What to Do With Joe: Observations on Patient Interaction and Pre-Approval Access

Author: David Peet, husband and caregiver to ALS/MND Patient Fellow Andrea Peet

In reflecting on my experience at the 28th annual International Symposium on ALS/MND, I did not immediately find myself thinking about treatment updates and research news.  Instead, my focus shifted to a rather unusual place: a fictional teenager facing jail time.

Let me explain.

When observing a panel of presentations focusing on pre-approval access to unapproved medications. The panel included comments from Alison Bateman-House, PhD, MPA (New York University), Jess Rabourn (WideTrial) and Richard Bedlack, MD (Duke University).  I couldn’t help but think of my “Legal Ethics” course in law school and the competing theoretical models for how lawyers should interact with their clients with respect to legal decision-making.

Upon returning home from Boston, I dug out my old textbook and found the following hypothetical about a similar life-or-death situation (paraphrased):[1]

  • You are an experienced attorney who has a long career litigating juvenile court matters. One day, Joe, a fourteen-year-old boy, and his parents come to see you.  Joe has been charged in juvenile court with robbery and assault.  Joe says he can’t remember anything other than being at the scene of the crime with two of his friends.  You agree to take Joe’s case and Joe’s parents agree to pay your fees. 
  • During your initial investigation, you learn that Joe has no criminal record but that he does have a history of severe learning difficulties and psychological abnormalities. An independent psychological evaluation indicates that Joe is in need of significant psychiatric counseling to avoid more serious problems.  Despite your attempts to contest the merits of the charges, Joe is found guilty in juvenile court and the district attorney now wants to place him in a detention facility that has little in the way of psychiatric counseling.  Based on your experience, you believe that with the help of a doctor and a sympathetic probation officer, you could still persuade the judge to grant probation with placement at home and therapy under a doctor’s direction. 
  • You discuss the options with Joe and he expresses a clear preference against psychiatric treatment. Based on your experience, you think that placement in the juvenile detention facility would be a decision he ends up regretting.  How do you advise Joe?

This hypothetical establishes the groundwork for a very difficult decision for Joe’s attorney, who finds herself stuck between her presumed understanding of Joe’s best interests and Joe’s stated preference.  Would lawyers taking a more paternalistic view of their role truly serve their client if they pursued a path leading him to psychiatric evaluation?  Alternatively, would lawyers who execute Joe’s plan to go to juvenile detention “diminish the legitimacy of the legal system and profession” by “abdicating responsibility for the consequences of their actions”?[2]

I would be interested in hearing clinicians’ views on how to act on Joe’s behalf.  I’d guess that most of them would quickly conclude that Joe is simply too young, too uninformed on the ways of the world, and too ignorant of the potential consequences of his decisions to have full autonomy in pursuing a particular outcome.  But at what point does caring for patients become thinking for patients?

Indeed, the parallels between the predicaments that Joe’s lawyer and ALS clinicians face are as plain as day.  At the Symposium, clinicians expressed the diverging perspectives on how to resolve concerns over the legal, ethical, and moral consequences of their actions.  For some clinicians at the Symposium, pre-approval access seems like a slippery slope that likens a doctor’s role to that of a pharmacists—a distributor medication incapable of exercising judgment as to the medical necessity of such treatment.  For other clinicians, knowledge pre-approval access is a necessary component of truly treating a patient, and clinicians who ignore avenues for access are guilty of not only some measure of professional negligence but also of failing to truly listen to their patients’ desires.

The tension for ALS clinicians is also magnified as a result of increased patient sophistication and other “gatekeepers” that don’t exist between Joe and his attorney.  First, methods for patients and caregivers across the world to obtain, share, and digest information about new and diverse potential treatment options are increasingly user-friendly and ubiquitous.  The collective sophistication of patients and caregivers is higher now than it has been at any point in the history of the disease.  The patients of today are well-informed, and, by extension, the knowledge gap between patients and physicians has narrowed.  Second, the reality that access to pre-approval treatments is not a bilateral agreement between patient and doctor could dissuade doctors from fully engaging in the process of gaining access.  Industry buy-in, health insurance cost uncertainty, and potential government regulation (albeit streamlined) are all factors that can influence the final outcome of a request. And, perhaps, as a result of the many players involved, clinicians do not feel it necessary to waste a patient’s time (or their own) on an effort that looks less like treatment and more like meeting of the United Nations.

So how do ALS clinicians address this conflict with the Joes and Joannas that ask them about access to unapproved drugs in clinic?  The Symposium did not offer a forum or a panel to address this question directly, nor did it present itself as the time and place for any form of debate.  Indeed, it is my understanding that the 2018 event was the first occasion to scratch the surface of these issues in the Symposium’s history.  That’s unfortunate, because I think clinicians would benefit from at least hearing where others fall on a spectrum of relevant questions, for example: Does the clinician’s view on pre-approval access depend on the patient’s progression rate, age, maturity, gender, or other factors?  If so, what are they?  To what extent, if any, do caregivers’ perceptions play a role in a clinician’s decision?

There is no “right” answer to any of these questions, just as there is no right answer for Joe’s attorney.  But the goal of a session to discuss these issues would not be to identify a right answer or to cause a change in the mindset and approach of clinicians worldwide.  Indeed, the purpose of a symposium is not to achieve uniformity in approach or medical judgment but rather to discuss, debate, and share a variety of views.  A clearer discussion of clinicians’ outlook on drug access—beyond the merits of the drug in question—could inform those clinicians who are uninformed or unfamiliar with the available avenues for unapproved treatments.

After all, shouldn’t all clinicians be ready in case the next Joe walks through their doors tomorrow?


[1] Zitrin, Langford & Tarr, Legal Ethics in the Practice of Law, 3d ed.

[2] Judith L. Maute, Allocation of Decisionmaking Authority Under the Model Rules of Professional Conduct, 17 Univ. Cal. Davis L. Rev. 4, 1049, 1050 (1984)

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