ADAPT-SMART

Accelerated development of appropriate patient therapies: a sustainable, multi-stakeholder approach from research to treatment-outcomes
ADAPT-SMART logo

FACTS & FIGURES

Start Date
End Date
Call
IMI2 - Call 4
Grant agreement number
115890

Contributions
IMI Funding
1 130 000
EFPIA in kind
2 187 631
Other
722 015
Total Cost4 039 646

Summary

Europe faces many challenges in bringing new medicines to patients in an efficient and timely manner. The reasons for this include the high attrition rates in medicine development, a regulatory environment lagging behind rapidly evolving science, and research and development (R&D) and price/reimbursement models that are no longer financially viable.

‘Medicines adaptive pathways to patients’ (MAPPs) refers to a concept that seeks to provide patients with timely access to beneficial medicines, starting from a small group of well-identified patients (often those with little or no alternative treatments). As evidence accumulates on the benefits and risks of a medicine, access may be extended to other groups of patients. MAPPs relate to a flexible pathway covering the entire life cycle of a medicine, from development, through licensing, to patient access (pricing/reimbursement and healthcare delivery).

ADAPT-SMART is a coordination and support action that brings together representatives from key European stakeholder groups - regulatory agencies, health technology assessment (HTA) bodies, companies, payers, patients and health care professionals. Its goal is to create a platform where the conditions and feasibility of MAPPs implementation within the EU regulatory/legal context can be discussed openly and also ensure that MAPPs-related work in IMI is well coordinated.

Achievements & News

ADAPT SMART issues discussion paper on engagement criteria for MAPPs
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.
(January 2017)

ADAPT SMART makes the case for early access
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.
(July 2016)

GetReal and ADAPT-SMART launch glossaries
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. (June 2016)

Participants Show participants on map

EFPIA companies
  • AbbVie Ltd, Maidenhead, United Kingdom
  • Amgen NV, Brussels, Belgium
  • Astellas Pharma Europe LTD, Chertsey, United Kingdom
  • AstraZeneca AB, Södertälje, Sweden
  • Bayer AG, Berlin, Germany
  • Boehringer Ingelheim International GmbH, Ingelheim, Germany
  • Bristol-Myers Squibb Company , Princeton, NJ, United States
  • Eli Lilly and Company Ltd, Basingstoke, United Kingdom
  • European Federation of Pharmaceutical Industries and Associations, Brussels, Belgium
  • F. Hoffmann-La Roche Ltd, Basel, Switzerland
  • Glaxosmithkline Research And Development LTD, Brentford, Middlesex, United Kingdom
  • H. Lundbeck A/S, Valby, Denmark
  • Ipsen Innovation S.A.S., Les Ulis, France
  • Janssen Pharmaceutica NV, Beerse, Belgium
  • Lysogene, Neuilly sur Seine, France
  • Merck KGaA, Darmstadt, Germany
  • Merck Sharp & Dohme Corp., Whitehouse Station, New Jersey, United States
  • Novartis Pharma AG, Basel, Switzerland
  • Novo Nordisk A/S, Bagsvaerd, Denmark
  • Pfizer Limited, Sandwich, Kent , United Kingdom
  • Sanofi-Aventis Research and Development, Chilly Mazarin, France
  • UCB Biopharma SPRL, Brussels, Belgium
Universities, research organisations, public bodies, non-profit groups
  • Danish Health and Medicines Authority, Copenhagen S, Denmark
  • Haute Autorité de Santé, Saint-Denis la Plaine , France
  • Massachusetts Institute of Technology, Cambridge, United States
  • National Institute for Health and Care Excellence, Manchester, United Kingdom
  • University of Oxford, Oxford, United Kingdom
  • Zorginstituut Nederland, Diemen, Netherlands
Small and medium-sized enterprises (SMEs) and mid-sized companies (<€500 m turnover)
  • Stichting Lygature, Utrecht, Netherlands
Patient organisations
  • European Organisation for Rare Diseases, Paris, France
  • Forum Europeen Des Patients - European Patients' Forum, Brussels, Belgium
Non EFPIA companies
  • European Medicines Agency, Canary Wharf, London, United Kingdom

CONTACT

Project leader
Hans-Georg Eichler
European Medicines Agency
Project coordinator
Andre Broekmans
Stichting Lygature
31 (0)6 10139041
andre.broekmans[at]lygature.org
Project management
Pieter Stolk
TI Pharma
pieter.stolk[at]tipharma.com