BURLINGTON, Mass., Oct. 29, 2015 /PRNewswire/ -- Decision Resources Group finds that more than half of surveyed US managed care organization (MCO) pharmacy and medical directors indicated incurring a higher cost per patient for rare diseases than more common diseases treated with specialty drugs, and survey respondents estimated that the budgetary impact of rare-disease pharmacotherapies (orphan drugs) will grow by 50 percent, on average, over the next three to five years. Nonetheless, there remains strong receptivity to high- and ultra-premium priced current and emerging orphan drugs among US payers, given the low numbers of patients affected, high unmet need, and paucity of treatment alternatives, and despite little expectation of realizing cost savings to the health plan with orphan drugs, according to survey findings and insights from interviews with MCO pharmacy directors.

Other key findings from the Niche Markets and Rare Diseases report entitled US Payer Perspectives on Reimbursement for Orphan Drugs: Market Access Implications for Drug Development in Rare Diseases:

  • Among the 60 percent of surveyed US payers who indicated that the review process for coverage decisions differs between orphan drugs and specialty treatments for more common diseases, the majority noted that greater consideration is given to potential improvements in patients' quality of life in reimbursement decisions for orphan drugs.
  • Ensuring broad formulary inclusion for a hypothetical high-price cystic fibrosis (CF) therapy targeting 40 percent of patients is dependent on a strong efficacy profile. Interviewed payers, however, expect to cover Vertex Pharmaceuticals' CF treatment Orkambi, which could target up to half of CF patients and demonstrated only moderate efficacy in clinical trials.
  • Most surveyed payers have an unfavorable view of confining factors in pivotal clinical trials of emerging Duchenne muscular dystrophy (DMD) treatments eteplirsen (Sarepta Therapeutics) and drisapersen (BioMarin), yet they anticipate generous reimbursement for both agents even at ultra-premium price points.
  • Among the two recently approved idiopathic pulmonary fibrosis (IPF) treatments Esbriet (Roche/Genentech) and Ofev (Boehringer Ingelheim), payers either already cover or expect to cover both, although one agent is preferred or will be favored in the future. Our findings suggest that price concessions are a key factor that could drive more favorable access to treatment.

Comments from Decision Resources Group Director Nadja Rozovsky, Ph.D.:

  • "Our study findings, perhaps surprisingly, suggest that US payers will continue to generously reimburse for high-cost orphan drugs in the near- to medium-term, particularly treatments for debilitating, life-threatening diseases, highlighting the remaining strong commercial opportunity in the rare-disease space."
  • "Leveraging commercial acumen from launch of non-orphan drugs to inform the commercialization of orphan drugs is a reasonable approach. However, identifying areas where market access processes for orphan drugs are differentiated, such as level of scrutiny during the formulary review process and the need for involvement of patient organizations and/or physicians, could ease and enhance market access."

Additional Resources:

  • Decision Resources Group will present a webinar based on this report entitled US Payer Perspectives on Market Access and Reimbursement for Orphan Drugs on November 24, 2015.

For more information on purchasing this report, please email questions@teamDRG.com.

About Decision Resources Group
Decision Resources Group offers best-in-class, high-value data, analytics and insights products and services to the healthcare industry, delivered by more than 900 employees across 14 global locations. DRG provides the pharmaceutical, biotech, medical device, financial services and payer industries with the tools, insights and advice they need to compete and thrive in an increasingly complex and value-based marketplace. DecisionResourcesGroup.com.

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Theresa Masnik

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