Patient advocacy organizations have been increasingly recognized as equal partners in the medical product development lifecycle.  This is based on the relatively recent realization, as articulated by FDA, that “patients who live with a disease have a direct stake in the outcome of FDA’s decisions and are in a unique position to contribute to the understanding of their disease.”

We help patient advocacy organizations, both small and large, provide a cohesive patient voice to inform sponsors and regulators on the outcomes to study that are of value to patients (that is, those that are “clinically meaningful” to patients), the daily burden of disease to patients, and what is the acceptable risk-benefit profile of treatment to patients.  For example, our attorneys have assisted with the planning and have moderated the majority of the externally-led Patient Focused Drug Development (PFDD) meetings held to date.

We also believe that patient advocacy organizations have a potential role in all stages of product development.  Specifically, we can provide training on product development and the U.S. regulatory framework, as well as help you:

  • Develop a strategy for engaging with sponsors and regulators that maximizes your impact
  • Request and prepare for meetings with FDA
  • Develop ‘patient experience data’ to inform product development and review, including:
    • Focus groups with your patient community or sessions at your patient meetings
    • Externally-led Patient-Focused Drug Development (PFDD) meetings
    • Surveys to capture patient experiences and preferences
    • Voice of the Patient reports and draft benefit-risk frameworks
  • Develop and submit Draft Guidance to FDA
  • Design patient registries and natural history studies
  • Plan for biomarker and Clinical Outcome Assessment (COA) qualification programs, including for Patient Reported Outcomes (PROs)
  • Prepare to provide public testimony at FDA Advisory Committee meetings and other FDA hearings
  • Develop written comments to relevant legislation and FDA policy proposals

Our attorneys contribute to the field of patient engagement by frequently presenting at biopharmaceutical and patient advocacy conferences, such as those hosted by DIA, NORD, EveryLife Foundation, and Global Genes, as well as by testifying before Congress and at FDA hearings.  We also helped develop the Clinical Trials Transformation Initiative’s recommendations on Effective Engagement with Patient Groups Around Clinical Trials, available here.



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