Gene therapy competitive dynamics: Winner takes all?
This article was originally published in Cell & Gene. It has also been published in Cell & Gene‘s sister publication, OutSourced Pharama. With many gene...
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
- 450+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 strategy
- 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
- 01Evaluating the potential impact of cross-border healthcare for gene therapiesIssue Our client sought to understand the current state of healthcare across borders (also known as medical tourism) and the opportunities and risks this...View engagement
- 02Global pricing and access strategic issues assessment for rare diseases gene therapy portfolioIssue Our client is developing gene therapies for different rare disease indications, some with effective treatments available and others with only symptom...View engagement
- 03Understanding 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