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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ABOUT
ISPOR
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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