5 Market Access Mistakes That Can Derail Your EU Orphan Drug Launch
Here are 5 key market access mistakes that can derail an EU orphan drug launch:
1. Insufficient Disease Knowledge and Awareness
- Failing to gather comprehensive disease background data, including treatment pathways, patient subgroups, and epidemiology7
- Underestimating the complexity of the science and difficulty in defining unmet needs for rare diseases7
- Not investing enough in disease education and awareness activities for stakeholders7
2. Inadequate Stakeholder Engagement
- Neglecting early engagement with key stakeholders like policymakers, payers, clinicians, and patient advocacy groups7
- Failing to collaborate with patient organizations to develop disease-level knowledge and tools7
- Not aligning with broader EU initiatives like EUnetHTA joint clinical assessments3
3. Underestimating Access Challenges in Different Markets
- Not accounting for wide geographic dispersal of patients and treatment centers across countries3
- Failing to develop tailored strategies for priority vs. non-priority launch markets3
- Overlooking operational challenges like legal/ethical hurdles in different regions3
4. Insufficient Evidence Generation
- Relying too heavily on clinical trial data without real-world evidence5
- Not addressing payer uncertainties around long-term value and budget impact5
- Failing to develop innovative contracting approaches to mitigate high costs5
5. Poor Launch Timing and Coordination
- Launching too quickly without adequate preparation across markets3
- Lack of coordination between global strategy and regional/affiliate execution3
- Not factoring in timelines for reimbursement decisions, which can vary widely (3 months to 2.5 years)1
By avoiding these common pitfalls and taking a strategic, well-coordinated approach to market access, orphan drug manufacturers can improve their chances of a successful EU launch and ultimately expand access for rare disease patients.
Sources:
1. https://www.iqvia.com/-/media/iqvia/pdfs/library/white-papers/from-orphan-to-opportunity-mastering-rare-disease-launch-excellence.pdf
3. https://mtechaccess.co.uk/orphan-medicines-europe-operational-challenges/
5. https://bluematterconsulting.com/insights/blog/shifting-landscape-european-market-access-2-orphan-drugs/
7. https://mtechaccess.co.uk/challenges-orphan-medicines-entering-european-market-disease-knowledge-awareness/