Rare Disease Report

Integrating Rare Disease Patients into Pre-Clinical Therapy Development. A Workshop Summary Report from BioPontis Alliance for Rare Diseases

DECEMBER 14, 2016
 BioPontis Alliance for Rare Diseases, an international charitable organization, is announcing a summary report on its initiative to develop best practices in integrating rare disease patients into the earliest stages of drug discovery.   Patients integration into drug development is being emphasized by many regulatory authorities, patient organizations, coalitions and pharmaceutical companies.  However, that effort rarely if ever extends into the earliest drug discovery and preclinical phase, when the actual therapeutic strategy is set.  

The over 7,000 rare diseases represent a huge diversity of illnesses with frequently poorly understood clinical presentation, heterogeneity and progression.  Therapeutics development in this arena must be addressed far more efficiently, which means that patients input needs to enter treatment development planning at the very beginning of drug discovery.   Preclinical development patients’ involvement should improve the later experience in clinical trials, regulatory review and commercial delivery of treatment to patients.  

As a nonprofit organization performing the earliest stages of drug discovery and development, BioPontis Alliance for Rare Diseases partners with academic research institutions and patient organizations with a dual mission: translate science into clinical candidates for rare neurologic diseases, and engage patient organizations to develop novel partnerships that enable patients’ participation on development teams.  BioPontis is uniquely positioned to explore how patients’ representation and engagement could be practiced in treatment development.  

We decided to engage first in a direct dialogue with patients’ organization leaders to learn about what is important to patients and to co- develop practical methods for this new way of working between scientists, clinicians and patients.  The results of two workshops, held in 2016 in the US and Europe with patient organizations within the rare neurological disease community, described in a summary paper entitled “Integrating Rare Disease Patients into Pre-Clinical Therapy Development”, is now available. Please register at http://biopontisalliance.org/patient-input/ to download a copy.
This initiative and workshops were supported by generous contributions from Shire, GlaxoSmithKline, the Baillet Latour Fund and private donors to BioPontis Alliance.

About BioPontis Alliance for Rare Diseases

BioPontis Alliance for Rare Diseases is a 501c(3) public charity in the US (North Carolina) and a foundation of public utility (fup/son) in Belgium (Brussels). Managed by an international Board and executive team, BioPontis partners with patient organizations and clinical researchers to bridge the gap between promising science and new medicines. BioPontis Alliance for Rare Diseases is being established as a global non-profit, inviting corporate, private and foundation philanthropic support. More information, including how contributions to support our programs and mission can be made, at http://biopontisalliance.org. 


Erik Tambuyzer, Chairperson
+32 475 615711 (Brussels, Belgium)
Barbara Handelin, CEO 
+1 610.636.2332 (Philadelphia, USA) 

Richard Basile, COO and Treasurer
+1 919.345.5386 (Raleigh, USA)

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