‘Right to Try’ is no magic wand
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It is impossible for a parent of a child who has received a catastrophic diagnosis to not feel incredibly helpless, particularly if there is no effective therapy available. You feel you’ve failed in your duty as a protector. In this situation, having the opportunity to try a new treatment with few or no strings attached can be a beacon hope. You search for a magic wand. The chance to try an experimental treatment, through a pathway facilitated by FDA called expanded access, is that magic wand for many families.

However, expanded access is not perfect. It requires a patient’s physician to request that a pharmaceutical company make an experimental product available outside of a clinical trial. If the company says yes, FDA must approve the request. In an attempt to simplify this process, the Senate passed a “right to try” bill, S. 204, which eliminates FDA approval of expanded access requests. It awaits a vote in the House. A federal right to try law, however, will not help boys like my sons, who suffered from Duchenne muscular dystrophy.


For 30 years, I have wished for a magic wand that would undo those heartbreaking diagnoses of DMD. Until recently, there was no approved treatment for this condition, which causes severe and rapid muscle loss. I remember the day my son Chris died. My husband and I sat in a room with a multidisciplinary group of physicians who outlined our options. They said these options would likely fail, causing considerable pain and suffering along the way.

By this time, our hearts had broken in places we did not know existed. Together, my husband and I were failures. But we could not, and agreed we should not, put Chris through more treatment. We agreed to stop. 

I walked out that door and into the ICU. I asked about the next antibiotic dose, surgery and dialysis. My mind and my heart were on two different planets. I found it impossible to reconcile my intellectual, rational understanding with my need to save my son. In that moment, I needed and wanted guidance, oversight and data. I wanted to stack the deck with the greatest degree of information possible, and learn from those who have gone before me. 

My search continues for a magic wand that will lessen the pain of a DMD diagnosis, and I am a fan of the FDA as a partner in my search. I do not deny that things should change, that the expanded access process should be easier to navigate and companies should be more responsive to requests. Decisions for parents like us are really, really, hard, and the FDA has significant experience and insight that is useful. That insight comes from experience in reviewing and understanding available data about products in development. It comes from the information about experiences and feedback from others who have used investigational agents that the agency receives. And it comes from years of analyzing and assessing potential benefit and risk—something that patients and parents can’t do in times of tremendous emotional stress and turmoil.

The fact is, patients already have a right to try investigational medical products. They have had that right for decades under a regulatory carve-out of drug development rules that permit patients who've exhausted therapeutic options to try products still in development if they hold promise. But this access is dependent upon the ability and the willingness of manufacturers to make them available. There are myriad reasons why companies are sometimes unable to provide their product outside of clinical trials. There is often a limited supply of the drug, and despite limited assurances in S. 204, complex issues about liability for adverse events remain. Right to try legislation does not change this.

FDA’s track record — it allows virtually all requests to go forward — demonstrates that the agency is not an impediment to access to unapproved products. In fact, FDA adds important guardrails to the process, helping patients and families that don't have adequate information make decisions about whether a particular investigational product is their best bet. This helps agency safeguard patient welfare with dosing suggestions or monitoring for likely side-effects, based on the patient's medical history and current condition.

FDA medical officers understand how products are metabolized, which is important for patients with renal or hepatic insufficiency whose doses must be carefully calculated and controlled. They agency may have information about how products interact with other medications the patient is taking. Slight changes to a treatment regimen can avoid deadly consequences that cannot be foreseen by the treating physician.

And FDA's Institutional Review Board (IRB) requirements ensure that patients and families are adequately informed about the investigational nature of the therapy they’re requesting; S. 204 would also eliminate this important benefit. Informed consent assures that patients understand what is known, and not known about the therapeutic approach, what potential benefits have been seen so far, the risks (both known and unknown) that might be associated with products in various stages of development, or associated with a particular drug class, or experience from ongoing clinical trials.

Investigational drugs are not appropriate for everyone. Most products in clinical trials are eventually dropped because they are too toxic, or simply have no beneficial effect. Some cause pain and suffering. Some cause fatal reactions. Some may extend life with no improvement in quality of life, or result in a profoundly diminished quality of life. 

In stressful, life or death medical situations, patients and their families should be given more information to help them make rational, well thought out decisions about their options, not less. FDA can do this. Right to try is not a magic wand.

Pat Furlong is the founding president and CEO of Parent Project Muscular Dystrophy. Richard Klein is the former director of the FDA Patient Liaison Program; he worked for many years on expanded access policy and contributed to FDA’s current regulations and guidances.