YourEncore Insights - Life Sciences

The Frontiers of Rare Disease Development: Advances in Regulatory Science with Dr. Tim Franson (Ep. 18 of The Pharmcast)

Posted by Jim Combs on 5/10/17 8:00 AM


This week on The Pharmcast, we catch up with Dr. Tim Franson to discuss the leading edge of rare disease drug development. Dr. Franson has been actively helping Parent Project Muscular Dystrophy, a Duchenne Muscular Dystrophy Patient Advocacy group, which is doing pioneering work collaborating with FDA to inform benefit-risk and establish FDA guidance.

Duchenne has been a great test case, so we sat down with Tim to share his lessons learned and understand what’s happening on the frontiers of rare disease drug development.

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Topics: Rare Disease, Pharmcast

Patient Engagement: Putting Rare Disease Patients First (Ep. 10 on The Pharmcast)

Posted by Jim Combs on 1/30/17 12:00 PM

This week on The Pharmcast, we turn our attention to the topic of rare disease, and more specifically: how to engage and educate rare disease patients to advance development. It's estimated that nearly 30 million Americans, or nearly 1 in 10 people,  are living with a rare disease - and almost 95% of rare diseases don't have any FDA-approved treatment. 

One of the unique challenges and perhaps keys to rare disease development is patient engagement. Patient populations are small by definition. Roughly 50% of patients are children, and benefit-risk equations are completely different from those of more traditional diseases. To learn more about engaging rare disease patients, we sit down with Dr. Lorna Speid, to discuss her stories of rare disease drug development, outline the unique challenges of engaging the rare disease population, and her advice to developers.

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Topics: Rare Disease, R&D, Pharmcast

Alternative Regulatory Pathways for Rare Disease: Breaking the ICE

Posted by Tim Franson, MD on 4/25/16 10:30 AM

There is a heightened sense of urgency by all stakeholders to accelerate development of therapies for rare disease.  The FDA shares this urgency, but it also must protect the public safety and follow the well-defined laws and regulations governing the process.

The challenge (or opportunity) is to develop creative approaches, which accelerate access and stay within the regulatory boundaries – or find ways to shape the boundaries.  Intermediate Clinical Endpoints, or ICE, could represent one such approach. 

I sat down with Dr. Joe Lamendola on April 7th to discuss this approach, and you can listen/watch the full converation below

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Topics: Pharmaceutical Industry, Regulatory, Rare Disease

Regulatory R.A.N.T. - Biomarkers, Pediatrics, Benefit-Risk - Oh my!

Posted by Peter J. Pitts, Tim Franson M.D. on 2/11/16 11:44 AM

To protect sustainable innovation, it is vital to look beyond present conditions and develop proactive interventions for anticipated environmental changes. With development times for new biomedical medicines and devices now spanning years (and sometimes over a decade), regular audits (at least yearly) are essential in recognizing and adapting to key "climate changes."

Given the importance of both identifying new trends and their impact on the development and commercialization of every product and process, YourEncore gathered an eclectic entourage of experts with deep experience in regulatory and industry leadership after the recent RAPS meeting in Baltimore. We call these sessions R.A.N.T.s - Relevant Assessments, New Trends.  We turned the panel discussion into a white paper, full of insider insights on the changing face of biomarkers, pediatric studies and benefit-risk, all of which are integral factors in development planning that exist in a state of constant flux.

The discussion is particularly timely if you've read the FDA's recently released 2016 Guidance Agenda.  We trust you will find this material useful in preparing for the upcoming guidance season as well as informing your product/portfolio management and regulatory strategies.

Download the white paper now. 

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Topics: Life Sciences, Pharmaceutical Industry, Regulatory, Rare Disease, Pharmacovigilance

BioMarin Advisory Committee Outcome - Canary in the Coal Mine?

Posted by Brian Daniels, M.D., Peter J. Pitts on 12/9/15 8:00 AM

The FDA recently held an Advisory Committee meeting to consider BioMarin’s investigatory treatment for Duchenne’s muscular dystrophy.  The outcome caught many by surprise - and many were watching this AdComm as a "canary in the coal mine" for broader patient group involvement.       

The Duchenne’s community has been at the forefront of working with the FDA on drafting guidance on how to bring new therapies to market for Duchenne’s, a deadly disease that has no approved treatments.

The outcome was not entirely expected.  The FDA Division provided the Advisory Committee with evidence that showed an imbalance between efficacy and safety and, despite well-prepared and delivered testimony on the part of patient groups, the science under discussion pointed to ambiguous review options inside the FDA.

How does this outcome impact and inform future rare disease development and  patient group involvement?  YourEncore Executive Partner, Peter J. Pitts, sat down with Brian Daniels, former SVP of Global Development and Medical Affairs at Bristol-Myers Squibb, to share their thoughts.

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Topics: Pharmaceutical Industry, Regulatory, Rare Disease