Paving the way for patient focused drug development

Jun 13, 2017
Paving the way for patient focused drug development

The U.S. Food and Drug Administration (FDA) has delivered a welcome message of cooperation and engagement to the rare neurological disease community during a recent externally led Patient Focused Drug Development (PFDD) meeting.

Organized by patient advocacy organization Cure SMA, the meeting on April 18 hosted senior FDA officials who highlighted the importance of patient engagement and pointed to the strong collaborative relationship between the FDA’s Division of Neurology Products, patients and the pharmaceutical industry that enables input into the design and analysis of clinical trials.

Some 400 individuals participated in the meeting either in person or online along with 20 panelists, representing SMA patients and caregivers, as well as key FDA officials.

Topics discussed included the diagnostic journey for patients, the impact of SMA complications, the use of technology to help patients and their families, and the community’s views on new treatments.

Wilson Bryan, director of the Office of Tissues and Advanced Therapies, told the session, “This mutual education process is what makes drug development most efficient and productive… what we hear will help us to think about clinical trial design, what outcome measures to use in clinical trials, what really matters to patients, and how we as regulators should think about the balance of risks and benefits for patients with SMA” [a genetic disease that causes muscle weakness and progressive loss of movement].

One of the centerpieces of the FDA’s move toward strengthened patient engagement has been the three-year Patient-Focused Drug Development (PFDD) initiative, which was required for the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). This has been put into effect through a series of disease-specific meetings to gather patient perspectives and provide the FDA with the opportunity to hear directly from patients and caregivers about their experiences, the impact of living with their condition, as well as their expectations and priorities for current and future treatments.

To date, 20 PFDD meetings have been held and have been praised for their importance in integrating the patient voice into the pharmaceutical review process; however, the sheer number of different diseases makes it an unrealistic approach going forward.

In response, the FDA has gradually moved to encourage external groups to host their own patient feedback sessions, and this shift will be a critical cornerstone of the next iteration of PDUFA due to be enacted October 1 this year.

To date, externally led meetings have covered a range of different conditions, including amyloidosis, myotonic dystrophy and spinal muscular atrophy. A further three – focusing on Friedreich’s ataxia, tuberous sclerosis and the rare kidney disease CG3 – are due to take place June 2, June 21 and August 4 this year respectively.

As Cure SMA revealed, the panelists and FDA speakers recognized the critical need for a strong community in SMA and to expand this to include industry researchers and regulators. This was a point echoed by Jonathan Goldsmith, associate director for rare diseases at the Office of New Drugs, who said, “The spirit of this organization is a real example to other communities about how you do drug development; how you raise money; how you invest that money in trying to advance things from the laboratory to the clinic; how you give out research grants… and how you get interest from regulated industry.”

For more information on Cure SMA, go to:


SMA community’s voice heard “loud and clear” at last week’s Patient Focused Drug Development Meeting with the FDA (April 26, 2017)

Paving the way for patient focused drug development

Astellas Patient Advocacy is a function within Corporate Affairs at Astellas that focuses on creating, building and maintaining third-party relationships. We serve as a conduit between Astellas and external stakeholders to help improve patient outcomes, improve access issues and address patients’ unmet needs head on.

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