Customized Market Access is the Better Approach for Rare Disease/Orphan Drugs

The growth of the rare disease therapeutics market and orphan drugs.
· 15 min read

While the number of patients impacted by orphan or rare diseases is relatively modest, the rare disease therapeutics market is growing as orphan drugs transition from niche markets to mainstream. Accelerated interest in these specialty medications, as well as emerging gene and cell therapies, is further augmented by transformative changes to research and development, as well as modernized regulatory pathways. Fortunately, more and more products to treat rare diseases have come to market. With such progress, the nation’s payers are evaluating options to address critical issues such as pricing, value, novel payment and reimbursement mechanisms.

In the U.S., the Orphan Drug Act defines a rare disease as any condition affecting less than 200,000 people. There are about 7,000 known rare diseases and collectively, about 1 in 10 people (or 30 million people) in the U.S. have a rare disease. Rare diseases can range from more well-known conditions such as multiple sclerosis and cystic fibrosis, to diseases most people have never heard of, such as Prader-Willi syndrome and Friedreich’s ataxia.

Bringing a new treatment for a rare disorder to market is often only half the challenge. Reaching niche patient populations, handling complex delivery needs and getting insurers to cover costly drugs all pose significant access barriers. When you think of a rare disease, it’s not simply super-rare genetic conditions. Today, areas like oncology represent areas that attract a lot of interest, and often specific designations that require an innovative approach to ensuring that patients are able to access drugs quickly and continuously and at a price that meets payer expectations for value.

For these and other reasons, market access issues for these drugs requires a customized, innovative approach. Organizations that have the expertise, experience and specialized knowledge set for addressing rare disease populations are few and far between.

Choosing the Right Market Access Partner

Manufacturers face unique challenges in the rare and orphan drug development sector. But as a just-published report by the Institute for Clinical and Economic Review (ICER) titled The Next Generation of Rare Disease Drug Policy: Ensuring Both Innovation and Affordability notes:

To ensure that patients enjoy broad access to future innovation, policymakers and health care industry leaders must consider solutions to focus incentives for innovation and improve affordability of rare disease treatments…

To succeed in bringing such therapies to market to achieve the ICER recommendations, pharmaceutical developers can benefit when they switch from a one-size fits all market access model to a customized management model for product commercialization.

This is the approach that distinguishes Optime Care, a subsidiary of AscellaHealth and a member of the AscellaHealth Family of Companies. Optime Care is a specialty pharmacy and patient management organization that is dedicated to patients with orphan, ultra-orphan and rare diseases, creating the trusted path to streamlined access to therapy, compliance with treatment and improved outcomes. Powered by dedicated teams, nimble, customized market access solutions and exclusive distribution partnerships, Optime Care offers a full suite of integrated services available as stand-alone options or bundled. These services are tailored to maximize therapeutic opportunities and enable drugs to reach their full market potential:

  • Pre-Commercialization for early-stage product insights
  • Outsourced market access solutions
  • Exclusive Distribution, Wholesale, 3PL Services
  • National SP Fulfillment including limited distribution drugs
  • Hub and patient support services
  • Compliance and clinical pull through programs
  • Custom financial solutions and copay programs
  • Data analytics and reporting: purely actionable data from one vendor source

Utilizing a consultative business model, Optime Care offers pharmaceutical manufacturers integrated, single-source strategic partnerships that focus solely on rare disease patients. These programs embed communication touchpoints to support the brand throughout pre-launch, launch and commercialization. This approach and capabilities bring consistency to all messaging and build patient loyalty.

Optime Care’s unique approach to market access for specialty drugs is gaining recognition among manufacturers worldwide:

  • Navigating the Payer Landscape: Our team has the experience and expertise to create an effective payer strategy that reflects a genuine understanding of why payers are willing to cover certain drugs, appreciation of the market forces that determine a drug's value and knowledge regarding the competitive landscape.

  • Flexible Client Market Access Options: We are positioned to partner on a project basis or function as an outsourced unit to address the entire spectrum of market access challenges and negotiate with payers on your behalf.

  • Proven Results: Through our custom programs, our pharma clients build trust with specialty and rare disease patients, providers who prescribe and administer treatments, payers and government agencies that issue treatment approvals and monitor utilization.

To learn more about Optime Care, contact

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