Competitive rare diseases: Setting the scene
In the first installment of a new series on competitive categories in rare diseases, CRA Life Sciences’s Bhavesh Patel and Owen Male explore the emergence of competition in this space and the unique challenges and opportunities it presents across stakeholders and stages of a rare disease. This topic will also be featured at CRA’s upcoming event during WODC Boston. Stay tuned for the next piece in the series, where we’ll examine strategies for successfully navigating competitive rare disease landscapes.
Historically, few manufacturers invested in rare disease (RD) therapies in part due to limited commercial viability, prompting the introduction of orphan drug policies to incentivize and reward development. Not all approvals have been for distinct indications, however, and many rare diseases now have multiple approved therapies, leading to the emergence of competition.
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RDs/RD groups with 2+ approved orphan medicines*
– 9 with 3 approved orphan medicines |
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RDs/RD groups with 6+ approved non-orphan medicines*
– 8 with 11–15 approved non-orphan medicines |
*As of April 2024
Challenges and opportunities
Treatment choice is hugely beneficial for people living with RDs, and competition can drive important economic advantages for payors. However, competition in RDs poses unique challenges and presents key stakeholder opportunities.
Key challenges
Evidence challenges
– Increasing competition further hinders patient identification and recruitment for clinical trials, including specific subgroups
– Higher bar for demonstration of remaining disease burden and unmet need than what is typical in RDs
– Expectations for more robust evidence beyond use of surrogate and/or less validated endpoints
Positioning challenges
– Need to differentiate and show efficacy vs. SoC rather than previously accepted demonstration of effect
– Varying needs and clinical approaches by geography or site of care due to disparities in RD care pathways
– Navigating the trade-off between positioning and potential cost per patient to ensure commercial viability
Key opportunities
Patients and HCPs
– More treatment options and innovation can address unmet needs and improve care
– Patients are more likely to access care and appropriate treatment
Payors
– Competition drives economic advantages and reduces treatment costs
– Optimized care pathways and better use of healthcare resources
Industry
– Early entrants establish the market by identifying KOLs and patients, developing endpoints, etc.
– There are still RD patients who could benefit from new treatments, including as disease prognosis evolves
Emerging competitive arenas
As the dynamics in a specific RD evolve, the stage of competitiveness in that arena will drive the potential impact of the challenges and opportunities and, ultimately, determine where the manufacturer needs to focus their launch or defence strategy.
