ADAPT-SMART

Today’s paradigms of bringing innovation to patients are challenged by new developments, such as a growing patient demand for timely access to promising therapies, and increased fragmentation of patient populations due to better disease stratification. To address these challenges, major adaptations to current paradigms are needed while still staying in the EU legal framework. The ADAPT-SMART project set out to address that gap. ###The project created a multi-stakeholder platform where the conditions and feasibility of medicines adaptive pathway to patients (MAPPs) implementation within the EU/legal context could be discussed. In an interview with the IMI Programme office, project leader Hans-Georg Eichler of the European Medicines Agency (EMA), project coordinator Andre Broekmans of Lygature and deputy project leader Solange Rohou of AstraZeneca explained how the project created a change in the mindsets of players in the healthcare ecosystem, and how this will affect drug development in the future. ‘The IMI project and the nature of this multi-stakeholder collaboration was a key catalyst to accelerate a process that would otherwise have taken much, much longer to happen,’ said Eichler.

• Read full interview
• Visit the project factsheet and website

A new paper published in Nature Reviews Drug Discovery by the ADAPT SMART project highlights the progress made in discussions on Medicines Adaptive Pathways to Patients (MAPPs) through the project, as well as areas where bottlenecks remain. ###MAPPs describes the move to find ways of providing faster access to treatments for patients in urgent need, while ensuring that patients, healthcare professionals and others have sufficient information on the benefits, risks and costs of the treatment. In the paper, the authors argue that ADAPT SMART has helped to foster the evolution of an ‘adaptive mindset’, and they set out a series of ‘building blocks’ to facilitate future discussions on MAPPs. These include the need to ‘focus on speedy access for patient (sub-) populations in urgent need’; the need to use a wide range of study methods for evidence generation (including real world data), and the need to involve all stakeholders, including sponsors, patients, healthcare providers, regulators, HTA (health technology assessment) bodies and payers. The authors conclude: ‘Overall, we remain optimistic that current dynamics driven by new types of products, a more prominent patient voice and the issues of drug access and affordability will bring stakeholders together to address the challenges. We continue to believe that the building blocks of MAPPs and a shared adaptive mindset are the best option to achieving timely access for patients to transformative products in an affordable way, without compromising evidence standards.’

The IMI ADAPT SMART Closing Meeting ‘Medicines Development in the Adaptive Era’ will take place in Budapest, Hungary on 21-22 March. During the event, the final results of the project will be presented and placed into the context of public health and sustainable pharmaceutical innovation in Europe and beyond. ### As part of the programme, MIT’s NEWSDIGS (‘NEW Drug Development ParadIGmS’) programme will host an interactive session to apply the learnings from ADAPT SMART to a case study. All audience members will have the opportunity to contribute to the case study discussions. The meeting will close with a look into the future of adaptive pathways beyond ADAPT SMART. Registration is upon invitation only and seating is limited – to request an invitation, follow the link at the bottom of the event web page.

The ADAPT SMART project has issued a discussion paper on engagement criteria for MAPPs (medicines adaptive pathways to patients) to aid in debates on how and when a MAPPs approach should be used and for which medicines and diseases/conditions. The paper proposes the following set of six questions to consider when selecting individual products for a MAPPs pathway.###

1. Can we define a target population with a high unmet need? Does the product hold sufficient promise to address the unmet need?
2. Can a prospective iterative post- (initial) marketing authorisation development plan be proposed, developed, implemented and agreed?
3. Are there workable tools to ensure appropriate product utilisation?
4. Are there workable ‘strategies’ for payers in case the product under-performs?
5. Is there sufficient commitment and resources from relevant stakeholders to ensure successful interactions?
6. Which critical aspects for pharmaceutical development would need to be considered?

The questions are designed to initially trigger discussions at the company level (i.e. the medicine developer) and subsequently during discussions between the company and other stakeholders. These questions were designed on the basis of input gathered from a wide range of stakeholders, including regulators, payers, HTA bodies, prescribers, patients and companies. The paper is intended to inform and drive future discussions on MAPPs, both within the ADAPT SMART consortium and in the wider scientific and healthcare communities.

The question of whether, and how, patients with serious diseases should be allowed early access to promising new treatments is hotly debated. Now IMI’s ADAPT SMART project has put together a simple guide to early access that begins with the story of two fictional patients.### Jane has advanced cancer and has at best a year or two to live. John has a family history of cancer, but is currently fit and healthy. If both are told about an experimental new treatment that looks promising but needs more testing, their responses will likely be very different. John hopes that if he is diagnosed with cancer in several years’ time, the treatment will be thoroughly tested. Jane cannot wait so long, and is willing to accept the uncertainties that come with the experimental treatment. However, as the project notes, ‘healthcare decision-makers have an obligation to cater to the needs of both Jane and John’. The project goes on to explain how Medicines Adaptive Pathways to Patients (MAPPs) seek to foster access to beneficial treatments for the right patient groups at the earliest appropriate time and in a sustainable fashion. The key features of MAPPs are:

• focuses on the promise to address a high unmet need;
• provides timely access for the target population in need;
• uses an iterative development and assessment plan with evidence generation over the entire life-span of the drug;
• harnesses real world data to inform follow-on licensing and reimbursement decisions and to manage risks;
• provides for the adjustment of the treatment-eligible population;
• manages utilisation;
• facilitates the collaboration of all stakeholders (pharmaceutical companies, patients, regulators, HTAs, payers, health care providers);
• ensures the sustainability of the innovation and healthcare systems.

Information on drug development is often filled with jargon. Now IMI’s GetReal and ADAPT SMART projects  have released new/updated glossaries to facilitate discussions in their respective subject areas.###

GetReal is investigating how to best incorporate ‘real world evidence’ into drug development. Its updated glossary, which was the subject of a consultation, also includes a more detailed discussion on questions such as the difference between ‘efficacy’ and ‘effectiveness’.

ADAPT SMART was set up (among other things) to coordinate IMI activities in the area of MAPPs (medicines adaptive pathways to patients). Many of the items in its glossary are drawn from the GetReal glossary. The project welcomes feedback on the glossary.