Trends in US payer management of competitive rare disease
Given that average launch prices for orphan drugs are seven times higher than those of non-orphan drugs, and with an estimated 1 in 10 Americans affected by a...
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.
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.
See all Rare Disease expertsRecent and Upcoming Events
Asembia’s AXS26 Summit
CRA is a proud supporter of the conference where attendees span the full spectrum of the pharmacy and healthcare ecosystem, bringing together key...
World EPA Congress 2026
CRA is a proud sponsor of Europe’s largest congress in market access, pricing and evidence featuring 250 speakers, 10 tracks and 1500+ attendees from pharma,...