That, I think, is part of why I’ve noticed recent controversy about whether patients with life-threatening diseases should have access to treatments that are being researched but have not been proven to be effective, at least to the satisfaction and approval of the Food and Drug Administration. Today the Federal Appellate Court for the District of Columbia today determined that there is no constitutional right of terminally ill patients to have access to drugs still in clinical trials. You can read news stories on that here or here.
For a wider discussion of the issues involved, check out this segment of NPR’s “Talk of the Nation,” from July 30th. The speakers frame the issues well, and provide specific examples. It offers an interesting reflection on today’s Appellate Court decision.
This is a classic discussion balancing individual rights against social needs - in the language of the Georgetown Mantra, autonomy vs. justice, with, admittedly, related concerns from Non-Maleficence and Beneficence. I think we can outline that discussion this way:
Autonomy: I'm dying. I've heard this drug might help me. So far, it looks like it's probably safe. Even if it isn't, I'm already dying, so I have little to lose. I can't get this as part of a study, not even for "compassionate use." My doctor is willing to give it. I'm willing to take the risks. I should have a right to receive this drug, even though it hasn't made it through clinical trials.
Non-Maleficence: As a professional, I am committed to "do no harm" to patients. At this point, the hard question is, what does less harm? If I help this patient get this drug outside of a protocol, I might contribute to the patient's wellbeing. On the other hand, I might contribute to pain and suffering, and might shorten life. I might contribute to false hope now, and despair later.
Beneficence: What is really in the patient's best interest? If this drug works, it may be in the patient's best interest. On the other hand, most drugs that begin clinical trials are never approved. Is it in the patient's best interest to be exposed to a drug that not only might not work, but probably won't work? Is it in the patient's best interest to pursue life at all costs? Or, is it in the patient's best interest to recognize that our knowledge and capacities have limits, and think about how to make the best of the patient's life as it is?
Justice: We have a process in the United States for assessing the efficacy and safety of drugs. While we sometimes complain that the process is too slow, we inevitably complain when a drug proves ineffective and/or unsafe. A critical part of that process is gathering trustworthy data. Trustworthy data comes from well designed and carefully followed study protocols. “Compassionate use” provisions take this into account by requiring that the physician supervising the patient’s care track some information, but don’t provide data as clear as the study protocol itself. Use of medications outside of some study provision would provide no data at all. It could actually delay the completion of a study, and so either release of a good drug or elimination of a bad one. It could put patients through suffering with no countervailing benefit. While an individual might be willing to take the risks of a drug that hasn’t been fully studied, such access could also create risks for others – folks who are ultimately best served by thorough study of drugs before approval – that we as a community cannot permit.
The NPR discussion touched on but did not explore an issue I think is important in the controversy: the ready access to information about diseases and drugs, both in news media and through the internet. Every little study, however preliminary, offering some promise of medical progress gets picked up from medical journals and published in the first section of the paper and in the middle of the evening news. Patient groups and advocacy groups share information on line about their experiences.
Unfortunately, much of that information is preliminary at best, and useless at worst. It’s a long way from promise of a drug in cell cultures or mouse models to determination of efficacy and safety for human beings. Correlation is not necessarily causation; and the fact that it looks like something works doesn’t mean we actually know how it works. This reflects on another ethical category, a secondary category from the four principles of the Mantra: Faithfulness and Truth-telling. A desperate patient may say he or she is willing to accept the risks; but how well does the person actually know the risks? How accurate is the patient’s information, and how preliminary? A patient may assert he or she has “nothing to lose;” but how carefully has the patient been advised about alternatives? How carefully has the patient considered what is involved in making the best of the time he or she has, recognizing that we have not conquered death and do not expect to before the Kingdom comes?
I have served on a hospital’s Internal Review Board for Human Subject Research (IRB). I have participated in addressing requests for “compassionate use.” I have participated in reviewing research protocols specifically written for patients in the latest stages of life-threatening diseases. I have raised the concern that the drug’s known unwanted effects were bad, only to be assured by physicians that the disease by this point was worse. At the same time, these cases always involved drugs for which there was already some documentation of results in human patients, and reasonable expectation that these drugs would meet standards for approval. To consider access to drugs that haven’t gotten that far in the process, that won’t have that level of supervision, even for the sickest patients should cause hard ethical reflection.
I expect that the advocates for this access will ask for review by the Supreme Court. They are our final arbiters of questions of constitutional rights. Many of us will be watching and waiting to see whether the Supreme Court will take the case, and if so, how the Court will decide. Many will watch and wait, fully realizing that some will not live to see the resolution. And, while we watch and wait, we are still called to care for them as best we can.