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MDF at 2017 BIO International Convention

MDF staff recently attended the 2017 BIO International Convention hosted by the Biotechnology Innovation Organization (BIO). BIO offers a partnering track to patient advocacy groups, enabling meetings with companies having potentially mutual interests.

In addition to corporate meetings, to discuss mutual interests and the case that DM is tractable for therapy development, MDF attended several scientific sessions—summaries of two are below.

How Regulatory Science is Advancing Innovative Drug Development
Speakers: Vikram Sinha Ph.D., Head, Quantitative Pharmacology & Pharmacometrics, and Vice President, Merck; Chris Leptak, M.D., Ph.D., Associate Director of Regulatory Science, Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration (FDA); Lisa LaVange, Ph.D., Director, Office of Biostatistics, Office of Translational Science Center for Drug Evaluation, FDA; Martha Brumfield, Ph.D., President and Chief Executive Officer, Critical Path Institute.

Key points:

  • The FDA Regulatory Science program, in efforts related to PDUFA VI and 21st Century Cures Act legislation, is working hard to establish communications on drug development tools, develop guidances, and promote use of novel trial designs, biomarker qualification, and innovative technologies. 
  • FDA is launching a new biomarker qualification program that provides for more engagement with submitters and is more transparent in making submissions and advice more transparent. Intent is to foster better education of biomarker developers. Information on biomarkers/endpoints qualified in the past will be made public.
  • Critical Path Institute reviewed their experiences in working with FDA on biomarker qualification. Emphasized critical nature of sharing data in precompetitive space. Efforts are to de-risk drug development to lower threshold for pharma/biotech involvement.
  • FDA is working to increase use of adaptive trial designs, including updating of a 2010 guidance document on the topic and advancing analytic data standards. Intent is to become more transparent about what types of trial designs FDA would be willing to consider.
  • Pharma noted that disease progression models are being increasingly used in drug discovery and development. The body of evidence that a drug works must come from multiple directions, including systems pharmacology, drug/disease models, patient factors, dose response studies, and assessments of patient variability.

The Tale of the Many Therapeutic Roads Leading to Treatment of Rare Genetic Diseases.
Moderator: John Mendlein, Ph.D., J.D., President, CEO and Chairman, aTyr Pharma, Inc.
Speakers: Gerry Cox, M.D., Ph.D., Chief Medical Officer, Editas Medicine; Annalisa Jenkins, MBBS, FRCP, CEO, Dimension Therapeutics.

Key points:

  • A novel perspective on natural history studies for rare diseases was presented, highlighting the value of a high-quality cross sectional study with large number of patients over a longitudinal study with smaller population collected over many years. Point was made that this model has facilitated endpoint development for multiple rare disease indications.
  • Criticality of basic understanding of the operative biological pathways in target selection—this was viewed as a key program launch criterion, with companies interested in fairly straightforward biology to address.
  • Problematic issues that have arisen from targeting “cures” for rare diseases were discussed. Defining what is curative may not be helpful; being able to control a disease with a novel therapy is a positive for patients and caregivers. Changing how a disease affects patients’ daily lives may be the best definition of curative. One and done cures can happen, but are very rare.
  • For rare diseases, difficulties that need to be addressed often are clinical trial design, integrating patients into the drug development process before programs reach trials, costs—both of the development program as well as for payers, and having adequate biomarkers and endpoints for early-stage decision making. The point was made that good biomarkers are not compromised endpoints. Rather the best biomarkers may be more accurate/more quantitative assessments of the impact of a drug on a disease.

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