Dr. Janet Woodcock, Director of FDA's Center for Drug Evaluation and Research (CDER) presented the opening keynote at NORD's Rare Diseases and Orphan Products Breakthrough Summit in Washington, DC on October 21.
Her remarks focused on how the patient advocacy community can most effectively accelerate therapy development along the drug development continuum. We were heartened to see such close alignment between PPMD's policy activities in recent years – particularly in the last several months – and the recommendations offered by Dr. Woodcock (see notes from her presentation below). These recommendations suggest that our current strategic path and collaborations with the Agency are having an impact and we believe will help accelerate the therapies we are working to advance.
The Patient Role in the Approval Process - Dr. Janet Woodcock
Dr. Woodcock focused on how the patient advocacy community can most effectively accelerate therapy development along the drug development continuum – from discovery to development to delivery.
Advocacy communities want to drive new interventions and find disease-modifying therapies as quickly as possible. Advocates play a key role in every stage of the process. Traditionally advocates have funded research grants and while this is critical there are many points along the development timeline that patient advocates can impact the speed and efficiency of developing therapies. There is more we can do as Advocates.
Funding research alone won't cut it.
In the last decade many groups have taken a nuanced approach. Intervening in a targeted ways can have a large impact - understanding the ecosystem of development and where to influence it using the patient voice. Dr. Woodcock provided a roadmap to ways patient groups can get involved and what they need to understand in order to be well-informed Advocates.
Arm Yourself with Information
Understanding Benefit Risk Equation
Additional Ways Advocates Play a Key Role