The impact of price controls on rare disease medicines access and lessons for the US
US policy has long recognized this challenge, beginning with the Orphan Drug Act of 1983 and continuing with recent exemptions for certain orphan indications...
Strategy in rare disease requires specialist expertise and understanding. CRA understands the unique challenges that rare disease companies face, and we implement a distinctive approach to help drive their success.
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We are rare disease specialists.
CRA’s experience stems from hundreds of rare disease engagements across many therapeutic areas.
- 50+Rare disease areas covered
- 500+Rare disease engagements since 2019
Our rare disease offerings span across the product lifecycle.
- Early developmentDiscover
- Pipeline opportunity assessment
- Diagnostics & screening
- Early Pricing Marketing Access (PMA)
- Licensing / commercially reasonable efforts disputes
- Late developmentDevelop
- Market access & evidence generation strategy
- Payer value proposition
- Patient finding
- Landscape assessment & policy shaping
- LaunchDeliver
- Launch pricing strategy
- Payer negotiation
- Launch excellence
- Stakeholder engagement
- Competitive simulations
- Uptake and diffusion
- Life cycle & LOEDefend
- Indication & geographic expansion
- Competitor response strategies
- Price/access/uptake benchmarking and assessments
- LOE strategies
Engagements
- 01Strategic and statistical consulting involving rare disease pharmaceuticalsThe CRA expert reviewed data from a variety of existing studies and analyzed limitations and opportunities regarding the use of such data. Client relied upon...View engagement
- 02Developed dynamic GTN model to guide pricing and contracting strategy for a rare disease assetA leading pharmaceutical company engaged CRA’s Life Sciences consultants for support in developing a pricing and contracting strategy for a rare disease assetView engagement
- 03Evaluating the potential impact of cross-border healthcare for gene therapiesCRA's Rare Disease team evaluated the dynamics of cross-border healthcare using secondary research to explore current precedence and/or previous product...View engagement
Contact a Rare Disease expert.
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Upcoming events
2nd Annual Washington Health Economics Symposium
Annabelle Fowler presents the CRA paper titled “An Analysis of Medicaid CPI Rebates and the Sustainability of U.S. Generic Markets.” Ashutosh Mishra presents...
World Orphan Drug Congress EU 2025
CRA is a proud sponsor of the the largest and most established orphan drug & rare disease meeting of its kind across the globe. From cell and gene therapy,...
Recent and Upcoming Events
The Rise of Competitive Rare Diseases: European Payer Perspectives
CRA and leading European payer experts hosted a 30-minute virtual webinar to explore how growing competitive dynamics are reshaping the rare diseases market...
ISPOR Europe 2024
CRA had four posters accepted at the conference. Kirsten Axelsen and Charlotte Poon present their poster titled “Assessing the economic burden of rare disease...