Rare Disease Report
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Patients & Caregivers

Is Compassionate Use in the Best Interest of Rare Disease Patient Groups?

JANUARY 31, 2016
Current debates about “compassionate use” and “right to try” legislative efforts leave out considerations important to rare disease patient groups. The stakes rare disease patient groups have in these debates stem from their financial investments in research programs, their efforts in facilitating rapid availability of treatments, and their hope that nothing will interfere with broad access to new treatments. While debates about compassionate use and right to try legislation often accommodate rare disease patient interests in special access to experimental treatments for individuals in desperate situations, they don’t account for how the broader application of compassionate use can actually work against their other general interests.
 
Given all the attention compassionate use is now receiving, rare disease groups would be prudent to develop a point of view on this subject before finding themselves needing one while in the midst of a policy crisis or media firestorm. They would also be wise to insinuate themselves in current right to try legislative efforts.

What is compassionate use?

“Compassionate use” refers to a particular way in which experimental medicines and medical devices can be made available to people who have life-threatening illnesses and no other established or experimental treatment options. Physicians, on their own volition or at the urging of a patient, work with the manufacturer of the particular experimental treatment or device they want. If the manufacturer is willing, then the physician and manufacturer seek Food and Drug Administration (FDA) approval. An institutional review board (IRB) is brought in if the manufacturer and FDA agree to the treatment request. The patient only gets the treatment after these approvals have been obtained.

When the heart speaks

Compassionate use has been a method for getting experimental treatments to people in desperate straits for decades. Only recently has compassionate use become more widely known and mostly as the result of social media and mainstream media attention a recent case generated. The case involved a young child denied compassionate use access to a drug needed for a life-threatening infection. The manufacturer of the experimental drug denied the request because of concerns about losing time and money needed to get the drug approved quickly for use in all patients who need it. The debates about the initial denial specifically and about compassionate use generally also spurred an orchestrated effort in states to adopt “right to try” laws.
 
The debates and legislative efforts aimed at lowering barriers to compassionate use access come from an understandable interest in helping people with no other options to forestall imminent death. It seems easy to support this idea, prima facie: get FDA out of the way; make it possible for physicians to prescribe experimental products under compassionate use without fear of sanctions; permit manufacturers to provide their products for compassionate use; and permit insurance companies to cover the costs of experimental products. Indeed, it has been easy enough to support legislative actions; by the end of 2015, right to try laws had passed in 24 states and right to try bills are pending in another 17. Were that access to unproved agents was really so simple. It’s not. It’s not in general, and it’s not for rare disease patient groups.

Is ‘Right to Try’ the right approach?

Right to try laws do not deliver on their promises to people seeking last chance treatments and they do not account for compelling reasons why they may not work for patient groups in all instances. They don’t deliver because even though they cast patient access to experimental therapies as a “right”, they do not entail obligations on any persons or organizations to do anything. A right only exists when duties are imposed on others to make it work. Right to Try laws so far create no duties on anyone, and thus, they are bound to disappoint many people who pin their hopes on them for compassionate use access.

Patient groups need to develop their own ‘compassionate use’ policy

The public debates and legislative efforts also fail to account for situations in which patient groups could have good reasons not to support a particular compassionate use case or the adoption of right to try laws. Many patient groups, and in particular rare disease patient groups invest their own money into the development of treatments. They also facilitate the research process by recruiting subjects and contributing data from their own natural history registries. In order for manufacturers to respond to a compassionate use request or to comply with right to try laws, they have to divert funds and personnel allocated for the regulatory approval that makes access more broadly available to a patient group’s constituents. Some of the funds diverted could come from a patient group’s investment, and the consequences of reallocating development personnel could prolong the time it takes before a treatment is available. Therefore, compassionate use cases and right to try laws can work against the objectives of patient groups. Patients groups are thus owed opportunities to bring their points of view into individual compassionate use cases and into right to try legislation.
 
In anticipation of such opportunities, rare disease patient groups should establish their positions on individual compassionate use cases as they would involve experimental treatments relevant to their constituents and that benefit from their direct investments and efforts. Groups should outline the situations in which they can support compassionate use cases and when they cannot. They could consider, for example, the degree to which their funding supports research and development activities, the potential for delayed approval, and the possibility of destructive public reactions. Groups should also develop positions on the considerations that should be given to them under right to try laws. For example, they could urge that patient groups have their own rights in determining when compassionate use cases are approved if the interests of their constituency can be harmed as a result. They should at least ensure that the Right to Try legislation cannot entail any obligations on their part when their interests could be materially harmed.
 
The possibility of not supporting a compassionate use case or right to try laws will seem unfathomable to many rare disease patient groups. They know only too well the need for access to promising experimental treatments among their constituents. And, further, they operate under a very strong societal principle conferring a duty upon all of us to do whatever we can to save anyone whose life is in danger (known as the Rule of Rescue). Yet, patient groups have duties and obligations to larger constituencies that sometimes take primacy over individuals. Compassionate use cases and right to try laws can pose hard choices. Pharmaceutical and medical device makers are now putting together compassionate use policies, legislatures are passing right to try laws, and patient expectations for access to experimental treatments are rising. Rare disease patient groups need to establish informed and thoughtful positions now and make them clear to manufacturers of any treatments that could be used for their members and any lawmakers drawing up right to try laws.

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