group C drugs and their indications. Recently approved drugs formerly in Group C include ifosfamide/mesna, deoxycoformycin (pentostatin), levamisole. fludarabine phosphate, and IL-2. Despite the formal review and strong demonstration of benefit accompanying drugs placed in this Group C category, there are no assurances that patient care costs associated with such ther-
DRUGS
Recognizing that important knowledge about the activity and benefit of a drug becomes evident even while a drug continues through the evaluation process, the NC1 and the FDA in 1976 established a category of drugs called Group C. The purpose of this category is to make active agents more rapidly and widely available before FDA approval. In the words of the NCI: “During the evaluation of investigational drugs by the Division of Cancer Treatment, NCI, certain drugs are found to have a role in the treatment of specific tumor
S. MCCABE
Table
1.
Grow
C Druas
and
Their
Group C Drug
Indications Indication
Asparaginase-erwmia
Acute lymphocytlc patients sensitive
Amsacrine
Refractory adult myelogenous
Azactldine
Refractory
Taxol Teniposide
leukemia Refractory ovarian carcinoma Relpased or refractory acute lymphoblastic leukemia
2Xhlorodeoxyadenosine
Active
asparaginase
(Escherichia
acute
hairy
leukemia to acute leukemia myelogenous
cell leukemia
co/i)
PERSPECTIVES
OF THE HOSPITAL
ONCOLOGY
apy will be reimbursed. The Health Care Financing Administration (HCFA), which administers the federal Medicare program, published formal guidelines in 1982, which recently have been restated, that “a Group C drug and the related hospital stay are covered if all other applicable coverage requirements are satisfied.“6 However, this statement of coverage is not necessarily transferable to other insurers, and many choose not to cover this important group of agents. Even the threat of non-reimbursement is a strong disincentive to the use of these drugs, and such discouragement is indefensible. Only the treatment-related costs are, in fact, at issue, since the drugs themselves are distributed free of charge by the NCI, and there is no expectation that this policy will change. EVOLVING
TRENDS
AND ISSUES
At this scientifically exciting juncture, debate is increasing over the cost of the United States health care system, estimated at $700 billion per annum.7 Concern is mounting as to how the current system will address the rapid advance of expensive technologies, the growing number of elderly, and the increasing number of uninsured and underinsured citizens, while at the same time stabilizing expenditures. The American health care system is in crisis because of escalating costs. Daily, the popular press and professional journals report the mounting problems. In the current health care climate, economic considerations are an integral part of any planning of research and care. They cross all subspecialty borders, but are particularly important in oncology. At no other time have financial issues and decisions so influenced the responsibilities of oncology nurses. REIMBURSEMENT
5
NURSE
OF INVESTIGATIONAL THERAPY
Third-party reimbursement for patients receiving investigational agents in clinical trials is a key element of the cancer economics discussion because such research is an essential and wellestablished health care priority. Historically, there has been a partnership of payers including the federal government, private institutions such as universities, the pharmaceutical industry, and third-party insurers.8 This coalition is changing, threatening reimbursement for both the research and patient care components of clinical trials.
There is a growing perception that lack of reimbursement is having a serious impact on current cancer patients and has implications for drug development, and thus for patients, now and in the future. Volgelzang and Richards at the University of Chicago have documented delayed completion and early closure of studies because of reimbursement problems with biologics such as IL-2.9 Such difficulties slow the development of new therapies. Reimbursement restrictions also could decrease the cost effectiveness of drug development, if the design of studies were determined by insurance coverage rather than by the pursuit of doses, schedules, and delivery systems designed to produce the best possible clinical outcome. Moreover, reimbursement restrictions could lead to reduced access to promising new therapies and to an elitist system of health care, with only patients who could afford the full cost entering clinical trials. Including only affluent patients may also skew clinical results, since socioeconomic factors may affect patients’ general health and response to therapy. Presently, a serious reimbursement problem is occurring with taxol, which, although not a biologic, is one of the most important cancer agents currently in development. Nationwide, patients are having difficulty securing insurance coverage for treatment through the NC1 Treatment Referral Center, despite strong data supporting its efficacy in refractory ovarian carcinoma.” Further problems in the reimbursement of clinical trials stem from the decentralized nature of the health insurance industry, which results in inconsistent coverage policies. Because insurers are regulated at the state rather than the national level, reimbursement policies can differ from state to state. A patient covered for a therapy in one state may lose this coverage on moving to another state. Even Medicare, although a national program, suffers from inconsistent reimbursement policies, because unless HCFA has developed a national policy for a therapy, local intermediaries are allowed to determine coverage. CONSENSUS FOR REIMBURSEMENT OF INVESTIGATIONAL THERAPY
Growing concern over the lack of reimbursement for investigational therapy has prompted a number of national organizations and coalitions
6
MARY
to study this issue and publicly present statements and recommendations to address it. For example, in 1988, the Institute of Medicine issued a report declaring that: “Third-party payers (government and non-govemment) should pay the necessary and appropriate patient care costs for beneficiaries enrolled in approved clinical investigational protocols. This requires a clarification in current Medicare regulations involving definitions of medically necessarycare. State regulatory agencies should require conforming changes by all other third party payer policies.” “There are diseasesfor which appropriate and required care involves investigational protocols. Such diseases include certain types of cancer, genetic diseases, and possibly other severe, life threatening diseases.In these cases, third party payers (government and nongovernment) should pay the standard patient care costs while costs related to the investigational conclusions should be borne by the sponsoring agency.“”
The American Society of Clinical Oncology (ASCO), which represents oncologists, released a similar position statement in 1990: “Both Medicare and private insurers should accept the views of experts to the effect that treatment under a clinical protocol represents the best available therapy for critically ill cancer patients and that patient care costs, e.g., the cost of hospitalization-should be borne by insurers.“‘*
A group of health care professionals, attorneys, and administrators convened by the NC1 developed a consensus statement advocating that: ‘6 . third-party coverage be allowed for patient-care costs of all nationally approved (NC1 or FDA) cancer treatment research protocols and that third party coverage should also be allowed for all cancer treatment research protocols not subject to national approval, provided these protocols have been approved by established peer review mechanisms .‘r8
An important group convened by then Vice President Bush, the National Committee to Review Current Procedures for Approval of New Drugs for Cancer and AIDS (the Lasagna Committee), also supported this position, recommending that: “The cost of investigational drugs, and marketed drugs prescribed for unlabeled indications, as well as all ancillary medical care, should be covered by Medicare, Medicaid, and private insurance, if the use has been approved by expert government agencies, in authoritative medical compendia, or by a committee established by the Secretary of Health and Human Services to deal with this matter.“13
S. MCCABE
The Association of Community Cancer Centers (ACCC), another important oncology organization, also has supported reimbursement for clinical trials. I4 However, despite these strong, consistent recommendations of national health care leaders and formal scholarly groups, reimbursement problems continue to go unresolved. Too often, the courts determine reimbursement for investigational therapy. All groups of health care providers, patients, and insurers seeking to resolve this serious issue acknowledge that such a pattern is inappropriate and adversarial. RECOMMENDATIONS
FOR NURSING ACTION
To be informed is one of the principal responsibilities nurses have when interacting with patients seeking information about clinical trials or when caring for those receiving investigational therapy. The economic issues related to such treatment are too integral to oncology nurses’ professional roles to be ignored. First, it is important to know the process of working with insurers. For nurses in the hospital setting, this may mean assisting and educating patients to negotiate with representatives of their insurance plans. For nurses in the outpatient setting, this may involve communicating directly with insurance companies about a clinical trial. It often is helpful to (1) assemble packets of appropriate data for review by the insurance company’s medical director; (2) update local plans about important studies at the institution; and (3) provide data demonstrating the standards of excellence of both clinical investigators and ongoing clinical trials. Many cancer centers have developed good working relationships with insurers in their area. Insurers see them as “centers of excellence” because of the quality of patient care and their use of appropriate, scientifically sound therapies that offer patients potential for real benefit. In addition to involving themselves in these institutional reimbursement issues, nurses can pursue individual activities that address the need to be economically efficient. Most important is the development of a care plan that takes into consideration (1) potential self-care options for patients, such as self-injection or the use of ambulatory infusion pumps; (2) the choice of an economical, but safe and comfortable, treatment
PERSPECTIVES
OF THE
HOSPITAL
ONCOLOGY
NURSE
7
setting for the patient; and (3) discharge planning strategies to minimize hospital stays and provide for continuity of care at home. Patients themselves have a need and responsibility to be informed of the financial issues related to the investigational therapy being considered. They generally assume that their insurance covers such therapy, and frequently are frightened and angry to discover that this is not the case. Thus, nurses should view patient education on financial issues as part of the informed consent process. Pharmaceutical companies provide reimbursement information services and indigent patient programs. A listing appears in the article by Xistris in this supplement. Instituting change in state and national policies on reimbursement for investigational therapy can and should involve nurses. Oncology nurses should join community cancer groups working to educate and influence insurers and officials. Membership in national organizations, such as the American Cancer Society, that promote the
development of new cancer therapies, can increase support for reimbursement. The Board of Directors of the Oncology Nursing Society has identified reimbursement for clinical trials as a formal Priority Issue. Reimbursement-related activities and legislation are tracked and evaluated for potential nursing input. The opportunity and need for nursing involvement in addressing this issue is growing, but first the commitment must be made to become involved. The issue of reimbursement for the patient care costs of investigational therapy is not easy to resolve. It involves assurances that ethically and scientifically important questions are being addressed, that patients are receiving quality care, and that outcome information is being obtained from trials so that the level of benefit can be determined. These are mutual goals of health professionals and insurers alike. Only by communicating and working together can these problems be resolved and progress continue to be made in the treatment of cancer.
REFERENCES 1. Smith MA, Parkinson DR, Cheson BD, et al: Retinoids in cancer therapy. J Clin Oncol 10839-864, 1992 2. Data from the Cancer Therapy Evaluation Program, Division of Cancer Treatment, National Cancer Institute, Bethesda, MD 3. Friedman MA, McCabe MS: Assigning care costs associated with therapeutic oncology research: A modest proposal. J Nat1 Cancer Inst 84:760-763, 1992 4. Drug Master File 2803, Division of Cancer Treatment, National Cancer Institute, Bethesda, MD 5. Investigators Handbook. Bethesda, MD, Cancer Therapy Evaluation Program, National Cancer Institute, 1986 6. Health Care Financing Administration: Certain drugs distributed by the National Cancer Institute, in Medicare Coverage Issues Manual, reprinted in Medicare and Medicaid Guide (CCH) 27:201, 1989 7. Hilts PJ: Demands to fix U.S. health care reach a crescendo. NY Times, May 19, 1989
8. McCabe MS, Friedman MA: Impact of third party reimbursement on cancer clinical investigation: A consensus statement coordinated by the National Cancer Institute. J Nat1 Cancer Inst 81:1585-1586, 1989 9. Vogelzang NJ, Richards JM: Third-party reimbursement issues during a phase I trial of interleukin-2. J Nat1 Cancer Inst 81:544-545, 1989 10. Treatment Referral Center Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD 11. Resources for Clinical Investigation-Report of a Study. Washington, DC, Institute of Medicine, 1988 12. Position statement: American Society for Clinical Oncology, Government Relations Office, Washington, DC, 1990 13. National Committee to Review Current Procedures for Approval of New Drugs for Cancer and AIDS: Final Report, August 15, 1990 14. Association of Community Cancer Centers: Cancer Treatments Your Insurance Should Cover. Rockville, MD, ACCC, March 1991
