Survey Shows Intensifying Focus on Cost and Demonstration of Value in Increasingly Competitive Orphan Drug Space

BURLINGTON, Mass., June 27, 2017 /PRNewswire/ -- Decision Resources Group finds that more than 40% of surveyed U.S. physicians perceive an increase over the last three years in the degree of payer challenges they face when prescribing for rare diseases. Separately, the majority of surveyed U.S. payers expect orphan drugs (ODs) to have a significant impact on their budgets in the coming years. Together, these data point to growing cost sensitivity in the management of rare diseases at time when innovation continues to fill the pipeline and the best approach to assess OD value remains a topic of debate. 

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Other key findings from the 2017 Access & Reimbursement report entitled Orphan Drugs (US):

• Surveyed payers are fragmented regarding how they expect to control rising OD expenditure on their commercial plans in the future, but stricter prescribing controls, caps on treated patients, and risk-sharing agreements are likely to be utilized.
• Most surveyed payers who have received pharmacoeconomic data from manufacturers report that the data positively affected coverage of the companies' ODs. However, payers' preferred types of outcomes data vary considerably by disease area, warranting early outreach by developers to ensure the most compelling data are provided.
• Months into its launch, Biogen/Ionis's first-in-class spinal muscular atrophy therapy Spinraza (nusinersen) achieved broad coverage on the largest commercial plans within our payer sample. Similarly, payers report that exclusion of Sarepta's Exondys 51 (eteplirsen) for Duchenne Muscular Dystrophy is uncommon, although surveyed prescribers indicate that nonclinical factors preclude drug access for some eligible patients.
• In rare diseases already served by marketed drugs, surveyed physicians expect their prescribing of emerging agents, including Vertex's ivacaftor/tezacaftor for cystic fibrosis and Global Blood Therapeutics' GBT-440 for sickle cell disease, to depend on pricing relative to established competitors. These data may reflect expected reimbursement challenges and/or physicians' perception of these emerging drugs' value.
• Physician survey data suggest that patient advocacy groups play an important role in the advancement of treatment for rare diseases, possibly by influencing developers' pricing decisions and payers' coverage decisions.

Comments from Decision Resources Group Analyst Ian Love, Ph.D.:

• "Our data suggest that surveyed physicians are facing some challenges in accessing orphan drugs for their patients due, in part, to high cost-sharing. Prior authorization restrictions, step therapy, and quantity limits are also commonly encountered, and physicians expect these challenges to intensify over time."
• "Many surveyed physicians and payers report that developers of orphan drugs should focus on improvement in key clinical outcomes against the standard of care; at the same time, an emerging therapy's impact on overall cost of care should also be considered and, ideally, justified when pricing novel therapies."

About Decision Resources Group
DRG, a subsidiary of Piramal Enterprises Ltd., is the premier source for global healthcare data and market intelligence. A trusted partner for over 20 years, DRG helps companies competing in the global healthcare industry make informed business decisions. Organizations committed to the developing and delivering life-changing therapies to patients rely on DRG's in-house team of expert healthcare analysts, data scientists, and consultants for critical guidance. DRG products and services, built on extensive data assets and delivered by experts, empower organizations to succeed in complex healthcare markets.

Media contact: 
Decision Resources Group 
Whitney Goldstein 
617-747-9037 
[email protected]

SOURCE Decision Resources Group

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