Periodic Reporting for period 2 - ADAPT-SMART (Accelerated Development of Appropriate Patient Therapies: a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes - Sofia ref.: 115890) Reporting period: 2017-01-01 to 2018-04-30 Summary of the context and overall objectives of the project Progress in life sciences and related technologies offers the potential to bring a wide range of beneficial new therapies to patients over the coming years. There will be more personalised or stratified medicines, drug-device products, and advanced therapies that may require new ways of evaluation and managing, following utilisation in daily clinical practice while investigating patients’ populations that could best benefit of them.Today’s paradigms of bringing innovation to patients are also challenged by transformative environmental developments: growing patient demand for timely access to promising therapies, increased fragmentation of patient populations due to better disease stratification, increased payer influence on product accessibility to patients, and pressure on pharma/investors to ensure sustainability of drug development. To address these environmental changes, while maximising the potential of scientific progress for patients in a timely and sustainable way, one needs major “adaptations” to current paradigms, albeit staying in the current EU legal framework. The changes required go far beyond the well-defined remit of today’s regulatory evidence standards. We posit that all decision makers and stakeholders in the healthcare ecosystem will need to explore an integrated and holistic life-span approach to drug development, marketing authorisation (MA) and pricing and reimbursement (P&R) and use in daily clinical practice and monitoring. This life-span approach is hereafter referred to as Medicines Adaptive Pathways to Patients (MAPPs).The overall scope of the project is to establish an enabling platform for the coordination of MAPPs related activities and engage a direct and open dialogue with all relevant stakeholders. MAPPs seeks to foster patient access to beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span in a sustainable fashion. Currently, several initiatives are exploring new pathways to market, e.g. the EMA’s Adaptive Pathway project, the New Drug Development Paradigms (NEWDIGS) initiative at Massachusetts Institute of Technology (MIT, USA), or the UK's Early Access to Medicines Scheme (EAMS). Parties directly involved with these initiatives, or other related projects such as IMiPACT or IMI GetReal, are joining forces in ADAPT SMART with the aim of defining and helping to implement the MAPPs concept.ADAPT SMART is thus aligning a number of major EU stakeholders and acts as a neutral collaborative platform to engage with all relevant stakeholders, including patients’ organisations, industry, SMEs (Small and Medium sized Enterprises), regulators, Health Technology Assessment bodies (HTAs), payers (national and European Networks), academia representatives, and governments/policy makers. Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far During the course of the project a number of stakeholder workshops were organized, which resulted in a number of reports, providing input for future (peer-reviewed) publications. In addition, ADAPT SMART has updated the glossary of terms and has contributed to the public debate about adaptive pathways with several podcasts, webinars, press releases and newsletters, which are available on the ADAPT SMART legacy website. The project’s Closing Event brought together a wide variety of stakeholders from across Europe, with a special emphasis on Central and Eastern Europen EU Member States. During this event, a fictional case was discussed from a MAPPs perspective. The major results of the project could be summarised as follows:1. Criteria established for the engagement of stakeholders in a life-span approach for true innovative medicines;2. Recognition that stakeholders, including payers, should be involved at an early stage of medicines development;3. Definition of the building blocks for an early access, appropriate use and continuous evaluation of medicines;4. There are no legal constraints in implementing an adaptive approach for medicines development, approval and reimbursement;5. Managed Entry Agreements with outcome-based arrangements could facilitate an early access;6. A glossary of terms to be used in the debate for better communication between stakeholders;At the project website (see below) an ‘infographic’ on the homepage summarises all the results of ADAPT SMART. Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far) The project aims to move beyond the state-of-the-art by identifying what is needed to address potential barriers to the implementation of MAPPs, and to address and align approaches/concerns from policy makers, regulators, HTA bodies, payers, patients and prescribers.This project is unique in that it is the most comprehensive attempt yet to involve all stakeholders in identifying relevant MAPPs activities, creating a knowledge repository and identifying scientific challenges and potential research/implementation activities that address unmet medical needs and create the right incentives for public and private actors to move beyond current pathways in the EU, and provide recommendations on how to implement MAPPs.The EMA pilots and ADAPT SMART raised the profile of the MAPPs concept and generated considerable interest and engagement from the wider scientific community, healthcare payers, consumer groups and non-governmental organisations (NGOs). The tools and methods available to make MAPPs happen are acknowledged, however the acceptance of certain tools in facilitating decision making is still under debate. Especially the use of Real World Data (RWD) is questioned by some stakeholders, such as HTA bodies and payers. Managed Entry Agreements are not yet fully embraced, mainly due to the lack of a proper infrastructure for collecting data. One thing is sure however, the more active involvement of payer organisations in the discussion of the affordability of medicines is one of the important spin-offs of the project.The topic of early access and affordability for the health care system will continue to be the focus of activities beyond the project. Understanding and potentially addressing stakeholder concerns were at the heart of all our activities and will continue after the completion of the project. ADAPT-SMART logo
