ADAPT SMART General Assembly: Patients’ Views at the Centre
On 17 and 18 January 2017, the Adapt Smart consortium held its Annual General Assembly at the European Medicines Agency, in London. The two-day event gathered representatives from regulatory bodies, pharmaceutical industry, HTA agencies and patient organisations.
ADAPT SMART is a project funded under the Innovative Medicines Initiative (IMI – JU) that seeks to establish collaborative solutions to foster the development of Medicines Adaptive Pathways to Patients (MAPPs) in Europe, encouraging more efficient ways of developing and regulating medicines. Its key objective is to investigate the conceptual and practical frameworks that could be used in applying MAPPs in the development of medicines, including the required tools and methodologies, and incorporating the viewpoints of all stakeholders. ADAPT-SMART runs from 1 June 2015 until 1 December 2017.
MAPPs is “a scientific concept for medicine development and data generation which allows for early and progressive patient access to a medicine”(EMA), using the various tools that exist under the EU regulatory framework, including scientific advice; compassionate use; conditional approval (for medicines addressing life-threatening conditions); patient registries and other tools that allow collection of real-world data on efficacy and safety for each medicine. MAPPs is not a new authorisation route: as for any other medicines, a marketing authorisation would only be granted if the balance of benefits and risks is positive in the targeted patient group.
The EMA ran a pilot between March 2014 and August 2016 to explore application of adaptive pathways to real medicines under development. (Click here for the report of the pilot.) ADAPT SMART is not linked to the EMA pilot, but started in parallel.
In the framework of the ADAPT SMART General Assembly, EPF was involved in the facilitation of parallel workshops exploring the preliminary results from two work streams: one concerning the uncertainties, legal and ethical issues related to MAPPs, and the second on questions related to appropriate use of medicines developed through MAPPs.
Uncertainties, Ethical and Legal issues
The concept of MAPPs is not yet widely understood, and its practical implications are being explored by ADAPT SMART. MAPPs raises certain uncertainties and concerns, especially for patients and health professionals but also for other stakeholders such as HTA and payers. The aim of this parallel workshop was to share an understanding of the ethical and legal aspects of MAPPs between all major stakeholders, with a special focus on the concerns of patients, healthcare providers and medicines developers under a potential MAPPs pathway; and to explore together potential options for addressing those.
Preliminary results of this work stream showed that concerns are not only around clinical aspects, i.e. the safety and efficacy of medicines developed through MAPPs, but also around potential ethical and legal implications. Through a “what-if? approach” participants looked at potential risks both at short and longer term and investigated options to mitigate those.
The workshop clarified that medicines approval under MAPPs would be under the same conditions as today, meaning the benefit/risk balance would need to be positive. Conditional marketing authorisation would probably be applied, and additional real-world data collection would be part of the authorisation. (For more information, see here.) Workshop participants agreed that appropriate information to patients and healthcare professionals is of key importance for understanding and acceptance. Patient education is also needed to empower patients to participate in equal partners in shared decision-making regarding the choice of therapy; and to increase patients’ willingness to contribute to the ongoing data collection.
The group also touched upon the crucial topic of “informed consent”, or informed decision-making in a clinical context, and what it means from a patient perspective. The medicines approved through MAPPs are authorised medicines, approved under the usual conditions of a positive benefit-risk balance. However, it is possible that there may be changes as new information is gained from ongoing data collection and thus it is vital that patients and health professionals have a proper dialogue about this, and that the patient is fully aware of all the implications of treatment – clinical, ethical and legal (for example what would happen if the indication of the medicine in question is changed after its reassessment, or it is no longer reimbursed or may even be withdrawn).
Appropriate use of medicines by the targeted patient groups
The second workshop EPF was involved in focused on the mechanisms that should be in place to ensure that medicines developed through MAPPs – which would be under conditional authorisation for a very specific patient group for whom the benefit/risk profile has been demonstrated – are prescribed, dispensed and administered only to those patients for whom they were intended.
The objective of this workshop was to present the preliminary results of a survey analysing the currently existing tools/systems for controlling use, their feasibility and possible usefulness for MAPPs; there was a discussion of the views of the various stakeholders on this analysis, and on the ethical and legal implications of restricting prescriptions.
After a lively discussion, participants felt that the definition of “appropriate use” will need to be clarified to ensure everyone has a common understanding of this. Besides this fundamental issue, a combination of different tools would probably work best, as each medicine might present different challenges. The group made some proposals for further research: an in-depth analysis of some medicines already in use which were conditionally authorised should be conducted to examine in more detail how restricting their use worked (or did not work) in the real world. Secondly, multi-stakeholder “managed access agreements” could be explored, where the conditions of use, decision points for revaluation, and implications for each stakeholder group would be clearly set out and agreed between patients, healthcare professionals, the pharmaceutical company and payers/the health system.
Participants also discussed the question of data collection that is such an important aspect of MAPPs, for monitoring patient outcomes and for providing further evidence on the efficacy and safety profile of the medicine. They concluded that registries will likely be key to the success of MAPPs in terms of post marketing data collection. From a basic ethical point of view, patients cannot be obliged to consent to enrol in a registry, but probably with good information, full discussion and by ensuring that data protection issues are properly addressed, patients’ concerns in this regard would be alleviated.
What happens next?
The findings from the workshops, as well as a third workshop which conducted an analysis of current and past IMI projects relevant to developing MAPPs, were shared with the plenary session of the General assembly on 18 January; the project leaders then provided a general update on progress.
The ADAPT SMART project will continue its activities to the end of 2017. EPF will continue to engage actively as a partner in this project and aim to involve the wider patient and healthcare stakeholder community, as we believe that this project offers a unique platform for an open discussion of the different points of view, hopes and concerns of different stakeholders. Especially, it will be vital to continue to have a strong patient voice in the project to ensure that its final recommendations for future research and initiatives are squarely centred around patients’ needs.
Further information is available on the ADAPT SMART website.