ADAPT SMART General Assembly at the European Medicines Agency, London (January 18th, 2017)

This report highlights the main outcomes of the General Assembly of the IMI ADAPT SMART consortium.
Co-chairs: Hans-Georg Eichler (EMA), Solange Rohou (AstraZeneca), André Broekmans (Lygature)

1. Reports from workshop sessions by Workshop Leaders with Q&A’s

Updates were given on each of the three workshops held the day before:

Workshop WP1: The workshop was used to discuss the outcomes of the first stage of the gap analysis based on the review of IMI mature projects, and to highlight the direction for future work. To progress the analysis, three topics of interest for in-depths analysis were identified: single-arm experimental studies, indirect comparisons & observational studies, and payer evidence requirements. This will be complemented with a further review of IMI projects which were completed recently in order to identify tools and methods to help generating the evidence throughout the lifecycle for a medicinal product entering the MAPPs pathway. The ultimate objective is to make recommendations for future research work.

Workshop D2.07/D3.09: This workshop jointly addressed deliverables in WP2 and WP3. The focus of the workshop was on the available tools to guide appropriate use of medicinal products, and their related ethical and legal challenges. As a result of the D2.07 part of the workshop (Appropriate use), it was decided to expand the survey with additional stakeholders, explore how scenarios’ can play a role to better understand how tools/systems can affect the appropriate use in the different Member States. The break-out session around D3.09 (ethical and legal aspects of prescribing and use by target populations) resulted in a listing of potential legal/ethical issues to be elaborated upon in WP3. This session also concluded with a number of clear recommendations on:

  • Patient education and informed consent;
  • Data collection;
  • Equity of access;
  • Disengagement/exit strategies;
  • Use of patient data.

An important focus for future work is how to involve patients in decision making and manage their expectations.In the meantime, D2.07 and D3.09 teams will jointly work on exploring the topics of the workshop.

Workshop D3.04/D3.08: During the workshop, different scenario’s in oncology, rare diseases and neurodegenerative disorders were explored. During break-out sessions in the morning and afternoon topics related to these scenarios were discussed in more detail. The workshop led to a confirmation of some of the insights from earlier work, but also to a prioritisation of some topics. A topic that came up during the discussions at the workshop was the communication around registries, and the extent to which participation in registries could be made mandatory (including legal and ethical implications). As a next step, findings from the workshop will be consolidated in a joint report of the workshop expected in Q2 2017. In the meantime, D3.04 and D3.08 teams will jointly work on exploring the topics of the workshop.

2. Organisational topics

André Broekmans, the consortium coordinator, highlighted some important issues for the General Assembly to consider including the following changes proposed to the General Assembly by the Navigator Group for the composition of the consortium:

  • AIFA will join the consortium formally per 1st of January 2017
  • BMS has indicated that it wants to leave the consortium per 1st of January 2017
  • As a consequence, Servier and Shire will join the consortium per 1st of January 2017.
  • The General Assembly endorsed the proposed changes which will be reflected in the next amendment.

The members of the International Advisory Board were presented and accepted by the General Assembly.

André Broekmans gave further information on key activities in the coming year about the Mid-Term Review, communications & stakeholder interaction in 2017, and the status of current thinking around post-ADAPT SMART activities. This topic was further addressed later during the General Assembly.

3. IMI perspectives on future projects touching upon “adaptive pathways”

On behalf of the IMI office Nathalie Seigneuret (IMI) gave a brief overview of the current status of IMI and indicated the central role ADAPT SMART plays in the Strategic Research Agenda of IMI2. Furthermore, she also highlighted some potential future projects touching on ‘adaptive pathways’. These include PREFER, BD4BO and RADAR-CNS (all launched), the upcoming Call 10 and the future scientific priorities (e.g. expanded BD4BO, RADAR expansion and potentially ATMPs).

4. Feedback from the “adaptive pathways” workshop held at the EMA, 8 December 2016

Francesa Cerreta (EMA) updated the General Assembly on the workshop held by the EMA in December 2016. She summarised the key conclusions from the workshop:

  • all proposals fit in the current regulatory framework
  • no relaxation of standards is needed
  • no major legal stopgaps have been identified
  • adaptive pathways is a voluntary exercise agreement on endpoints and data is crucial regulators can drive post-authorisation data generation engagement of all relevant partners is an important criterion when selecting cases

5. Interactive session with short presentations and discussions

During the General Assembly the potential future activities of ADAPT SMART were discussed.

It is clear that by its existence ADAPT SMART has already played an important role in bringing together stakeholders with a wide variety of backgrounds and with different agenda’s. The mere existence of such a platform for dialogue will benefit the discussion on MAPPs/adaptive pathways.

Future activities could continue the work of the consortium around specific topics that were identified (e.g. in the WP1 work). Furthermore, it could create synergies with other IMI projects and identify research work be done.

The key to the success of any future activity is the successful conclusion of the work by ADAPT SMART in 2017.