HBA Women in Science (WIS) event focuses on the Future of Personalized Medicine: Navigating Uncertainty in Regulations and Reimbursement

On Wednesday, November 6, 2013 at the Boehringer Ingleheim (BI) campus in Ridgefield, CT, the HBA Metro chapter’s Women In Science (WIS) affinity group in partnership with BI brought together an expert panel of speakers to present and discuss the topic: “The Future of Personalized Medicine (PM): Navigating Uncertainty in Regulations and Reimbursement.”

Personalized medicine (PM) is a rapidly emerging state-of-the-art approach to diagnose, treat and prevent a range of common chronic and degenerative diseases. Public awareness and enthusiasm over PM and its therapeutic potential are quickly growing. As we continue to pave the pathway for PM, there are still some key factors and issues that need to be considered and resolved, especially from the regulatory, reimbursement and patient advocacy perspectives. This motivated the HBA Metro chapter WIS team to bring together a group of experts in the field of PM to discuss some of these issues and challenges in an open forum setting.

The distinguished speakers included Pamela Strode, executive director regulatory affairs, Boehringer Ingelheim; Steve Phillips, senior director, global health policy, Johnson & Johnson; Dr. Sean X. Hu, president, BioStrat Advisory and Sharon Terry, founder and CEO, Genetic Alliance. The moderator was Dr. Jennifer Davidson Hamilton, director of predictive medicine, translation medicine, Regeneron Pharmaceuticals.

Dr. Joanne Palmisano, VP, Regulatory Affairs, Boehringer Ingelheim and Nancy Di Da, executive director, chief diversity and inclusion officer, Boehringer Ingelheim, opened the event with welcome remarks, highlighting the interest and support of BI for the event.

Pamela Strode spoke on the regulatory challenges while describing the unique role and responsibilities of the FDA as well as providing a brief history of diagnostics and therapeutics approved thus far. From Pam’s introduction, it was clearly apparent that there has been significant advancement in the areas of oncology compared to other therapeutic areas. Steve Phillips presented on the reimbursement challenges and questions of PM. The key take-home message was the fact that regulatory approval is essential, but not sufficient to ensure reimbursement. There lies a hard road ahead for payers to determine which PM reimbursement scenario would work: favorable coverage and reimbursement; uncertain coverage and reimbursement; lastly no coverage and reimbursement.

Dr. Sean Hu next spoke to the commercialization considerations for PM. He tied together the regulatory and reimbursement challenges by presenting a case study on Maraviroc (Selzentry by Pfizer), a CCR5 co-receptor antagonist for treating CCR5-tropic HIV-1 and its associated CDx is Trofile ®Test. Sharon Terry gave an inspiring presentation on empowering the public as it relates to healthcare and how patient advocacy plays a critical role as a tool within the healthcare ecosystem. Whilst providing the history was important, highlighting more progressive and impactful ways to engage the public via a participant-directed national disease registry (PEER system) elevated the level of audience engagement.

The panel discussion led by Dr. Hamilton provided further elaboration on the topics presented by each of the panelists. The speakers discussed why the majority of companion diagnostics (CDx) have been developed in oncology versus other therapeutic areas. Dr. Hu stated that he intentionally chose  the case study to highlight PM in other therapeutic areas. The discussion also touched on whether “predictors of efficacy” have more/different regulatory and reimbursement challenges than “predictors of safety”? From a regulatory stand point, if safety was compromised, then a drug would be a no-go in early approval stages. However, it is not a truly straightforward answer. The reimbursement world is still grappling how best to answer the value of PM and whether it is clear that such tests provide sufficient evidence and confidence to confirm the clinical validity and associated predicted health outcomes.

Another question that stirred a lot of dialogue was how the involvement of patients, consumers; patient reported data etc, were going to impact PM? Sharon’s own personal experience (which included having two children with special needs) as well as many years working in this field has been that a well-aware and informed patient and consumer will be the only way forward for PM. The patient has to participate in what is the right treatment for the disease and which treatment demonstrates better efficacy without compromise to safety. The other aspect reflects the tight balance between privacy and access, and how much sharing of data is actuallyneeded for development and advancement of PM-based therapeutics. In summary, the expert panel speakers provided their perspectives on PM and and the focus of what hurdles and challenges regulatory bodies, pharma, the healthcare provider and the patient face in the future.

This event was led by WIS co-leads, Dr. Deshanie Rai, Yasmin Khan and Dr Ashley Goss. Also present to support the event was the rest of the WIS team: Dr. Jean Hom Chow. The corporate BI sponsor volunteers included: Andrea Colombo, Kathryn McCulloch, Heather Morgado, Allison Pinkham, Besty Silva, Julie Staudenmier and Dr. Mary Sylvester. The event achieved high attendance and interesting questions were raised by both the speakers and the audience. 

Click here to view photographs of the event.

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