Growing Numbers of Orphan Drugs Challenge Health Plans

New Orleans, LA, USA—May 20, 2019—ISPOR, the professional society for health economics and outcomes research (HEOR), explored the issue of orphan drug approvals and how they challenge health plans at its ISPOR 2019 annual conference this morning with the issue panel [IP3], “Can Health Plans Address the Challenges to Accommodating the Growing Number of Orphan Drugs Entering the Marketplace?”

Panelists for the session included:

  • Moderator: James Chambers, PhD, MPharm, MSc; Tufts Medical Center, Boston, MA, USA
  • Susan Cantrell; Academy of Managed Care Pharmacy, Alexandria, VA, USA
  • Paul Melmeyer, MPP; National Organization for Rare Disorders, Washington, DC, USA
  • Chris L. Pashos, PhD; AbbVie US LLC, North Chicago, IL, USA

The growth in orphan drug approvals has offered hope to many people who have rare diseases. Innovation in orphan drug development has also provided a significant challenge to health plans in the United States. Orphan drugs target relatively small patient populations, however their high costs, rising numbers, and strong support from patient groups require health plans to confront issues of access and cost. Additionally, since the US Food and Drug Administration often approves orphan drugs with less rigorous designs (eg, smaller sample sizes, lack of study control groups, etc), health plans may have less confidence in the evidence supporting these therapies.

Dr Chambers moderated the session. He provided an overview of the orphan drug market and summarized new research that indicates that health plans restrict orphan drug coverage approximately 30% of the time. Mr Melmeyer offered insights on patient experiences and the consequences that restrictive policies can have on patients. Ms Cantrell provided a health plan perspective and outlined a variety of approaches that plans are taking to address issues presented by the increase in high-cost orphan drugs. Dr Pashos represented an industry perspective. He provided an overview of the difficulties that companies face in evidence generation for orphan drugs as well as the challenges that must be overcome to gain market access.

ISPOR is recognized globally as the leading professional society for health economics and outcomes research and its role in improving healthcare decisions. ISPOR 2019 is the leading global HEOR conference and draws nearly 4000 healthcare thought leaders and stakeholders, including researchers and academicians, assessors and regulators, payers and policy makers, the life sciences industry, healthcare providers, and patient engagement organizations.

Additional information on the conference can be found at:
Conference Information | Program | Registration | Short Course Program | Sponsors | Exhibitors | Press

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ISPOR, the professional society for health economics and outcomes research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE®-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.

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