Partners4Access notes that Duchenne gene therapy, the second approved for Duchenne, received conditional EMA approval and national HTAs struggled to quantify its incremental benefit, with NICE recommending it in England with a commercial agreement in place.
Published
Signal category
Legal & Regulatory
Quote
“2. 𝗗𝘂𝘃𝘆𝘇𝗮𝘁 received conditional EMA approval, and national HTAs have struggled to quantify its incremental benefit. NICE recommended it in England with a commercial agreement in place, accepting a greater level of uncertainty than usual and routing funding through the Innovative Medicines Fund.”
— Partners4Access team
Company
Partners4Access
Global Multi-Award-Winning Access Experts for Orphan Drugs, Cell and Gene Therapies
- Industry
- Business Consulting and Services
- Location
- London, GB
- Company size
- 25 employees
At Partners4Access, we believe patients with rare diseases deserve the right to access the most appropriate treatment. Our vision is ensuring fair access for all key stakeholders. By that, we mean fair access to drugs for rare disease patients, fair value for those that fund treatments, access to the smartest clinical option for the disease expert and a fair re-turn on investment for the orphan drug manufacturer. We work with our biotechnology clients in partnership, to think differently across 5 best practices, and smartly secure access for orphan drugs.
Founded 2015