Issue

Orphan Drug manufacturer has submitted dossiers to the FDA and EMA, the product was a new class and treatment for a rare condition.

They had requests from physicians and patients from across Europe and Globally. The company although established had conducted no Access Programs before and did not have a global regulatory or operational footprint. They wished to assess the options and potentially implement a global program.

Action

Uniphar specialist teams

• Provided strategic consultancy reviewing all regulatory options in differing countries designing a European and Global Access Program.
• Implemented a range of country specific programs encompassing
  • Cohort programs – charged and free of charge
  • Named patient programs – charged and free of charge
• Designed all materials, processes, procedures and Liaised with Country regulatory agencies to submit relevant applications.
• Provided bespoke registration & data collection systems with a specialist online portal.
• Provided all logistics operations including storage, dispatch and monies collection.

Outcome

• Over 1500 patient treatments delivered in compliant programs
• RWD collected and analysed for HEOR and regulatory submissions
• Increased experience of product by thought leaders.