CRA Insights: A landscape assessment of newborn screening (NBS) in Europe
Newborn screening (NBS) has become an integral part of many public health programmes. NBS programmes have minimised patient suffering which results from...
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
- 300+Rare disease engagements since 2017
Our rare disease offerings span across the product lifecycle.
- Early developmentDiscover
- Early PMA assessment
- Pipeline opportunity assessment
- Diagnostics & screening
- Incentives review
- Licensing / commercially reasonable efforts disputes
- Late developmentDevelop
- Market access & evidence generation strateg
- Payer value proposition
- Patient finding
- Landscape assessment & policy shaping
- LaunchDeliver
- Launch pricing strategy
- Payer negotiation
- Launch excellence
- Stakeholder engagement
- Competitive simulations
- Regulatory and P&R policies
- Uptake and diffusion
- Life cycle & LOEDefend
- Indication & geographic expansion
- Competitor response strategies
- Price/access/uptake benchmarking and assessments
- LOE strategies
Engagements
- 01Understanding and segmenting a rare disease marketClient Issue Our client sought to develop a better understanding of the market for a rare disease, including patient diversity and treatment, in anticipation...View engagement
- 02Identification strategy planning support for future disruptive policy trends for orphan drugsOur client wanted to identify key disruptive trends impacting the HTA, pricing and market access environment for orphan drugs across a range of EU and emerging...View engagement
- 03Long-term partnership for pricing and market access launch strategy supportOur client was preparing for the launch of a new orphan drug in Europe and needed support with tactical planning and negotiation skills since most of the...View engagement