Joint workshop: Patient uncertainties and exploring the ethical and legal considerations of MAPPs (D3.04 & D3.08)

In order to continue the instructive discussions of the ADAPTSMART programme of work, on January 17th 2017, the ADAPT SMART consortium hosted a multi-stakeholder workshop on ‘Patient uncertainties and exploring the ethical and legal considerations of MAPPs’, bringing together two work streams – D3.04 and D3.08. This workshop comprised of 40 representatives including; European regulators, Health Technology Assessment (HTA) bodies, the pharmaceutical industry, patient organizations, health care professionals, and academics.

The workshop was opened by Alicia Granados (Sanofi Genzyme) who provided a background to the MAPPs concept and a status report. Paul Robinson (MSD) provided an industry perspective on the reality of early access schemes, and Anne-Sophie Lapointe (EURORDIS) provided a patients’ perspective.

Participants then joined one of three parallel breakout groups for the day. Participants considered one for three hypothetical scenarios – one oncology, one paediatric rare disease, and one gene therapy.  They were asked to explore uncertainties that may impact on an individual patient and any ethical and legal barriers to implementation of MAPPs. As a follow up, participants were presented with additional possible scenarios to consider, such as a negative shift in benefit: risk balance following further data collection, or a negative shift in cost effectiveness such that continued access to the medicine is placed in jeopardy.