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BIO Patient & Health Advocacy Summit: The Power of Partnerships

October 22, 2014
This week, the BIO Patient & Health Advocacy Summit powered partnerships in the industry to advance drug discovery by bringing together patient advocacy organizations, government agencies and industry leaders to discuss how to accelerate R&D, preserve patient access, leverage data and build collaborations focused on empowering patients.

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The rallying cry that reverberated throughout the event and over Twitter seemed clear from the beginning and Paul Hastings, Chairman and CEO of OncoMed Pharmaceuticals, Inc. captured it by saying, ‘Let’s stop talking and start doing’ by implementing a new framework for patient engagement. Richard Pops, Chairman and CEO of Alkermes urged everyone to contribute, stating, "Voices of patients are so critical to biotech, we can all get better at engagement – In order to change the world, we must become more organized." Since several speakers pointed out that there are only approximately 500 treatments for the estimated 7,000 rare diseases that exist, clearly the challenge could not be more urgent.

Annie Kennedy, Senior Vice President of Legislation & Public Policy of Parent Project Muscular Dystrophy echoed the sentiment later in the Summit, saying, “There should be nothing about us that happens without us.” John Walsh, President and CEO of Alpha-1 Foundation suggested that advocates in attendance should try to learn from each other and ‘fight the fight together.’

Sally Okun, RN, MMHS of PatientsLikeMe summarized the need for transformative changes by saying, "The patient experience will illuminate the real opportunities."

Throughout the event, speakers shared ideas for moving toward a more patient-centered drug development process – below are highlights:

  • Accelerate drug development – Representative Joe Pitts (R-PA) pointed out that ‘medical progress is not inevitable; we have to bring the future into being.’ Representative Pitts focused his remarks on modernizing the clinical trials process and utilizing new technologies in drug development. He encouraged attendees to submit ideas for consideration to the 21st Century Cures Initiative, which aims to accelerate drug development. Janet Woodcock, MD, Director of the Center for Drug Evaluation and Research at the FDA emphasized that patient input on everything from discovery to translation is critical. Dr. Woodcock pointed out that FDA is hosting 20 disease-specific meetings with patients, but added that there is a need to identify how the input is translated into actual practice. Speakers seemed to be in agreement that collecting data and anecdotes is not enough to bring about change; there needs to be a systematic process for using the data to guide drug discovery.

  • Informed Benefit-Risk Assessment – Advocates stressed the need to incorporate patient preferences when assessing the benefits and risks of a treatment. Kim McCleary, Director of Strategic Initiatives at Faster Cures, asserted that agreeing on facts is not the issue since a person’s values come into play when making decisions, adding, “We have the opportunity to move beyond agreement on facts and begin to understand values.” The Food and Drug Administration Safety and Innovation Act (FDASIA) created the opportunity to incorporate the patient viewpoint, but now we must determine how we put that into practice.

  • Leverage data – Speakers discussed the need to capture patient preferences and the patient experience into data that FDA can use. Richard Pops urged biotech companies to collect and share patient-reported outcomes with FDA. FDA’s Ashley Slagle, MS, PhD, suggested that patient groups engage in the development of outcome assessment instruments to better inform the process. Sally Okun called for the creation of an "innovation zone" to facilitate better use of registry data.


The urgency to move quickly served as the underlying theme throughout the Summit. Jill Wood, the parent of a child suffering from Sanfilippo Syndrome, co-founder and treasurer of Jonah’s Just Begun and co-founder of the virtual biotech Phoenix Next, set the tone of urgency. Jill shared the moving story of her fight to save her son’s life by securing funding to research the disease and working to commercialize treatments. “We made the decision to go for it despite the challenges, and that is exactly what we are doing,” said Wood.

More than 300 attendees, representing more than 140 patient groups, government agencies and industry, participated in the Summit, showing a commitment to transform drug development into a patient-centric process.

BIO Buzz caught up with several attendees to get their thoughts on the best idea that they heard during the two-day Summit: