TBD: A pressing call for harmonising Compassionate use in Europe!
Senior Director for Global Regulatory & Scientific Policy (GRASP)
Merck Serono Limited United Kingdom
- Reflection on the present landscape and future of compassionate use in EU/EEA.
- Discuss on the challenges of compassionate use in EU/EEA.
- How can we achieve more harmonisation and convergence on compassionate programmes use in EU/EEA.
- How can the revision of the Pharmaceutical Legislation can enhance compassionate use in EU/EEA.
- How do we ensure transparency on compassionate use cases.
Industry is committed to developing new medicines for patients and conducting clinical trials to assess the safety and efficacy of unauthorised medicines . Clinical trials are the most common route for patient access to unauthorised medicines,1 however, patients with life-threatening or seriously debilitating illnesses who have exhausted all available treatments, and are ineligible or unable to participate in a clinical trial may seek access to unauthorised medicines. Such pre-approval access is referred to as compassionate use in the EU. During the last revision of the EU pharmaceutical legislation in 2003, the European Commission introduced the Article 83 of Regulation (EC) No 726/2004, which allows the use of an unauthorised medicine to patients with no satisfactory authorised therapies and who cannot enter clinical trials. Twenty years have passed since the last revision of the pharmaceutical legislation and only 6 CHMP opinions were issued on compassionate use by EMA/CHMP. Despite Article 83, there is high variability and significant differences in the requirements and level of guidance given, which currently leads to inequity of patient access due to the following factors:
• Delayed patient access
• Inequity in patient access and duration of access
• Significant variability in local medical practices
• No or inconsistent data collection.
• Slow and fragmented response to emergencies
• Inconsistent regulations across countries
There is a huge opportunity to improve this important regulatory pathway. By improving this regulatory pathway, we can achieve convergence and harmonisation of compassionate use programmes across the EU.
Participants: EC, Member States, patients EMA, EC; Industry
1. Intro: Setting the scene with a short presentation on compassionate use in EU/EEA.
2. Industry, Patients and Member States views
3. Panel discussion: to debate on the present and future of compassionate use in Europe.
4. Audience voting