June 2016 – by Luk Maes

LukMaes-100x100This April, thought leaders in the field of adaptive pathways met at the DIA EuroMeeting 2016 in Hamburg to discuss the progress made in MAPPs (“Medicines Adaptive Pathways to Patients”) —as well as the challenges still ahead. Co-chairing the panel “MAPPs: The IMI ADAPT SMART Project” was Luk Maes, Executive Director Scientific Policies Europe, Bristol-Myers Squibb, Belgium. He was joined as co-chair of the panel by Hans-Georg Eichler, Senior Medical Officer, European Medicines Agency, UK. Here, Luk Maes provides some key takeaways from the panel.

While co-chairing the DIA Hamburg 2016 session “MAPPs: The IMI ADAPT SMART Project” it became clear that one of the paramount challenges faced by ADAPT SMART is the need to illuminate the science that drives MAPPs. MAPPs is a concept that, based on scientific innovation, seeks to provide timely access to specific medicines, with a potential to address unmet need in specific, well-defined, patient populations, in a sustainable way. Within this context, ADAPT SMART is an IMI2 Coordination and Support Action that seeks to further the discussion around MAPPs, moving it from a “concept” closer to reality. ADAPT SMART is investigating and exploring conditions and feasibility of the MAPPs concept, uniting all stakeholders for reflection on this holistic perspective on development, evaluation and use of specific medicines, over the totality of their life cycle.

Towards this end, the DIA Hamburg 2016 session “MAPPs: The IMI ADAPT SMART Project” included perspectives from payers, consumers, and HTA bodies. The panel included views from:

  • Ad Schuurman, MBA, MSc, Head of the Business Contact Centre & International Affairs, National Health Care Institute, Netherlands, giving the Payer’s Perspective
  • Francesca Cattarin, Health Policy Officer, BEUC, Belgium, giving the Consumer’s Perspective
  • Sarah Garner, PhD, MPharm, Associate Director – Science Policy and Research, National Institute for Health and Care Excellence (NICE), UK giving the HTA Perspective

Beginning with the consumer’s perspective, Francesca Cattarin emphasised the need to ensure consumers full and harmonised benefit from high quality healthcare across all EU Member States. All consumers should be guaranteed timely access to safe and innovative treatments, allowing them to make informed decisions regarding their health. Cattarin voiced the concern—reportedly shared by many consumers—that an early access scheme via a mechanism such as MAPPs could eventually be detrimental in terms of safety and efficacy for new treatments developed and registered through this pathway.

Cattarin stated that “new” or “innovative” does not automatically equal “effective”. Early access is a worthy and important goal to meet the needs of patients, who do not always have the time to wait for a lengthy traditional development and approvals process. However, it should not come at the cost of lowered standards of scientific evidence for the marketing authorisation (MA), or of the value assessment of these new treatments. The early access versus evidence trade-off is an often acknowledge hurdle in MAPPs discussions—but not an insurmountable one.

By supporting an iterative “life-span approach” to learning about a new medicine and putting in place post-MA knowledge generation, MAPPs foresees to provide more useful data grounded in quality real world evidence. The MA and reimbursement conditions of a product can be re-assessed and revised as more knowledge about a product is gained—and adjustments made to the eligible treatment population. Ultimately, if used correctly, MAPPs can provide more and more useful data than traditional development models.

This kind of flexibility can provide crucial data for patients for whom current treatments aren’t working—and who need new options as soon as possible. It is exactly this flexibility that can invite scepticism, however. So-called “show stoppers” from the payer’s perspective, elucidated by Ad Schuurman, included concerns with:

  • Control on volume (indication, start-stop)
  • Control on data (real life, transparent)
  • Control on costs (adaptive reimbursement, mutual acceptable prices)
  • Restricting MAPPs to appropriate situations
  • Realistic exit or partial exit strategy

[Call out box with a picture of Ad “Half [of payers] do think MAPPs can be a promising way of working for a few diseases where immediate life-threatening issues are at stake. But payers want to see control on volume and costs and data”.

While acknowledging that MAPPs can be a promising way of working, Schuurman made it clear that many payers in Europe remain unclear on the intended scope and aim of MAPPs. MAPPs is indeed not meant to replace traditional development and access pathways—instead, it provides us with another tool to addressing unmet needs. MAPPs also is not meant to create a new regularly tool—another fact that has caused confusion in similar discussions. Rather, it makes use of existing regulatory and legal tools, resulting in a unique toolbox that encourages a new way of looking at how we develop specific medicines in well-specified patient populations and/or conditions. Getting payers on board will require addressing the points laid out above, said Schuurman—and including payers in the conversation early on is a step in the right direction.

Sarah Garner of NICE likewise emphasised the importance of including the full gamut of stakeholders in the conversation around MAPPs early on—precisely for the purpose of clarifying the questions around issues like evidence-versus-efficacy. ADAPT SMART, she noted provides a “breathing space” as the consortium includes stakeholders from payers, HTA’s, patient groups and regulatory bodies—all open to discuss issues like those outlined above. The output, she noted, will be useful in informing policy makers at EU and national level alike.

HTA’s are engaged and interested in working with innovative approaches to development, such as MAPPs, to address areas of unmet medical need. Key points of concern from the HTA standpoint include the need to ensure safety of patients; to collaborate and manage uncertainty in terms of evidence standards; and to develop financially sustainable models. Like Schuurman, Garner pointed out the importance of agreeing on exit strategies—involving payers in the development of MEA’s and ensuring that MEA’s encompass fair risk sharing.

Ultimately, there was no doubt among the panellists that real world data can provide evidence of effectiveness—and that it’s increasing use will undoubtedly impact current medicines development paradigms. The question is just what the role of this data will be—and how we can align HTA, regulator and especially payer needs in a way that, first and foremost, addresses the needs of the patients. Following the discussions of the panel “MAPPs: The IMI ADAPT SMART Project”, I was struck once again by the lack of understanding in what MAPPs actually is and certainly about the misconception about the functioning and role of ADAPT SMART in this process. Further information sessions and publications are in the planning within this Consortium to take care of this.