AMCP Partnership Forum on Rare Diseases Identifies Solutions to Prepare for and Manage Orphan Drugs and Gene Therapies

ALEXANDRIA, Va., Sept. 17, 2020 /PRNewswire/ -- Earlier this month, more than 30 experts representing health plans, integrated delivery systems, health economists, providers, academicians, pharmaceutical manufacturers and other professionals gathered for AMCP's Partnership Forum to address the challenges in managing and providing timely access to rare and orphan disease therapies.

"At AMCP, we strive to ensure every patient has access to the medicines they need at a cost they can afford," said AMCP CEO Susan A. Cantrell, RPh, CAE. "The complex and diverse nature of rare diseases poses a unique challenge in planning for and managing orphan drugs and gene therapies. Our AMCP Partnership Forum participants identified smart and innovative solutions to better serve these small, yet important, patient populations. AMCP is excited to continue pushing these transformative strategies forward alongside pharmaceutical leaders."

AMCP's Partnership Forum "Preparing for and Managing Rare Diseases" was held virtually Sept. 8-10,  and facilitated discussion on identifying key barriers around access and coverage for rare disease therapies, developing recommendations on how to manage one-time versus long-term treatments in managed care settings, and collecting examples of early adopters to create benefits for rare disease therapies.

Attendees analyzed case studies and lessons learned of early adopters to identify opportunities to maintain the affordability of treatments for rare diseases. From this, the following strategies were developed:

• Improve collaboration between the FDA, payers, clinical experts and patients to increase confidence in aligning the FDA-approved label, published evidence and coverage criteria.
• Implement more transparent, timely, and consistent pre-approval communication between biopharmaceutical companies and population health decision makers to improve planning around investigational products for rare diseases.
• Utilize the totality of evidence available for coverage policy development, including input from physician experts and patient advocates, real-world evidence (RWE), and multidisciplinary groups throughout the evidence review and coverage development process.
• Address both the FDA approval requirements and payer evidence needs earlier in the drug development process.
• Consider new patient incentive programs to support the collection of real-world evidence (RWE) and long-term data.
• Share best practices around value-based agreements.

All findings and recommendations from the AMCP Partnership Forum "Preparing for and Managing Rare Diseases," will be published in an upcoming issue of AMCP's Journal of Managed Care & Specialty Pharmacy. In addition, AMCP will host a webinar in March 2021 reporting the findings & recommendations. The event was sponsored by AstraZeneca, Dicerna, Genentech, Novo, NPC, Pfizer, Precision Value, Sanofi, Sarepta, Seattle Genetics, Spark Therapeutics, and Takeda.

About AMCP
AMCP is the professional association leading the way to help patients get the medications they need at a cost they can afford. AMCP's diverse membership of pharmacists, physicians, nurses, biopharmaceutical professionals, and other stakeholders leverage their specialized expertise in clinical evidence and economics to optimize medication benefit design and population health management and help patients access cost-effective and safe medications and other drug therapies. AMCP members improve the lives of nearly 300 million Americans served by private and public health plans, pharmacy benefit management firms, and emerging care models. Visit www.amcp.org.

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SOURCE AMCP