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Implications of the Introduction of Recombinant Factor VIII Janice R. Dillon

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HERE ARE three broad legal issues to be considered with respect to the introduction of recombinant factor VIII (rFVIII). The first concerns the scope of legal liability that is attracted through continued use of conventional blood products in the face of the new recombinant product and asks the ultimate question of whether legal considerations will render the use of rFVIII mandatory. The second issue concerns the requirement for information diselosure to patients about rFVIII, assuming its availability. The third issue addresses the principle of equal access and distribution of resources within the health care system and assesses the position of patient demands, the standards of provision of service as set out in legislated health care schemes, and the right to equality as constitutionally protected. There are certain limitations and assumptions that are implicit in this article. The first is a bias in favor of the Canadian example. AIso, the focus is on the_l'lrovidęr!, of the nrodllct._ narticllla!lv IWVernment, blood distribution agencies, and physicians , and not on the manllfacturer or the patient as the ultimate consumer. Whereas there may be medical risks inherent with use of traditional blood products related to the exposure to alloantigens and transfusion-associated viral diseases and with use of rFVIII presumably associated with the frequency of inhibitor formation, l relative risk factors do not seem to be a significant differential between the two product types. However, it is appreciated that the risks are different. The benefit from the use of the recombinant product pertains particularly to expectations of increased supply because medical benefit has been characterized as marginal. 2 At the same time, it is anticipated that the cost of the recombinant product might be several times the price of plasma-derived factor VIII, at least initially. An interesting comparison for the industry was the introduction from 1984 to 1986 of low-osmolar contrast media in radiology. The new low-osmolar (nonionic) intravascular contrast media provided a safer and more comfortable angiography and myelography than conventional contrast media. These advantages made the new media the preferred Transfusion Medicine Reviews, Vol VI, No 4 (October), 1992: pp 265-270

choice among radiologists even though conventional media were not inherent1y unsafe or unfit for use. Despite the option of selective use ofthe new media in patients known to be at increased risk,3-7 there was a tendency to tum to low-osmolar media for a11 investigations, particularly when it first came onto the market. This was so even though the cost was initially estimated to be 10 times the amount for conventional media. Some provincial governments funded the additional costs for the newer prodllCt and others did not. Funding then had to come from changed priorities within established hospital budgets. Radiologists had requested sole use of the new media presumably to relieve "the individual radiologist of much of the medicolegal responsibility if a serious adverse reaction to a conventional, high-osmolar medium occurS.,,3 There was also the perception that patients would demand use of the newagentonce the alternatives between the agents were explained during the process of obtaining inforIlled consent. Legal response to the demand for 100% use was tempered by the realization that physician response was provoked less by medical and patient concerns based on relative risk, than by concerns for legal responsibility if the newer media was not used. 8 Legal duties of care were satisfied by selective use of the new media in patients known to be at increased risk once it was elear that economic considerations preeluded 100% use of the more expensive media. 9 In the introduction of rFVIII, the risk-benefit ratio does not suggest significant risk reduction and the social cost is high. LEGAL L1ABILlTY FOR USE OR NONUSE OF RECOMBINANT FACTOR VIII

The basic question is whether the new product should be used to avoid potentialliability for contaminated blood. The standard of resource use is Address reprint requests to Janice R. Dillon, UB, 2080 First City Bldg, 777 Hornby St, Vancouver, British Columbia, V6Z 1S4 Canada. Copyright © 1992 by W.B. Saunders Company 0887-796319210604-0007$3.0010

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set by those who fumish the product and by physicians. For present purposes, it is assumed that rFVIII will be considered within the scope of blood products as opposed to a pharmacological product simpliciter and will therefore come within the mandate ofthe blood distribution agencies, inc1uding the Canadian Blood Agency. As such, the mandate of the Canadian distribution agency inc1udes the establishment of policies, guidelines, and coordinated planning for the use of blood products and their substitutes. The Canadian Blood Agency could establish c1inical protocol for the use of rFVIII. What legal limitations and standards would affect the development of this protocol? Limits and Standards Applicable to Blood Distribution Agencies

Govemment-funded and -controlled blood distribution agencies are exempt from the traditional tort law (negligence) duty of care if an explicit statutory exemption exists or if a decision is a resuIt of a policy decision. 10 Thus, a c1inical protocol for use of rFVIII developed by the Canadian Blood Agency would not be subject to negligence liability in Canadian law if there was a specific legislative exemption or if the usage guidelines could be characterized as policy. There is not a specific exemption in Canadian law at present. The 1990 report entitled Liability and Compensation in Health Care (called the Prichard report) recommended that no grant of immunity from suit be granted to producers, suppliers, distributors or other participants in the Canadian blood supply system. 11 Instead, blood product injuries would be treated as part of the general category of health care injuries. 11 "True policy decisions are exempt from [negligence] c1aims so tbat govemments [and their agencies] are not restricted in making decisions based upon social, political or economic factors. However, the implementation of [these] decisions may well be subject to c1aims in tort. ,,10 The difference between a policy and operational decision as described by a judge of the Australian High Court and considered a helpful guideline by the Supreme Court of Canada is; The distinction between policy and operational factors is not easy to formulate, but the dividing line between them will be observed if we recognize that a public authońty is under no duty of care in re1ation to decisions which involve or are dictated by financial, econornic, social OT

political factors or constraints. Thus budgetary allocations and the constraints which they entail in terms of allocation of resources cannot be made the subject of a duty of care But it may be otherwise when the courts are called upon t~ apply a standard of care to action or inaction that is merely the product of adrninistrative direction, expert or professional opinion, technical standards or general standards of reasonableness. *

A decision reached as to allocation of resources, in this case the funding of rFVIII, is likely to be considered a policy decision that, taken alone, cannot be subject to a c1aim in negligence. So, a decision not to fund or selectively to fund rFVIII because of cost and competing budgetary priorities is an unassailable policy decision. However, once a policy decision has been made to use the recombinant produet, the courts may reviewuse guidelines to ensure that they are reasonable and have been reasonably carried out in light of all of the circumstances to determine if the agency has met the requisite standard of care. 10 The govemment agency would be entitled to demonstrate that, balanced against the nature and quality of the risk involved, the use guidelines were reasonable in light of budgetary limits, personnel, supply, and other factors. lO However, if the evidence showed that the guidelines lagged behind other similar groups who had adopted more rigorous standards, then it might not be considered reasonably prudent in law to maintain the standard even though it may be widely adopted in the industry. 13 It would be open to show that the custom and practice in the industry itself was unreasonable. On this basis, a Colorado appeals court ordered a new trial in a case involving blood allegedly contaminated with the acquired immunodeficiency syndrome (AIDS) virus to determine whether a whole blood center was negligent in 1983 for not recommending donor screening on the basis of sexual preference or surrogate testing, that is, testing for factors believed to be associated with AIDS, even though the American Association of Blood Banks and the American Red Cross did not recommend this screening in their January 1983 joint statement, and the American Food and Drug Administration did not recommend surrogate testing in its first recommendations in March 1983Y

* Underlining by Cory, J of the Supreme Court of Canada in Just v British Columbia. 10

LEGAL IMPLICATIONS OF RECOM81NANT FACTOR VIII

Within the Canadian framework, the point is that decisions about the use of rFVIII based on funding are not likely to render blood distribution agencies liable in negligence. But manifestations of the implementation of that policy decision are operative in nature and would be subject to judicial review to detennine whether the appropriate standard of care was followed. This standard in negligence is best described within the context of the duty of care owed by physicians to their patients with one proviso. The duty of care for blood centers might not be set as the professional standard, which is a preferred position based on the attributes of a profession and that is met by custom in the industry, because these centers or agencies are not professional bodies. Therefore, the more general reasonableness approach may prevail. Standards Applicable to Physicians The standard of care required in law combines a complex of factors to conclude that physicians must deliver a "roughly equal minimum quality of care,,14 to all their patients. Factors to consider in assessing the likely legal standard for the selective use of rFVIII include custom in the profession, risk, and costs. Whereas custom of the profession is the touchstone for the medical malpractice standard, it is not conclusive of negligence but a powerful indicator. IS -18 Customary practice is established by a contemporary survey of professional members who will, in tum, most likely base their practice on clinical protocols issued by blood agencies or professional groups. In the introduction of rFVIII, the task is to define who will benefit medically from the new product. By developing an industrywide consensus on this point, the accepted or customary practice defence should act as a powerful shield for physicians. But, it is not an impregnable shield. If it is apparent "that the entire profession has made an unreasonable assessment of the socially optimal use of a medical procedure, the law is capable of a de novo evaluation of the relative costs and benefits ... " 19 Courts have long recognized the rule that the degree of care demanded is proportionate to the degree of risk. This is especially so when using an older treatment in the face of a newer one that exposes certain risks, especially if the newer treat-

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ment involves precautionary or safety measures. 16,19,20 Thus a higher degree of care may be required when choosing between use of plasma-derived factor VIII products and rFVIII because of the known risks associated with blood products. However, whenever risk is taken in a medical situation, there will be someone who is affected adversely. This does not make a physician liable unless exposure to the risk was not reasonable. 21 The ability to foresee an unusual and unlikely risk, for example, transmission of the AIDS virus through contaminated blood, does not establish a duty to prevent it; rather, the duty is to provide reasonable care, treatment, and facilities in the circumstances. 21 The requirement for reasonable care in the circumstances may inevitably lead to a judicially applied costs-benefit analysis, something that the courts are reluctant to undertake but will do, especially if the custom-based rule seems not to have reasonably included cost considerations. It becomes a matter of balancing the risk against measures necessary to eliminate it.2 2-24 On this basis, it may be unreasonable to suggest use of the new recombinant product in all cases, in light of the degree of risk as compared with expenditure. This is not to say that there is a privileged defence for poor or inadequate funding. However, it is a legitimate consideration in establishing the legal standard of care. Unless there are plenty of financial resources available, one cannot be expected to take precautions against every foreseeable risk. 23 ,2S,26 Naturally, if the cost involved is minimaI, liability is more likely to follow for failure to take extra precaution. 18,19,24 In the case of rFVIII, costs to the individual patient may seem minimal when one considers that the patient may be able to go out and buy the product as a pharmaceutical on a contract theory of resource coverage where the patient chooses what to pay.14 However, if rFVIII falls for distribution within the nationally based Canadian blood or blood substitute distribution system, the option of individual purchase is not open. It is also not open if rFVIII is provided within the hospital setting under comprehensive public health insurance. The overall cost of use in all cases is projected to be higher than contemporary blood product use. Consideration should be given to criteria for selective use of rFVIII and to the impact of the diversion of funds from other useful expenditures if additional overall. funding is not forthcoming from governments.

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What level of care does the physician legally owe his patient? There is no legal duty to provide the optimum or very highest level of care. Whereas it is obviously often desirable to provide the very latest and the very best, it may not be possible. The law takes this into account. The standard of resource availability is set by the payers for the resource, govemments, insurance agencies, and the individual purchaser of health services in private health care systems. Physicians should protest against inappropriate cost constraints and make efforts to ensure their patients receive necessary care. 14 ,27 But, the level of care provided is within a resource availability standard set by others. As economic advocates for their patients, the physicians must make a systematic effort to help their patients obtain resources, but this does not extend to bypassing legitimate restraints, commandeering property that does not belong to them, or "gaming the system" with duplicity or coercion to gain resources not intended for patients. 14 The legal standard of care does not demand physicians to bypass legitimate program limitations. THE DUTY TO PROVIDE INFORMATION

With the availability of rFVIII comes the legal duty to inform patients about this altemative to traditional blood products. The legal doctrine of informed consent requires disclosure to the patient of the nature of the proposed treatment, its risks, and available altematives. 28 ,29 Risks to be disclosed are those that are material, special, or unusual. Even mere possibilities must be disclosed if their occurrence would result in serious consequences such as death. The risks and benefits of rFVIII must be explained to patients as an altemative to traditional therapy once it becomes available. If the clinical protocols established for use include medical criteria for selective use of the newer product, this should be explained. Disclosure within the informed consent process should not be viewed as a hindrance to the continued use of plasma-derived factor VIII because it is still an acceptably safe product. Experience with the low-osmolar contrast media has shown that patients are generally accepting of criteria for use when best use of the expensive product is explained. It can be anticipated that with theknowledgeable population of hemophili-

acs, full disclosure of comparative risks and benefits will be the order of the day. EQUALlTY IN ACCESS AND DISTRIBUTION OF HEALTH RESOURCES

In privately funded health systems, an individual basically contracts for the level of care he wants by purchasing a medical insurance plan or paying for health care directly. There is no operative concept of a right to treatment so nobody could be compelled to make rFVIII available to any person. Canadians have astatutory right to "reasonable access" to "medically required" health services. 30,31 Whereas this right of access goes beyond minimaUy decent health care, it does not encompass a right to optimal treatment without regard to cost. AIso, it does not cover the cost of drugs, except those administered in the hospital setting. The blood distribution network could be considered a "health service" under the Canadian legislation. If rFVIII falls within the network, it is unlikely that govemments could be compelled to supply the newer recombinant product to everyone and may be obliged only to supply selectively if it was considered medically necessary. Patient attempts to force govemments to supply services under public medical insurance schemes have failed. In a 1980 case heard by the British Court of Appeal, an attempt was made by those who had waited 4 years to force the minister to supply orthopedic services. Lord Denning said that the duty is to provide reasonable requirements within available resources. It could not be supposed that the govemment had a duty to provide all the latest equipment, or all the kidney machines asked for, or all the new developments in every case in which people would benefit from them. 32 The United States Supreme Court came to a similar conclusion in 1985 when it upheld Tennessee's decision to reduce the number of covered hospital days under Medicaid. The Court said that the benefit is not adequate health care but a package of health care services that states could alter as they saw fit, thereby rejecting any notion of a minimum benefits requirement. 33 The Canadian threshold requirement is that the treatment must be "medically required." Will it be possible to say that a certain patient requires rFVIII to the extent that harm will follow if it is not

LEGAL IMPLlCATIONS OF RECOMBINANT FACTOR VIII

provided? If so, then there could be a duty to provide the new product to that individual provided distribution of rFVIII is considered a "health service. " There is the problematical consideration that provision of rFVIII may not be a provision of a medical or health service but a prescription for a drug within the meaning of the Canadian Food and Drug Act. 34 There is no coverage for drugs in Canadian public health insurance, except if administered in the hospital. 30 If considered a drug, patients could demand access to rFVIII outside the institutional setting, ineluding blood distribution agencies, on the contract theory of access where they get what they pay for. The net result would be that patients could gain access in different ways and, depending on the policy developed by distribution agencies, access may not be equal as a resuIt. Some patients would pay for the drugs and others would not. This becomes even more difficuIt when it is realized that hemophiliacs now benefit from govemment-funded blood products that are highly manufactured and are also considered drugs under the Food and Drug Act,35 whereas other patients with nonhemophiliac-related AIDS or diabetes, for examples, pay for their drug products personally except when administered in the hospital. Section 15 of the Canadian Charter of Rights and Freedoms states that Canadians have a right to equal benefit under the law without discrimination based on physical disability. 36 The goal of equality is stated to be "that a law expressed to bind all should not because of irrelevant personal differences have a more burdensome or less beneficial impact on one than another. ,,37 Equality is violated if the law works to an individual's disadvantage by denying an opportunity to that individual that is available to others. 38 A law is discriminatory if it distinguishes between individuals because of personal characteristics of the individual or group to which they belong and has the effect of imposing burdens, obligations, or disadvantages on such individual or group that are not imposed on others or which withholds or limits access to opportunities or benefits available to other members of society. 37 It is arguable that govemment policies that favor hemophiliacs in the payment for drugs over other disabled groups is discriminatory. Assuming that such an argument could be made,

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it seems unlikely that it would be made. It would pit one physically disabled group against another. There are substantial legal arguments to the contrary, some of which are beyond the scope of this artiele. But it could be argued that disabled groups other than hemophiliacs are treated no differently under the law than other Canadians who pay for medications so that the benefit they would be elaiming is not one that is generally available to others. Also, Section l of the Canadian Charter of Rights and Freedoms only guarantees rights "subject to such reasonable limits prescribed by law as can be demonstrably justified in a free and democratic society." 36 It is possible through this provision to override a Charter breach if the law is pursuing a pressing and substantial objective; the means selected is not unfair, arbitrary, or irrational; the right is impaired as little as possible; and there is proportionality between the effect on rights and the importance of the govemment objective. 39 Such an argument could be made to justify payment for blood distribution because of govemment funding of production of blood products, voluntary public participation in the Canadian blood collection process, and the not-for-profit basis fractionation of blood products in Canada. 35 How elosely distribution of rFVIII reflects these considerations remains to be seen: but introduction of the produet should not jeopardize the present funding arrangements by inviting a Charter challenge based on inequality of funding between disabled groups. CONCLUSION

In conclusion, the first matter to be resolved in the introduction of rFVIII is whether distribution of the product should falI totally within the blood distribution system or whether it should be available independent of that network, perhaps as a prescription drug. Second, physicians should establish elinical protocols for use that would, hopefully, indicate a basis for selective use of the newer, more expensive product. Legal considerations do not require use of rFVIII in preference to traditional blood products unless there is substantial reduction of risk. Benefit-to-risk assessment within the distribution agencies and the profession is required not only to establish customary use in the profession, but to satisfy legal concems that optimum social use has been made of rFVIII, assuming economic constraints.

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17. Brown v Rolls Royce, WLR 210, 1960, pp 215-216 18. Helling v Carey, 519 P 2d 981, 1974 19. Hall M: The malpractice standard under health care cost containment. Law Med Health Care 17:349, 1989 20. Holt v Nesbitt, I DLR 671, 1953 21. Worth v Royal Jubilee Hospital, 4 W Med Q 59, 1980 22. Latimer v AEC Ltd, 2 QB 701, 1952, P 711 23. Clerk and Lindsell on Torts (ed 14): 1975, para 901 24. Linden: Canadian Tort Law. Tor, 1977, p 90 25. British Railways Board v Herrington, AC 877, 1972 26. Southem Portland Cement Ltd v Cooper, AC 623, 1974 27. Wickline v State of Califomia, 228 Cal Rptr 661, 1986, P 671 28. Hopp v Lepp, 2 SCR 192, 112 DLR (3d) 67, 1980 29. Reibl v Hughes, 2 SCR 880, 114 DLR (3d) l, 1980 30. Canada Health Act, SC 1984, c 6, s 12 31. Medical Service Act, RSBC 1979, c 255, si 32. R v Secretary of State for Social Services ex Parte Hincks, unreported British Court of Appeal, March 18, 1980 33. Alexander v Choate, SCt 712, 1985, pp 713, 722 34. Food and Drug Act, RSC 1985, C F-27, as amended, s 192-196 35. Rodgers S: The Canadian blood delivery system: Liability for blood related injuries. Liability and Compensation in Health Care (vol 3) p 19 36. Canadian Charter of Rights and Freedoms, Pt I of the Constitution Act, 1982, Sch B, Canada Act, 1982 (UK), 1982, clI, s 15 37. Andrews v Law Society of British Columbia, l SCR 143, 1989, P 165 38. R v Turpin, l SCR 1296, 1989, p 1329 39. R v Oakes, l SCR 103, 1986