Voice of the Patient – The Biopharma perspective

by Molly White, Dyne Therapeutics

It has been a banner year for the biopharma industry, with record investment, groundbreaking COVID vaccine efforts and incredible scientific advances. It is a particularly hopeful time, both for the industry and for the people living with diseases that our companies are addressing. At the end of the day, though, it only matters if the resulting therapies drive real improvement in the quality of life of affected community members. To be truly transformative, drug developers must understand the burden a disease presents, what meaningful change looks like to the people living with it and how to design the resulting therapy with that information top-of-mind. That information is equally important to the regulatory agencies that seek to sensitively and effectively make drug assessment decisions based on what patients say matters most.

Achieving this requires dogged and continuous consideration of the end user, and the FDA’s Patient-focused Drug Development initiative is a powerful tool to facilitate this focus.

The newly-released FSHD Voice of the Patient Report is a case in point. The FSHD Society organized the externally-led PFDD meeting, partnering with other advocacy organizations to extend its reach and engagement. The report is now listed on the FDA website.

Capturing how profoundly FSHD changes people's lives

A pre-meeting survey gathered almost 600 responses to help hone and target the content and discussion. Despite the impact of COVID-19 and the necessity for a virtual meeting, the event went swimmingly, with illuminating panel presentations, inclusive conversation, and real-time polling feedback that demonstrated how diverse and geographically diverse the audience was.

More importantly, the report captures in compelling, visceral detail what living with facioscapulohumeral muscular dystrophy is like, and how profoundly it changes peoples’ lives:

• The impact of facial weakness on speech: “Each word I use must be weighed. Will it be difficult to pronounce? Is there another word that would convey the same emotion in a meeting that might be easier for me to use?”
• How quickly and profoundly the disease can progress: “Twenty-two was the last time I used a regular [bathroom stall] because I couldn’t stand without grab bars. [Age] 23 was the last time I drove because, I couldn’t pick my foot up off the pedals… Twenty-five was the last time I stood to take a shower because I fell often. Twenty-six was the last time I walked and … stood on my own.”
• The effect of chronic pain and muscle loss on everything from eating to maintaining employment.

The resulting picture of FSHD disease burden is thorough, explicit, and compelling. And the definition of what constitutes ‘meaningful benefit’ is clear.

We've shared the report with every employee

At Dyne, where we are laser-focused on developing transformative therapies for serious muscle diseases, we’ve already shared copies of the report with every employee. We know what a meaningful FSHD therapy must do. And the same will be true at regulatory agencies when FSHD drug development programs are submitted for consideration.

These meetings are huge undertakings, and we are incredibly grateful for the individuals, organizations, resources and hard work that made this one possible. To those who contributed to this report, you did a superb job of illuminating and elevating the face and voice of the FSHD community. We applaud you for increasing knowledge and understanding for those of us working on investigational FSHD therapies. Thank you.