PPMD Testifies at FDA PDUFA Stakeholder Meeting

PPMD's Annie Kennedy has been actively representing our Duchenne community as discussions around the next version of the Prescription Drug User Fee Act (PDUFA VI) have begun between the FDA, industry, and the patient advocacy community. Today, Annie testified before the FDA as to our community's experiences under PDUFA V, our innovation related to Patient Focused Drug Development, and some of our recommendations as to how patient-community engagement in drug development could be further enhanced going forward.

Read Annie's testimony:

Annie Kennedy, Parent Project Muscular Dystrophy (PPMD) Testimony

FDA PDUFA Stakeholder Meeting
White Oak ~ December 17, 2015

Good afternoon, my name is Annie Kennedy and I am the Senior Vice President of Legislation & Public Policy for Parent Project Muscular Dystrophy – PPMD. We are most grateful for the opportunity to reflect on our experiences, and share our ideas here this afternoon.

PPMD is the world’s largest organization focused on ending Duchenne muscular dystrophy. Though treatments have eluded us to date, PPMD and our partners have worked tirelessly to build a robust therapeutic pipeline. The FDASIA reauthorization and PDUFA V aligned perfectly with the dawning of a new day for our Duchenne community – one in which our pipeline of experimental therapies was becoming more robust than ever. We immediately embraced the opportunities presented to us through PDUFA and have worked over the past few years to apply the science of patient input to our Duchenne community.

Specifically, PPMD conducted the first-ever scientifically rigorous survey of parents of Duchenne patients to obtain quantitative evidence as to their views on benefit-risk. Today, we are expanding our patient-preferences studies to include a broader caregiver demographic and young people living with Duchenne, work that will add to this body of evidence and hopefully be factored into agency decision-making.

We have published a series of white papers analyzing PDUFA through the lens of the Duchenne community. These publications highlight our Duchenne community’s policy priorities around the use of adaptive approval, limiting exposure to placebo, and the regulatory importance of a benefit-risk framework specific to each product. They also include narratives that speak to the urgency and unmet need within our community.

We have also led a comprehensive and multi-stakeholder effort to prepare the first ever patient initiated draft guidance to industry developing Duchenne therapies. This PPMD-led guidance was submitted to FDA in June 2014. Our guidance - along with a Duchenne Community Policy Forum convened by PPMD in December of 2013 - was the foundation used by the agency to develop its own draft guidance on the same topic issued in June of this year.


We are encouraged by these tremendous developments and are eager to work with FDA and other stakeholders to continue advancing PFDD and the evolution of the Science of Patient Input to inform and shape the forthcoming user fee package. 

As we look ahead to PDUFA VI, PPMD is focused specifically on two areas:

  1. The need for a mechanism to measure how PFDD tools are being used by the agency and impacting the drug development and review process; and
  1. The need to build upon the PFDD foundation of Benefit Risk within FDASIA with additional process, definition, and structure.

1. Related to a mechanism to measure how PFDD tools are being used and impacting the drug development process:

FDASIA and the corresponding industry agreement contained several provisions focused on greater patient engagement in the review process.

PPMD and other patient groups have developed novel and innovative ways of collecting patient data and information for industry and the FDA. We’ve done this not only in light of the law but in order to better educate ourselves, FDA and companies about things such as the unmet medical need, patients’ risk tolerance, treatment preferences and priorities, and what qualifies as meaningful benefit to our populations.

While patient stakeholders strongly supported these reforms and are working to compile critical information to aid drug review and development, concerns exist as to whether or not  - and, if so, how - these new tools are being used by the FDA.

To address this concern, we are proposing the development of a patient engagement assessment tool, the results of which would be included in the publicly disclosed safety and effectiveness data package of any approved drug.


Essentially we are asking for a checklist that reviewers would fill out indicating what tools were available for a given application and how those tools were used in the review process.

The list of tools could include:

  • Benefit/risk data for the indicated populations
  • Draft or final guidance produced by a patient group
  • Patient-preference data
  • Patient-reported outcomes data
  • Qualitative reports from patient and experts in the community

This simple check list would provide a helpful feedback loop to stakeholders who are spending time and resources on these tools. We need a way of knowing whether or not the tools we are creating are useful to review. If they are not being used or used as desired, this feedback is needed so the community can develop instruments that better meet the needs of the agency. This concept builds upon the success in recent years to strengthen the patient voice in the medical product development process.

Our second area of particular interest and focus as we look to PDUFA VI is the opportunity to…


2. Build upon the PFDD foundation of Benefit Risk within FDASIA with additional process, definition, and structure.

We recognize that regulatory agencies make decisions based on rigorous data, and to that end, we, the patient community believed it would be critical to the FDA’s decision making process if we were able to provide data related to caregiver and patient preferences. So, we as a community set out to ‘quantify the tears’, in an attempt to turn the voice of the patient into accessible data.

In order to accurately measure opinions or preferences we used scientifically validated approaches to ensure that whatever we did, we did correctly. We partnered with social scientists and health economists from Johns Hopkins University. In addition, we collected the narrative stories of our families and found that the stories provided qualitative data in support of the quantitative data collected.

The primary study objective of our first patient-preference study was to explore how parents/guardians of individuals with Duchenne prioritize risk and benefit in the context of new therapies.  Overall, we have found that parent participants prioritized protection of muscle function over any other attribute, including longer lifespan.

In response to requests from FDA, PPMD has been working to expand this work further to capture a larger segment of the Duchenne community population.

Most recently, we partnered with an industry collaborator to understand patient preferences regarding a specific pulmonary candidate therapy. Through this work, involving more than 130 patients and caregivers, we again found that patients are willing to accept risks and burdens to achieve pulmonary benefits, notably improvement in cough strength.


PDUFA VI – Evolving the Building of the Foundation of Patient-Preference and Establishing A Permanent Place for Benefit-Risk within the Regulatory Framework

Informed by ongoing developments including a recent Advisory Committee meeting on a Duchenne candidate therapy, PPMD has been struck by the need to establish a designated forum for the review and discussion of patient preference and related data within the regulatory framework that places no additional burden on either the sponsor or the patient community.

Thanks to the leadership of FDA and this Division, we are seeing Benefit-Risk considerations incorporated throughout the drug-development life cycle and sponsors who are actively engaging with patient communities during pre-clinical development and protocol design, not just when looking to move products into the clinic. BIO, CTTI, and many others have brought thought leaders together to systematically build upon the existing benefit-risk frameworks and the National Health Council, together with Genetic Alliance are working to develop draft guidance around the incorporation of patient perspectives into the drug development process.

We at PPMD are proud to be a part of all of these efforts and to see that the incorporation of patient preferences into regulatory decision-making is not a trend, but rather a paradigm shift that is being carefully and thoughtfully undertaken.

To that end, as we look ahead to PDUFA VI – and PFDD 2.0 – and build upon the tremendous foundation of patient preference work that has been laid to date, we see an opportunity to address questions that have arisen over the past few months as to where and how this information fits into the overall application and review process.

As we look ahead, we believe the patient community would benefit from the following:

  • Clarity and guidance from FDA as to how, exactly, such patient-preference data can be entered into the review process of a specific application, including instances where such information is supported by the sponsor and situations where it is not product-specific yet highly relevant to the issues being considered.
  • A requirement that FDA complete a benefit/risk evaluation for every candidate therapy, even in situations where the agency may question the overall benefit or efficacy of a candidate therapy. If this is the case, such views can still be noted but should be done as part of a defined benefit/risk assessment.
  • Related to the point above, require that applicable patient preference and related information be reviewed at a designated point during any advisory committee meeting on a candidate therapy. Current procedures place the burden on either the sponsor or the patient community to relegate time during respective Advisory Committee review presentations or Open Public Hearing to allow for the presentation of such data, and we believe there must be a clear time and place for such a discussion.

We believe that formalization of the processes for review and discussion of applicable and scientifically rigorous patient-preference studies relevant to a product review throughout the drug development cycle and product review is a natural and critical next step in the evolution of the implementation of the PFDD tools that are being invested in and developed.


In Closing, PDUFA V transformed the landscape – and has changed the way that patient communities, academia, clinicians, industry, and federal partners engage on another. The Duchenne community now stands at a hopeful time – with more than 30 companies working on potentially life changing therapies – and 2 new drug applications filed with the FDA this year alone.

The Duchenne community, like so many of our partners in the rare disease community, has come a long way, but we know that we still have far to go. We at PPMD believe that the patient voice is the blockbuster drug of the century for rare diseases.

We are grateful to the FDA for your leadership and continued commitment to placing patients at the heart of product development – and we look forward to continuing to collaborate and innovate alongside you.


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