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PDUFA and the Patient Voice in Drug Development

Jul 29, 2016 - 2 min read
PDUFA and the Patient Voice in Drug Development


With the government’s reauthorization of the Prescription Drug User Fee Act in 2012 (PDUFA V), the FDA is trying an experiment to gather patient input by disease group rather than an individual treatment. The program launched for 20 disease areas, in which meetings take into account the current state of drug development, the specific considerations of the FDA review division, and the needs of the patient population by eliciting patient perspectives on their disease and treatment approaches.

We caught up with BioMarin Pharmaceutical Executive Director Mary O’Donovan at the DIA Annual Meeting after her panel discussion with representatives from the FDA and the rare disease patient community about patient-centered drug development.

BioMarin’s focus is on treatments for rare diseases, often fewer than 5,000 patients, so gaining additional insight by including the voice of the patient is essential to the company’s drug development plans, O’Donovan says. She notes the importance of incorporating the patient voice into clinical development as early as possible.

“My hope is that there are opportunities for patient organizations…to provide their input and opportunity to speak with the FDA prior to the end of Phase II, that really is the most important time, before the pivotal study is performed, so that the appropriate endpoint is collected and the appropriate degree of evidence of safety and efficacy is collected.”

The benefit-risk assessment is the bedrock of the FDA’s drug approval process, and establishing a therapeutic context – the severity of a condition and the current treatment options – is an important aspect of that assessment. Patients are uniquely positioned to inform understanding of this therapeutic context, according to FDA Director of Strategic Programs Theresa Mullin, who was on O’Donovan’s DIA panel.

Mullin noted that the FDA previously has not had a systematic way of collecting this information from patients. The current mechanisms for obtaining patient input are limited to specific applications under review.

PDUFA V placed a large emphasis on patient-centered drug development and included steps toward better communication between sponsors and patients, which Mullin views as a “real move forward for the patient community.”

Mullins lists several new learnings through its patient-focused drug development initiatives:

  • Patients with serious chronic disease are experts on what it’s like to live with their conditions
  • For progressive degenerative diseases many patients/parents feel an ideal treatment would at a minimum stop progression of their or their child’s loss of function
  • Patients’ chief complaints may not be factored explicitly into drug development plans, such as including measures of drug benefit planned in trials
  • Patients want to be as active as possible in the work to develop and evaluate new treatments
  • Patients want their experience described using words that they consider to best describe how it feels
  • Patients don’t expect the FDA to address all the gaps in current treatment or current approaches to drug development, but they do want the FDA to help identify the most effective pathway for patients to play a major contributing role in the process.

As far as next steps, the FDA wants to advance the science of patient input by developing a more systematic way of collecting patient experience.

The FDA will hold public meetings to discuss the next reauthorization for PDUFA. There is hope from both the FDA and industry that PDUFA VI will take further steps to find better ways to incorporate the patient voice into clinical development.

What would you like PDUFA VI to include to better incorporate the patient’s voice in clinical development?

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