ADAPT SMART in 2017 – Putting Stakeholder Opinions at the Centre of the Process

It is difficult to imagine, but the IMI’s ADAPT SMART project has been operational for eigtheen months, having launched officially at the EMA on September 4, 2015. ADAPT SMART is investigating the tools and (processes) interaction moments required for the use of Adaptive Pathways, but seeks a consensual, multi-stakeholder approach to the continuous lifespan evaluation of the evidence used in regulatory decision making for new therapies that address medical needs.

Since going live, ADAPT SMART’s consortium members have made considerable progress, results of activities undertaken according to plan are now being published on its website. However, whilst many of these deliverables were clearly defined at the project’s inception, what is also required is for the ADAPT SMART consortium to absorb the feedback of the many impacted stakeholders in Europe to address their criticisms, incorporate their suggestions and questions into the evolving scope of the deliverables and research.

MAPPs require a broad agreement amongst stakeholders on the evidence required before going live. Given this, the decision of which products are allowed to be placed into a MAPPs pathway, and how these products are defined for reimbursement, is proving to be the most sensitive set of issues.

Falling under “Engagement Criteria”, ADAPT SMART has been structured around patient needs at its core and several of the key deliverables were lead by EURORDIS and MSD, supported by a number of other colleagues. The recently published discussion paper on engagement criteria is available for download here.

According to Mathieu Boudes, Operations & Projects Manager at EURORDIS, “We, as well as the other patient representatives of the project, support the idea of MAPPS as a concept that would allow earlier access to promising transformative products addressing high unmet medical needs, and this, without lowering the level of evidence required which would put patients at risks. Of course, many challenges remain on the way and need to be addressed during the second half of the project”

In order to meet the needs of patients to successfully balance safety and access, ADAPT SMART’s Work Package 1 (WP1) is working closely with consortium members to analyse and monitor tools and methods previously developed by IMI and other EU projects which can be of direct benefit to MAPPs. Led by Elin Haf Davies (CASMI) and Solange Rohou (AstraZeneca) with the co-leadership of Sarah Garner and Jacoline Bouvy (NICE), WP1 will complete its analysis and provide recommendations before the project’s completion. You can read the recent update on WP1 findings soon.

One of the most challenging aspects of the ADAPT SMART project has been the need to develop a comprehensive overview of each and every stakeholder engagement point in the current regulatory and pricing/reimbursement pathway. In doing so, the consortium has a clear picture of what is needed to develop a viable MAPPs concept.

This substantial effort was accomplished by a team led by Mark Mayer of Lilly, Vinciane Pirard of Genzyme and André Broekmans of Lygature, who reviewed each and every step of the current approval process from a MAPPs perspective.

“It was a significant, collaborative task,” said Mayer. “But ultimately, the entire exercise was done for what would come next – the development of a workable adaptive pathway approach that shows the key engagement points of all stakeholders who must be part of the process. It wouldn’t have been possible to achieve this progress without the directional input of all stakeholders along the way”, he concluded.

Designing the MAPPs pathway lies at the centre of Work Package 2 (WP2), where the opinions of payers, regulators, patients, HTA bodies and health care providers need to be aligned to the new methodologies.

Manage Entry Agreements (MEAs) are a tool that could be used by payers or HTA agencies for a MAPPs product to define both additional evidence generation needs and the financial arrangement for launching a product or the required outcomes that an approved product is meant to achieve in order to receive a certain level of reimbursement. Two of ADAPT SMART’s deliverables are focused on exploring the feasibility of different types of MEAs, resulting in the drafting of a paper that will be submitted for publication in a peer reviewed journal as well as a public report of a workshop held in July 2016 now available for download.

Jacoline Bouvy of NICE has been one of the leads of the MEA research stream in ADAPT SMART, holding a series of meetings with other European HTA agencies, payers, and industry stakeholders to ensure their experiences provide the foundation for subsequent work in the project. She has found that, “most MEAs that have been used in recent years are financial, did usually not involve the routine collection of observational data in clinical practice, and payers who have engaged in MEAs have concerns on their practicality.”

Bouvy also says that, “While there is substantial ‘push-back’ on the concept of MAPPs by payers, by conducting this series of meetings we’ve come a long way to raising awareness and building trust. We hope to explore ways MEAs might be able to successfully manage the introduction of MAPPs therapies in areas of high unmet needs”, she said.

The need for robust and workable MEAs supported by all stakeholders is a crucial tool identified within ADAPT SMART’s Work Package 3, which has been outlined in a report here

With ADAPT SMART moving into the final year, the project will continue to deliver meaningful results to aid in the implementation and understanding of MAPPs. While there is a long way yet to travel in reaching the project’s final goals, ADAPT SMART will continue to work with the many impacted stakeholders to address the remaining barriers that need to be overcome before MAPPs is implemented in Europe.