Early Access Strategy — Rare Disease
Rare Disease • Market Access & Medical Affairs
CLIENT
Emerging biotech entering Europe
CHALLENGE
Ahead of the European launch of a life-saving medicine, the company wanted to provide early access for patients while preparing the market with real-world experience, securing a competitive advantage and generating early revenue.
WHAT WE DID
- Evaluated the unmet medical need — a structured assessment of disease characteristics, treatment guidelines, competitor regulatory status and the current treatment landscape
- Assessed clinical differentiation — analysing the medicine's clinical data against competitor products to gauge its differentiation potential
- Mapped Early Access Programmes across Europe — by eligibility criteria, initiator (company vs. HCP), individual vs. cohort coverage, free-of-charge vs. funded, and the impact of EAP data and price on pricing and reimbursement decisions
OUTCOME
- Defined an early-access strategy aligned to the client's preferences — EU4 + UK, a sustainable mix of paid and free-of-charge programmes consistent with the exit strategy, and a mix of individual and cohort programmes
- Built the business case — forecasting EAP potential sales by country, expected patient numbers over time and programme type, ahead of the official EU launches
- Delivered an implementation and resource roadmap — mapping EAP supply and packs, identifying HQ and country FTE needs across Clinical, Medical and Regulatory, and recommending EAP vendor scope and budget
