Really Listening to Patients

It was not easy for the Food and Drug Administration to grant accelerated approval of the drug eteplirsen (exondys51) for Duchenne muscular dystrophy.

But on balance, it was the right decision.

Duchenne muscular dystrophy is a rapidly progressive genetic disease with no cure. It causes muscles to progressively weaken, resulting in loss of mobility, difficulty in breathing, and ultimately death. The condition primarily afflicts boys and is typically diagnosed between the ages of three and six.

In a split vote, an advisory committee of experts recommended that the FDA deny approval of eteplirsen. The evidence showed that the product was safe, but some reviewers were unconvinced the drug slowed the effects of Duchenne.

The 12 families whose sons took the drug in the clinical trials believe that their children benefited from eteplirsen. Understandably, some advisory committee members had trouble reconciling the compelling patient and family testimony with the limited scientific trial results.

In the end, the FDA Commissioner and the Director of the FDA’s Center for Drug Evaluation and Research approved the drug with the requirement that the manufacturer conduct additional studies to better demonstrate that the medicine works.

I recognize the objections to the Agency’s decision, but the FDA’s determination was grounded in both clinical data and the values and experiences of families affected by Duchenne, not public pressure. All the families reported eteplirsen slowed progression of the disease based on the aspects of function they value.

Critics of the FDA’s decision might believe that there is insufficient evidence of impact on the length of life. However, incremental changes, preserving function such as walking several years longer than one would without treatment, or maintaining/regaining dexterity to type out a message on an iPad are considered more important to individuals with Duchenne, as was determined by a study by Parent Project Muscular Dystrophy and Johns Hopkins University.

Historically, surrogates have spoken for patients and their family caregivers. Yet, paternalistic views on what is best are often different from what people with chronic conditions and their family caregivers want.

In recent years, the FDA has renewed its commitment to advancing the science of patient input using a methodical approach to supplement – not supplant – traditional clinical research. The Agency recognizes that people with chronic diseases and disabilities have critical information that needs to be part of the approval process.

Since 2013, the FDA has hosted a series of disease-specific meetings with patient stakeholders to address their key considerations and challenges. Some thought the public forums would be a complete waste of time. However, after every meeting people walked away realizing that what surrogates had said was most important to patients was not what people with chronic conditions seek from treatments.

We need to incorporate the patient voice into the entire life cycle of drugs, including the regulatory review process. This recent decision on eteplirsen is a learning moment that should inform our efforts to develop the science of patient-provided information.

In an ideal world, the FDA would have all possible facts about a new treatment before the Agency renders a decision. But we don’t live in a perfect world, and life-altering regulatory decisions are rarely black and white.

In areas of intense gray, the patient perspective is particularly relevant in regulatory decision-making.

Utilizing convincing evidence about eteplirsen’s safety but with limited data on efficacy, the FDA carefully considered the views of the clinical trial participants when granting approval of this drug.

Now boys with Duchenne and their families have an option, and it is one that may give them a chance to live a better life as they define it.


Marc Boutin is CEO of the National Health Council.

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