June 17, 2019 / Advocacy

Sharing Our Patient Experience

PPMD has been an active voice in rare disease to ensure that our community’s experience of Duchenne is collected meaningfully and integrated fully into all aspects of the drug development life cycle. Today we participated in the newest phase of the effort through our submission of input to the federal register to inform the enhancement of FDA’s inclusion of patient experience data within the benefit risk framework throughout the medical product lifecycle.

To that end, we are focused on three ways that patient experience data and related information can further serve to inform the benefit risk assessment and regulatory decision making, PPMD is focused on the following:

  • The need for reliable integration of patient preference data within the Benefit/Risk Framework itself for use in both premarket and post-market processes.
  • The need for clarity – and inclusion in the Guidance – as to processes for incorporation of relevant patient preference data within regulatory submissions and reviews.
  • The need to establish a designated forum within the Advisory Committee review process for the review of the completed Benefit/Risk Framework and introduction of relevant patient experience data, independent of, and complementary to, sponsor presentations and the Open Public Hearing.

Our Duchenne community has been so integral in so many paradigm shifting policy efforts that it can sometimes be difficult to keep track of how all of these efforts are connected and what progress has actually been achieved. Over the past few weeks, PPMD has been engaged in efforts with the FDA that is the result of direct advocacy and engagement from our Duchenne community — and we wanted to provide you with an update about the impact we are making.

From informing clinical trial design, to participation in clinical studies, to providing context for regulatory and payer decision making, to producing powerful patient preference data — our data must be included in the frameworks that drive these decisions.

For that reason, not only has PPMD and our community been leaders in the collection of this data, but we have also worked to ensure that incentives and requirements for inclusion of this powerful resource exists for all stakeholders. Through collaborative engagements with the FDA and industry — and our leadership on the Patient Focused Impact Assessment Act (PFIA), the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, the 21st Century Cures Act, and PDUFA V & VI – we have worked to carve new pathways for ensuring that our experiences are fully incorporated in all levels of decision-making.

We learned a great deal as our first few products moved through regulatory review. Our community produced powerful, scientific, data to reflect patient experience and we saw firsthand how that data could (and could not) be woven into the product review.

Our Duchenne community is more than a contextual consideration, and our congressional champions recognized this. So together with FDA’s collaboration, we worked with partner organizations and created new mechanisms for ensuring that the relevant patient experience data would be a part of the medical product lifecycle.

These ‘pathways’ were enabled through provisions within both the 21st Century Cures Act and PDUFA VI. Since their passage, FDA has been working hard to develop regulatory frameworks that reflect the spirit of this legislation. Through a series of public workshops and regulatory guidances, FDA is trying to modernize the way that all stakeholders within the ecosystem engage.

We are hopeful that today’s submission will ensure that patient experience data is used throughout the lifecycle of medical products approved in Duchenne.

Our community continues to successfully change the way that families, academia, clinicians, industry, and federal partners engage one another — and we will continue to work to improve the processes that guide development and decision-making.



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