Moving MAPPs into Practice through ADAPT SMART’s Legacy – interview with Hans-Georg Eichler and Solange Rohou
Moving MAPPs into Practice through ADAPT SMART’s Legacy Now that the ADAPT SMART project is coming to a close, one of the project’s goals was to develop the frameworks and knowledge base that [...]
How will the normal timelines be impacted by MAPPs given there is no hard starting point? – interview with Stuart Faulkner
How will the normal timelines be impacted by MAPPs given there is no hard starting point? The Centre for the Advancement of Sustainable Medical Innovation (CASMI) is one of the world’s thought leaders [...]
Has ADAPT SMART been successful in changing the way we think about approving new therapies? – interview with Ad Schuurman
Has ADAPT SMART been successful in changing the way we think about approving new therapies? Ad Schuurman sits at the confluence of three key circles in European healthcare. His principal day job is [...]
Have Stakeholders Been More Involved at the Centre of Decision Making? – interview with Yann Le Cam and Mathieu Boudes
Have Stakeholders Been More Involved at the Centre of Decision Making? Yann Le Cam from Eurordis and Mathieu Boudes from the European Patients’ Forum, two of Europe’s leading patient representatives participating in ADAPT [...]
Evidence Generation Throughout the Life Cycle The National Institute for Health and Care Excellence (NICE) provides national guidance and advice to improve health and social care in England. NICE co-leads work package 1 [...]
Work-stream D3.09 has explored some general ethical and legal issues that may arise through the introduction of the MAPPs concept, the implications for each stakeholder – in particular, patients and healthcare professionals (HCP) – and any liability change or shift between stakeholders and ethical considerations of prescribing control.
One of the original thought leaders of the concept of Adaptive Pathways was the NEWDIGS initiative of MIT's Center for Biomedical Innovation. ADAPT SMART has had the pleasure of collaborating with NEWDIGS throughout. ADAPT SMART interviewed NEWDIGS Strategic Director Mark Trusheim about the interactive case study he will facilitate at the ADAPT SMART closing event, as well as some of their current research.
Within the ADAPTSMART consortium, the remit of work-stream D3.08 has been to explore general ethical and legal issues that may arise through the introduction of the MAPPs concept, the implications for each stakeholder - in particular, patients and healthcare professionals (HCP), and what recommendations could be made to mitigate against identified issues. Specific legal issues relating to intellectual property law are the subject of another ADAPTSMART work-stream and are not discussed here.
A broad review of the main legal areas governing medicinal product development, approval and market access, in the context of MAPPs, was undertaken - except any potential impact of MAPPs on Intellectual Property and Regulatory Exclusivity Rights that will be analysed by D3.06 working group, within Work Package 3. The two documents should be read and understood together.
Impact of adaptive licencing and adaptive access on Intellectual property and regulatory exclusivity right periods (D3.06)
A broad review of the rules governing IP and RER as they are today, and with respect to MAPPs, was undertaken in the context of the following 4 scenarios deemed likely to occur with MAPPs: (i) Stand-alone MA (i.e. single indication only); (ii) MA with subsequent indications with the same compound, i.e. indication 1, subsequently followed by indication 2; (iii) MA under exceptional circumstances or conditional approval that subsequently gets approved for ‘full’ MA; and (iv) MA (full, conditional, or exceptional MA) that subsequently is revoked / suspended from a regulatory perspective. Any direct legal impact of MAPPs were assessed along with an exploration of several perceptual issues pertaining to the incentives of adopting and using MAPPs from an IP and RDP perspective.
The objective of D2.07 was to provide an overview of which tools and systems are available at the national level to guide the appropriate use of medicinal products in EU Member States. This report looks at all tools/systems for guiding appropriate use at the national level, even if these may not be directly applicable in the context of a MAPPs-type pathway.
Decision points in current vs. future processes by stakeholder groups, and resource implications for a roadmap for implementation (D3.02)
The introduction of a new concept introduces new dynamics to which the involved stakeholders need to adapt. When considering the feasibility and practicalities of being involved in an adaptive pathway to medicines – which includes multi-stakeholder interactions, iterative cycles of development and assessment, and long term real-world data (RWD) collection – each stakeholder must have a clear understanding of their remit (current or new), the type of input requested from them (advice or decisions), and the appropriate resources needed.
Podcast: “The Regulatory Needs of MAPPs – What are the Evidence Requirements to Make Adaptive Pathways Work in Practice?”.
On 25 October 2017, ADAPT SMART had a discussion with François Meyer, Advisor to the President & International Affairs at the French National Authority for Health (HAS), Solange Corriol-Rohou, Senior Director Global Regulatory Affairs & Policy, Europe at AstraZeneca Global Medicines Development, Elin Haf Davies, CEO of Aparito and Stefan Lange, Deputy Director at IQWiG, moderated by Richard Barker, Director, CASMI.
Webinar Report: “Implementing Real World Data to Make Adaptive Pathways Work” On 18 October 2017, the ADAPT SMART consortium presented a webinar hosted by the Estonian Ministry of Social Affairs. The webinar was the [...]
EMA answers to questions from industry partners in the ADAPT SMART consortium regarding the EMA Adaptive Pathways pilot project
EMA answers to questions from industry partners in the ADAPT SMART consortium regarding the EMA Adaptive Pathways pilot project Response: Adaptive Pathways is a concept that seeks to foster access to beneficial treatments [...]
On April 19th, 2017, the ADAPT SMART Consortia, comprising over 30 international partners including regulators, patients, academia and industry, held its first public webinar to give an update on the project’s progress and milestones, as well as its efforts to incorporate the feedback of key stakeholders groups during the calendar year of 2017.
This report proposes a framework with pictographic representation of an adaptive pathway containing key moments, events and involved stakeholders, in each phase. This model is purposefully presented rather simplistically in this first evolution, acknowledging that there remain several questions to be answered – both unique to each individual potential medicine as well as to full implementation of an integrated adaptive pathway approach.
D1.02 aimed to analyse and monitor IMI and non IMI project outputs, and to perform a gap analysis of the wealth of evidence generation in the context of MAPPs. In other words, tools and methods developed by IMI and other EU projects, which could benefit MAPPs have to be identified. To this effect, a two-phase process was identified.
By Neena Brizmohun
The “glass is half full,” the European Medicines Agency’s senior medical officer says of efforts to get everyone onboard the adaptive pathways approach for accelerating the approval of drugs for high unmet needs.
By Neena Brizmohun
Payers often worry about where they would stand if a drug that was approved in the context of adaptive pathways should subsequently underperform. But they should not be concerned, according to the latest discussions on this relatively new concept for getting drugs to market faster.
Putting Patients at the Centre – The Ethical and Legal Considerations of MAPPs By Paul Robinson of Merck Sharp & Dohme One of the key factors in the future success of any MAPPs implementation will be the need for continuous monitoring, in health data registries, of how patients respond to new therapies. On January 17th, 2017, the ADAPT SMART consortia hosted a multi-stakeholder workshop to explore the ethical and legal considerations of MAPPs and the uncertainties of patients. The workshop covered several topics including the use of patient data in registries.
ADAPT SMART’s General Assembly, held at the European Medicines Agency (EMA), agrees to focus its final year on incorporating stakeholder feedback into the development of Medicines Adaptive Pathways to Patients (MAPPs) tools and methodologies.
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.
On December 14th, IMI’s ADAPT SMART project heads and WP1-2-3 work package leads hosted an internal year-end webinar, updating on the project’s status and next steps for members of the consortium. Some 70 consortium members were guided through the project’s delivered first-year objectives and explained what will occur over next 12 months, until the end of ADAPT-SMART in December 2017.
Report by Charles River Associates: use of managed entry agreements for products with a conditional or exceptional authorisations
The European Federation of Pharmaceutical Industries and Associations (EFPIA) asked Charles River Associates (CRA) to undertake an interview programme to understand how managed entry agreements (MEA) have been used for products with Conditional Marketing Authorisations or Market Authorisations under exceptional circumstances as a proxy for Medicines Adaptive Pathways to Patients (MAPPs).
An issue of divergent views became apparent early in the course of the ADAPT SMART project, namely; to what kind of novel medicines and clinical scenarios/conditions should a MAPPs approach be applied? The ADAPT SMART consortium convened discussion fora with stakeholders to elaborate MAPPs engagement criteria that may be acceptable to all concerned within the existing legislation. In this paper, we summarize viewpoints from these discussions.
On the 29th of February, ADAPT SMART hosted an invitation-only multi-stakeholder workshop around selection criteria for MAPPs in Amsterdam, The Netherlands.
On 6 September, Nicola Bedlington from The European Patients Forum (EPF) and Yann Le Cam from Eurordis, two of Europe’s leading patient representatives, discussed the need for Adaptive Pathways (MAPPs) in Europe.
ADAPT SMART welcomes the European Medicines Agency (EMA) report on its adaptive pathways pilot, considering this as a positive step in the current multi-stakeholder discussions about early access to medicines via a Medicines Adaptive Pathways to Patients in the EU.
On 5 July, key stakeholders involved in the MAPPs conversation met to discuss the tools needed to define endpoints and success measures in MAPPs. Participants included Richard Barker, founding director, Centre for the Advancement of Sustainable Medical Innovation (CASMI); Claudine Sapede-Kniffel, Global HTA & Payment Policy Lead, F. Hoffmann-La Roche; Anja Schiel, Senior Advisor and Statistian, NOMA (The Norwegian Medicines Agency).
There is much debate about the need to allow patients with serious diseases early access to promising new therapies. Some concerned stakeholders argue for “the right to try any treatment option for every patient in need” while others oppose this position, saying “only thoroughly studied treatments should be allowed on the market - we need to take our time”.
On the 29th of February, ADAPT SMART hosted an invitation-only multi-stakeholder workshop around selection criteria for MAPPs in Amsterdam, The Netherlands. The programme from the event can be found here. A full report of the event [...]
On the 29th of February, ADAPT SMART hosted an invitation-only multi-stakeholder workshop around selection criteria for MAPPs in Amsterdam, The Netherlands. Angelika Joos of MSD and Mathieu Boudes of EURORDIS attended the workshop in their capacity [...]
On 7 April, key participants associated with the IMI ADAPT SMART project met to discuss the consortia's progress in finding ways to implement MAPPs in Europe. Participants included: Hans-Georg Eichler, Senior Medical Officer at the European Medicines Agency (EMA); Luk Maes, Executive Director, Regulatory Scientific Policies – Europe, for Bristol-Myers Squibb; Ad Schuurman, Head of the Business Contact Centre & International Affairs National Health Care Institute of the Netherlands (ZIN); and Sarah Garner Associate Science Policy and Research, National Institute of Health and Care Excellence (NICE).
Podcast on Managed Entry: Claudine Sapede-Kniffel of F. Hoffmann-La Roche and Jacoline Bouvy of NICE
On 17th February, Jacoline Bouvy of NICE and Claudine Sapede of F. Hoffmann-La Roche met in London to discuss managed entry in the context of the ADAPT SMART initiative.
On 8th January 2016, the ADAPT SMART work package leads participated in a webinar to share status updates, and compare notes on progress made and hurdles to overcome. ADAPT SMART Consortia members were invited to participate in the webinar, and were invited to ask questions via live chat after the work package leads finished their presentations.
In December 2015, ADAPT SMART work package leads André Broekmans and Luk Maes met to discuss the ambitious objectives of ADAPT SMART, the impact of the diverse participants involved in the project, and what sets this initiative apart from other programmes focussed on MAPPs.
With the first few months of the ADAPT SMART project underway, coordinator André Broekmans (Escher Platform, TI Pharma) shares his views on what is needed to continue to drive the project forward.
On 27th October, thought leaders in the field of adaptive pathways met in Washington D.C. to discuss the global objectives of IMI ADAPT SMART and what the landmark project hopes to achieve.
On Friday September 4th, 2015, a coalition of 90 European healthcare stakeholders from 32 organisations including patients, payers, regulators, HTAs, academic institutions, and industry took their first steps towards investigating MAPPs tools and methodologies, and [...]
Press Release: Innovative Medicines Initiative Launches ‘ADAPT SMART’, an Adaptive Pathways Project with 32 International Participants
The project will establish a platform that enables the coordination of Medicines Adaptive Pathways to Patients (MAPPs) related activities within the Innovative Medicines Initiative (IMI) and engages in dialogue with all relevant stakeholders. MAPPs seek to foster access to beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span in a sustainable fashion. The outcomes of the ADAPT SMART consortium will help inform future research agendas and discuss approaches for the implementation of MAPPs.
The adaptive pathways approach (formerly known as ‘adaptive licensing’) is part of the European Medicines Agency’s (EMA) efforts to improve timely access for patients to new medicines.
The Innovative Medicines Initiative (IMI) yesterday launched its 5th Call for proposals under the IMI 2 programme.
Interview with Sarah Garner of NICE: IMI’s GetReal – Using real world data for decision-making in R&D and health technology assessment
In this interview, Professor Sarah Garner, Associate Director for Science Policy and Research at NICE, speaks about The GetReal project is another IMI initiative addressing MAPPs. Real world evidence holds the potential to both increase efficiency in R&D and to bridge the gap between the proof of efficacy required to pass regulatory scrutiny and the demonstration of likely effectiveness needed to satisfy health technology assessment bodies.
The inaugural meeting of the NEWDIGS Data Program was held in January 2015, and brought together international experts from academia, government, information technology, and the healthcare sector. A series of panel discussions focused on identifying the gaps and opportunities that might be addressed through better international cooperation in data system design and use.