A guide for approaching controversial, high tech procedures. (Managing Organ Transplant Issues).

Key Concepts: Organ Transplants/High Technology/Medical Policy/External Review/Developing a Strategic Plan

With the increase in the requests for high intensity medical procedures such as organ transplantation, the physician executive often is placed in the middle between competing interests. Whether he or she represents the provider or payer side, there is frequently great pressure to make decisions involving complex medical situations in short timeframes. It is necessary to have in place a fair, consistent approach to handling such issues in order to withstand both medical and ethical scrutiny. A process is detailed that will lead the organization to developing such an approach. Although presented in the context of transplant issues, this process can be applied to any similar high technology procedure.

AMONG THE MANY MEDICAL MATTERS THAT ARE becoming increasingly more complicated for health plans, physicians, hospitals, and patients, human organ and tissue transplantation is a prime example. The reasons are evident. As high intensity procedures, transplants demand huge allocations of resources, both financial and technological. Given that many transplants are matters of life and death, the moral, ethical, and emotional overlays are significant. The science of transplantation is frequently shifting, often dramatically.

For some procedures and indications, the medical evidence and protocols are clear and well established. For example, kidney and cornea transplants have become so routine as to be rarely a source of any discrepancy between requesting provider and authorizing health plan. By contrast, other types of transplants, notably autologous bone marrow transplants (ABMT), elicit much controversy as the medical justification for their use for a variety of diseases (such as breast and several other cancers) is still uncertain. Despite years of ongoing clinical trials, there exists a lack of demonstrated overwhelming medical benefits from ABMT that would help move It from the controversial to the routine. (1,2) This lack of clear direction only serves to generate difficulties for all involved in the decision process.

Hence, transplants have a tendency to become high profile issues for health plans and providers. Spilling into the lay media, some these cases have led to lawsuits (3) and fueled some of the anti-managed care legislation by playing to the public's fears and misconceptions about managed care. (4) Managed cares image has thus been tarnished, often inappropriately, by the anecdotal nature of so-called 'horror stories' that fail to accurately reflect the facts of a given clinical situation. Pressure can rapidly build on a health plan and/or employer to approve a transplant that may have scant support from an evidence-based decision model, but is being demanded by a severely ill member and the treating physician. All too often such situations spiral out of control and end up in the legal, media, and legislative arenas, far removed from the original medical context.

Front and center in these cases is the physician executive, representing the plan. employer, or the provider side of the discussion. Frequently, the individual case arises in short order with minimal warning before it is presented, already steeped in a crisis mode, for a decision. It is the physician executive, acting at the junction of the administrative and medical spheres, who then is called upon to separate fact from emotion, evaluate a complex procedure that may well be both life-threatening and life-restoring, and do so within the parameters of the members benefit contract. All too typically, this process must also be done on an expedited basis without the luxury of time to perform an extended analysis and consultation. This scenario demands that the best decision possible be made in the shortest time span. The only way to accomplish this is to be prepared in advance with a coherent approach to handling transplant issues.

For the medical director at a health plan, as well as at the provider organization, the challenge is to develop and implement a consistent, fair, scientifically sound process for handling transplants. This goes well beyond the concepts of "experimental and investigational" language that have long been the mainstay of contracts and regulations. The question today is not simply one of categorizing the procedure as being experimental, but, rather, can the available medical evidence reliably demonstrate a superior outcome due to the procedure versus traditional or less intensive therapy.

In the realm of some of the proposed applications for transplants, that can be difficult. For example, a center recommends a complex, multi-organ transplant for a rare condition. Each of the individual organs has been successfully transplanted for other indications for years. But for this larger procedure, only a handful of previous patients have been so treated, with mixed results. To further complicate the picture, the patient is very Ill and an answer must be given in a short timeframe. What is the physician executive to do in this scenario?

The purpose of this article is to stimulate the reader to research and discuss this dilemma within one's organization, be it on the provider or payer side of the equation. Hopefully, it will lead to the creation of a process to rationally and expeditiously deal with requests like the one described, and so calm the highly charged atmosphere around them. This will facilitate arriving at a decision that is as medically solid as possible. The ultimate goal should be to provide the most appropriate therapy based on the current state of medical knowledge.

Elements of a strategic plan for dealing with transplants

The result of the development process for handling transplant issues should be a strategic plan with the following elements:

* Consistency

Few transplant patients are alike. One problem in discussing this subject with patients, the media, legislators, etc., is explaining that a given diagnosis has a variety of treatments possible based on the individual clinical characteristics. One patient with liver failure may be a transplant candidate; another with the same diagnosis, but a different clinical etiology and status. may not. If the process results in a consistent approach to all cases, then different determinations due to these various clinical situations can be justified.

* Flexibility

This stresses the need to account for the unique nature of each case. This does not mean abandoning consistency of approach, but that the process must be able to account for all relevant, medically related factors.

* Full consideration of all medical factors based on best medical evidence

The process should be directed to elicit the most up-to-date information and incorporate it into the final decision. The field of transplantation is dynamic and requires that new research and data must be evaluated and addressed.

* Ethical consideration for the patient

The best way to achieve this is through a fair, open system. One may disagree with the final conclusion as to whether the transplant is appropriate for a particular patient, but all should be able to acknowledge that the process leading to that endpoint was not operating within a black box.

* Focused on optimal outcomes

Selecting the treatment that will deliver, to the best of our medical assessment, the optimal outcome is the best way to fulfill our ethical obligation to the patient.

Key issues

In developing this strategic plan, there are several key issues that must be understood. This is important, regardless of what role the organization plays in transplants: referring them, performing them, or paying for them. Each step of this path is potentially fraught with trouble; one only needs to recall the tremendous fallout over the liver transplant given to Mickey Mantle to see how all the participants in his care came under intense scrutiny. In broad terms, the following areas are ones that any physician executive who has some responsibility for human organ and tissue transplantation decisions should analyze and understand.

1. Benefit issues

Despite the assertions of various state legislatures and medical societies that claim that health plans are practicing medicine, what a plan really does is determine whether a benefit exists and how to administer it. This is distinct from the provision of service.

It is useful to understand the cascade of events that must occur to determine if a benefit exists. First, is the patient eligible, for example, is the contract in force at the time the service is requested? Second, is the proposed procedure a benefit covered by the contract? Frequently, both physicians and patients may not realize that a certain employer, in purchasing a contract from a carrier, may decline to add coverage for some or even all types of transplants. The employer may also exclude a particular type for whatever reason. Despite any state mandates that may require coverage for a class of transplants or procedures, if the employer is exempted under ERISA, the benefit may not then exist for that patient. This is a common source of confusion and frustration for providers who see similar clinical cases that are covered by the same carrier, yet one patient is approved and another is denied.

2. Medical policy

This is the basis for the medical evaluations made by a health plan. It should be rooted in a solid foundation of scientific evidence, regularly reviewed and updated. Such a policy obviously provides a much better basis for the tough decisions than one that does not have a rigorous base. Managed care is Ideally focused on identifying and promoting best practices. The medical policy should be the embodiment of this philosophy. Contracting issues and cost considerations are not appropriate elements of an evidence-based transplant policy. A policy that seeks to distinguish the most appropriate treatment will inevitably lead to the best cost solution since It will achieve the best outcomes.

As such, the question becomes is the procedure for this diagnosis consistent with the plan's medical policy? An eligible member may be covered for a transplant for one diagnosis but not for another (for example, a heart transplant may be approved for primary cardiomyopathy, but denied for amyloidosis).

3. Mechanism far review of requests

Transplants have entered the public consciousness in the same way other major medical interventions have: we no longer fear having a hysterectomy, open heart surgery, or a transplant as we did 30 or 40 years ago. Most people, including those in medicine, look on them as being almost routine, with high success rates and few complications. Anyone who has worked in this field knows the fallacy of this, yet it has meant that transplant centers are deluged with requests from marginal candidates for marginal indications. These requests, in turn, are passed to the carriers, who may often bring the larger employers into the decision process. As a result, just about everyone m the loop for this decision must consider how to establish a credible review mechanism. It is not enough to simply shift the burden onto another branch of the decision tree.

The first step is to have an internal reviewer with specialized knowledge who can perform a preliminary triage of the request. Frequently this may be enough to identify the answer for your organization. More and more, however, there is a movement towards an outside review panel that overlays the internal review with a (perceived) higher degree of objectivity. The President's Commission on the Patient's Bill of Rights has such an outside panel as a key element. Some of the states are beginning to consider this as well. (5) This becomes increasingly valuable as the possibility of a denial decision may lead to adverse publicity or worse. The routine use of a disinterested third party to pass judgment on controversial issues builds into the ultimate decision a framework of fairness that should be apparent to even a critical observer. (*)

Such a panel is expensive and means surrendering a certain amount of control over the ultimate decision. Certainly, if there is a lapse in the medical policy in terms of its support in the available scientific evidence, an external panel may frequently return decisions that are counter to the answers expected by the organization. However, this is simply part of the new paradigm of medical care. We have moved beyond the era when life and death decisions were made behind closed doors between a physician and patient, and into a time when accountability for decisions is appropriately shared among a variety of stakeholders. Given the asymmetry of information inherent in such interactions in the past, the sharing of accountability was actually minimal compared to the present. It will continue to grow as various stakeholders in the delivery of medical care (consumers, large employers, state and local governments, HCFA, etc.) become even more sophisticated.

4. Multidisciplinary teams dedicated to the process

One of the pitfalls of handling such complicated areas as transplants is the lack of in-depth knowledge of the people assigned to handle them. Often it is a "catch as catch can' situation, with whomever is available being given responsibility for an individual situation. What is needed is a dedicated team that begins to develop experience and institutional memory as to how cases are done. This team should be a multidisciplinary one, with input from legal, contracting, and network management. There should be a medical director who is accountable for the medical evaluation piece. As these people become skilled in addressing the different challenges of each case, a consistent approach and result will be more likely. Obviously, consistency in approach will be directly tied to the overall quality of the process and of the final determinations.

5. A network must be identified and implemented

Key to any medical delivery is a network that Is structured to meet the criteria necessary for good care. It should be of high quality, with measurement of care processes, clinical indicators that are relevant to outcomes, and the outcomes themselves. It should also be structured to provide access that is reasonable and appropriate for the clinical purpose.

Note that for transplants in particular, there are large dollar amounts at stake. There is certainly a motivation on the part of some institutions to set up transplant services in order to obtain a piece of this pie. (6) There is also the "glory factor:" having a transplant unit is a prestige factor that adds to the perceived reputation of a medical center. These considerations may well lead a hospital to establish, promote, or to refuse to close a transplant unit even though there is insufficient volume to justify its existence. When creating a network to support a transplant program, health plans must be aware of the potential problem of low volumes, and avoid including Institutions that objectively have no compelling reason to be delivering this service.

A rigorously credentialed network that can demonstrate meaningful quality outcomes based on statistically significant sample sizes will permit patients to be offered the best location for their procedure. As patients are directed to them, these centers will see their volumes increase (admittedly at the expense of those with marginal volumes), leading to a more experienced staff and unit, with, ultimately, improved quality. Hence, a network based on quality and volume will benefit all participants in the process: patients, physicians, the hospital, and the health plan.

6. Possible legal issues

As transplantation becomes more widespread, it also has become more controversial. From such concerns as when to harvest organs to how to respond to lawsuits over benefit interpretations, it is more common to see what was confined to a medical discussion now include the legal ramifications. This has been demonstrated in cases of adverse benefit determinations leading to legal action against an employer or health plan to force coverage, as previously noted.

Regardless of how the organization perceives such external pressures, it means that a close working relationship between the transplant team and a representative from the legal affairs area is essential. As already stated, this representative should ideally be someone who is regularly involved in the team's work so as to become fully knowledgeable.

While including the legal department in the loop is critical, as is seeking out legal advice whenever it is appropriate, the final decisions must be made by the group primarily responsible and accountable for transplant cases. Although a certain course of action may be deemed of possible risk in terms of leading to a lawsuit by a dissatisfied party, if the decision is based on solid medical reasoning, and withstands ethical and professional scrutiny, it should be followed. Fear of a suit should never dissuade us from pursuing the proper course.

7. The role of regulation

One result of the controversy over transplantation procedures and decisions has been more intervention in the process by state regulation. Just as the anti-managed care movement has taken recourse to the various legislatures, so have advocates of certain treatments succeeded in having enacted state mandates for transplantation. This is a double-edged sword. It frees the health plan and provider from spending time and resources on benefit determination, authorization, candidate screening, etc. But by mandating procedures, it may also prevent the flexibility mentioned earlier as being essential to the process.

If, as in Kentucky, all women with breast cancer regardless of stage, cell type, clinical status, etc., are mandated by law to have autologous bone marrow transplantation if they desire, then much of the preceding discussion is moot. Consumer activists may well see this as a "win." However, this may well lead to unintended results that ultimately may prevent patients from receiving the best care, and even keep physicians from offering it.

Those in health care should realize that much legislation comes about not as a consequence of randomized clinical trials, but from anecdotal information from constituents. By freezing in time the mandates for a certain procedure, along with the indications for it, such regulations are by nature unable to keep up with the dynamic change that characterizes high technology medicine. Using Kentucky as an example again, the regulations that were based on 1991 HCFA data authorize liver transplantation for cirrhosis but not for liver failure due to pulmonary hypertension. This latter indication was not established in 1991, but is now a part of transplant therapy for some patients with cystic fibrosis.

Unfortunately, a child in Kentucky is prevented by law from receiving such a procedure because of this medically outdated mandate. Even if the child were to travel to an institution in another state, since the family's policy is written in Kentucky, payment would still be refused. Once passed, these laws are rarely updated on a consistent basis to take advantage of new information, especially since lawmakers often consider an issue resolved once a law is in force. Those in both the lay and medical communities who see regulation as a means to circumvent transplant decisions that they do not like are best to be wary of the long-term ramifications of bringing the legislators into a complicated medical process.

Moving forward

This outline can be applied to any high technology, high intensity medical procedure. Jt is particularly important that any subject of controversy, such as transplantation policies, be carefully evaluated and a detailed process developed in advance of a problem case being dropped onto the medical director's desk. Pasteur said, when asked why he, of all scientists, seemed to be so prolific in his discoveries, that "Chance favors the prepared mind." An organization with the foresight to have the systems In place to deal with controversy is most likely to avoid it.

At the same time, it will be able to deliver results that maximize the goals of the organization, while it benefits all participants and stakeholders. There can be no substitute for a fair, thoughtful process. The reward will be a high quality decision that the team members charged with providing it can be satisfied with, and reproduce for all future situations. It will also be one that can be taken to the outside world with no concern over its validity and appropriateness. In this era of change and evolving accountability, that is an endpoint to be cherished.

Note

* The use of external panels, endorsed by the President's Commission and required by some states, presents a challenge to the consumer activists and critics who have agitated for them in that they must be prepared to abide by the recommendations of the panel. My concern is that there is an underlying assumption that panels will routinely overrule the decisions being reviewed. If these decisions have been grounded in the medical literature, such reversals may well be uncommon. The litmus test of the sincerity of the advocates of external review panels as a mechanism of control over providers and payers is how they then accept a medically correct, but politically incorrect, determination. References

(1.) Canellos, G.P. Selection bias in trials of transplantation for metastatic breast cancer: have we picked the apple before it was ripe? Journal of Clinical Oncology 1997:15(10), 3169-70.

(2.) Stepehnson, J. Researchers struggle with trials of stem cell transplants for breast cancer. Journal of the American Medical Association 1997:23, 1827-29.

(3.) "Michigan Blues settle cancer lawsuit." Modern Healthcare. September 1. 1997. 30.

(4.) "Questions of Ethics: HMOs establish protocols for 'last chance' treatments." Modern Healthcare, March 10. 1997. 106-8.

(5.) "The Consumer Bill of Rights and Responsibilities: Report to the President". Prepared by the Advisory Commission on Consumer Protection and Quality in the Health Care Industry. November. 1997. 55-60.

(6.) "Cardiac Conflict." The Wall Street Journal. December 12. 1996, A-1 Derek van

Derek van Amerongen, MD, MS, is National Medical Director at Anthem Blue Cross and Blue Shield in Mason, Ohio. His new book, Networks and the Future of Medical Practice, is published by Health Administration Press and is available from the American College of Physician Executives. He can be reached by calling 513/336-3841, via fax at 513/336-4757, or via email at [e-mail]