Resources

The Patient Voice Podcast - Episode 1: Co-creating a clinical trial

This episode of The Patient Voice discusses the importance of collaboration when designing a clinical trial and how the early engagement of all stakeholders is critical to long-term success. Hear from those actively involved in this process on how they're achieving exactly that for a rare genetic condition.

Developing a Decision-Making Framework for Expanded Access to Gene Therapy in Rare Neuromuscular Diseases

Developing a Decision-Making Framework for Expanded Access to Gene Therapy in Rare Neuromuscular Diseases

Benefits of Bionical Emas – Phase 1 Services

Pharmacovigilance and CIOMS

Pharmacovigilance and CIOMS

Nuances in Pharmacovigilance case processing in Early Access Programs

Nuances in Pharmacovigilance case processing in Early Access Programs.

Four things to think about: PVG in EAPs

Four things to think about: PVG in EAPs

Ethical Decision-Making for Early Access to Investigational Medicines in Rare Disease

Ethical Decision-Making for Early Access to Investigational Medicines in Rare Disease

Global Early Access Program Takeover

Rescue of a global oncology EAP on behalf of a large pharmaceutical company.

Global EAP for a specialized biotech company

Requirement for a global EAP of a rare disease on behalf of a US biotech partner.

European EAP with tailored RWD strategy

Rapid set-up of EAP for a rare oncology product.

Guide the evolution of a pre-approval access policy for gene therapy (DMD)

Working with a top 20 pharmaceutical company to guide the evolution of their public policy for providing pre-approval access to gene therapy.

Operationalise an EAP for a rare neurodegenerative condition (ALS)

Facing a high unmet need and a history of challenges around early access to treatments.