Company intelligence
Partners4Access
Global Multi-Award-Winning Access Experts for Orphan Drugs, Cell and Gene Therapies
About Partners4Access
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.
Verified activity
Signals from Partners4Access
4 published signals
Legal & Regulatory
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.
Reported by Partners4Access
Legal & Regulatory
Partners4Access notes that Agamere faced pricing tensions as payers benchmarked it against low-cost traditional corticosteroids, leading to strict prior-authorization gatekeeping by insurers and scepticism from cost-effectiveness bodies.
Reported by Partners4Access
Legal & Regulatory
Partners4Access highlights that Duchenne gene therapy, the first approved for Duchenne, now carries a boxed warning for acute liver injury and an FDA indication narrowed to ambulatory patients aged 4 and over.
Reported by Partners4Access
Research & Knowledge
Partners4Access developed PICO+ to identify and consolidate clinical evidence for PICOs in disease areas with limited HTA precedent and clinical guidelines.
Reported by Partners4Access