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.

Diagnosis

• The foundation of treatment is diagnosis.
• What is the state of knowledge?
• Is the disease well characterized? 
• Need to determine phenotyping and develop pro's 
• Are there biomarkers and are they well understood?
• Looking under the hood. 
• Needs to be a push to getting biomarkers standardized and qualified.

Natural History

• What is the natural history of the disease?
• How does it progress over time? 
• Understanding the degree of variability so you can understand treatment affect. Participating in NH studies is an important way you can help. 

Identifying Patients

• Registries play a key role in finding and stratifying patient data.
• We need to be able to find and recruit patients so that trials can be properly conducted. 
• Speed up process by having people ready to go into trials identify the people interested in being study 

Funding Research

• Many groups are funding translational research to further what we learn in early stage drug development and translate that to usable data to develop therapies. 
• Not all basic research is reliable. 

Arm Yourself with Information

• Understanding the state of therapeutics in the disease
  • Are there existing interventions? What is in development? 
  • Is there commercial interest?
  • What is the current environment like? 
• Cause of the disease
  • Understanding the pathogenesis is a key piece of the puzzle. 

Understanding Benefit Risk Equation

• What is meaningful benefit to Patients? 
• What can be measured? 
• Developing PRO’s
• Video testimony and visual evidence of improvement
• Patients can best describe what matters to them. 

Additional Ways Advocates Play a Key Role

• Patient Advocacy groups should be focused on process and how to engage on every stage of development. 
• Participation in Patient Advisory Committee by community is critical to approval process.
• After approval - post market surveillance and data collection with help of patient community is key to understanding long term impact of drugs. 
• Involvement in reimbursement and access issues should also be a priority to all patient groups.