Interview Questions for Julie Louise Gerberding

 

 

Q1: Public-private partnerships have been instrumental in recent advancements in medical research. Can you elaborate on FNIH’s approach to fostering collaboration between NIH and life-sciences companies?

 

Q1A:  

Many of today’s medical challenges are so complex that no single institution or organization can solve them alone, so cross-disciplinary “Team Science” is essential. FNIH has mastered the design and management of private-public-patient partnerships that allow NIH and other government agencies to tackle these challenges together with non-governmental organizations. FNIH connects leading biomedical scientists at the NIH with their counterparts at life sciences companies, foundations, patient organizations, academia, and regulatory agencies (including the Food and Drug Administration and European Medicines Agency), to build “bridges to breakthroughs”. FNIH designs and manages partnerships that turn the traditional siloed research model on its head, promotes collaboration among diverse experts, shares data, de-risks investments, and catalyzes rapid and efficient progress in the pre-competitive space.

 

 

Q2: Translating scientific discoveries into tangible medical products can be challenging. How can FNIH, along with biopharmaceutical companies, improve the efficiency of this process to bring new treatments to patients faster?

 

 

Q2A:

One overarching issue is that many promising areas of research and development are not pursued because the science is immature and/or investments are too risky, even when the unmet medical need is great.  By working together, harmonizing and standardizing data collection, and sharing access to diagnostics and other tools, new pathways for disease causation and new targets for drug development can be identified. For 10 years, the Accelerating Medicines Partnership® (AMP®) program has demonstrated the success of this approach. AMP programs have advanced many therapeutic areas, including Alzheimer’s disease, heart failure, autoimmune diseases, Parkinson’s disease, and most recently ALS, among others, and provide open-access databases that other qualified investigators can also utilize.

 

Rare and ultra-rare diseases pose another opportunity for collaborative science to catalyze progress in therapeutic development. The Bespoke Gene Therapy Consortium (BGTC) includes 10 NIH institutes, FDA, and 39 non-governmental partners, including biopharmaceutical companies, patient organizations, and foundations who are working together with FNIH to advance gene therapies for eight rare diseases. Early in 2024, the partnership released a regulatory playbook that provides a roadmap for streamlining product development. It is designed to help investigators and companies navigate the regulatory approval pathway for adeno-associated virus (AAV) gene therapies for rare diseases. We expect this resource will change the definition of a “disease of commercial interest,” and improve the incentives for developing therapies that treat or even cure many rare diseases.

The FNIH Biomarkers Consortium (BC) is another example of how long-standing collaborative partnerships help regulatory approval of new therapeutics. By assessing and validating the utility of diagnostic, therapeutic, safety, and other biomarkers, the speed, efficiency, and affordability of clinical trials can be improved.

 

 

Global Health and FNIH’s Future Priorities

 

Q3: The biopharma industry faces unique challenges such as regulatory hurdles, high costs of research and development, and the need for scalable solutions in addressing infectious diseases. How can FNIH, alongside academic and industry partners, contribute to developing innovative treatments and preventive measures for these critical health issues?

 

Q3A: 

The public-private partnership model unlocks opportunities to tackle the biggest biomedical challenges through dynamic collaboration. With so many diverse biopharmaceutical companies, academic researchers and government laboratories working toward the same goal, the critical resources and information that one organization holds may provide the missing key to a biomedical breakthrough–advances that are only possible through knowledge-sharing.  

 

The Accelerating COVID-19 Therapeutic Interventions & Vaccines (ACTIV) project is a case in point and was critical to accelerating the development of COVID vaccines and therapeutics during the pandemic. Achieved in an unprecedented two weeks, the initiative engaged over 40 organizations across our extensive and ever-expanding network of public and private partners to prosecute eleven master protocols for therapeutics that tested 37 agents within those trials. Rapid, broad data-sharing was essential for advancing patient treatment in a timely fashion, allowing physicians to keep pace with the changing disease.  Lessons learned about expedited product development during this emergency are now being adapted to other scenarios. For example, leveraging existing research networks, centralizing prioritization and governance, employing adaptive trial designs, staging efforts in parallel rather than in series, and perhaps most importantly, engaging patients and their communities end-to-end in the design and conduct of trials are processes that FNIH is employing in our new programs. As a result, our timelines are shortened, as we demonstrated in the recent launch of our AMP for ALS (amyotrophic lateral sclerosis).


 

Q4: Looking ahead, what are FNIH’s key priorities in supporting the development of innovative medical products? How can biopharmaceutical and academic institutions best collaborate with FNIH to achieve these goals?

 

Q4A:

Leading the FNIH is a wonderful opportunity to help accelerate medical progress and build bridges to breakthroughs that truly matter. Our mission, to support the mission of the NIH, builds on three pillars: 1) supporting collaborative research through creative and productive public-private-patient partnerships; 2) supporting promising early- and mid-career scientists, and 3) building trust in science and the value it brings to people. We are expanding our partnerships, engaging in new therapeutic areas, improving our timelines, and buidling our resources to support talented scientists at NIH and in academia.  We are also a founding member on the Coalition for Trust in Health & Science that aims to promote informed health decision-making by identifying and correcting misinformation, and assertively calling out dangerous disinformation.

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