Here are some articles from the industries leading online publications.
On 28 October 2016, the European Medicines Agency (EMA) will hold the EMA registries workshop regarding the use of registries in real world evidence. In advance of this discussion, Emma Du Four, Senior Director Regulatory Policy & Intelligence at AbbVie, and Chris Chinn, Head of Real World Data Strategy and Partnerships at Sanofi, (Co-Chairs of the EFPIA-EBE-VE Real World Evidence-Registries Community), outline the key principles that need to be fulfilled for registries to contribute to the success of real world evidence.
On 29 June 2016, key stakeholders and decision-makers in the field of real-world evidence met to take part in the DIA 2016 seminar “Europe and the US: Making Outcomes-Based Health Care Possible”. As Europe and the US continue to examine ways to develop therapies on the basis of their “real world” performances, the session aimed to discuss benefits and challenges of outcomes-based health care, as well as the remaining barriers to implementation.
This 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.
As the keynote speaker in the EMIF Conferences, E-managing the Future of Health Data, Dr Sarah Garner spoke on a number of initiatives making use of real world evidence—specifically highlighting the significance in RWE in furthering the conversation on MAPPs. Here, she reflects on how the opportunities changes in technology allow us today and how initiatives like ADAPT SMART are paving the way for MAPPs.
When it comes to rare diseases, it’s important to find the fastest path and look beyond traditional trial models to meet patients’ needs, says Marshall L. Summar, MD, co-chair of the rare disease advisory panel of Patient-Centered Outcomes Research Institute (PCORI) and the Scientific Advisory Committee of the National Organization for Rare Diseases (NORD). He is one of the world’s leading authorities on accelerated pathways in orphan diseases and the uses of Real World Evidence in pediatric clinical research.
Last week, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted the Priority Medicines (PRIME) scheme. PRIME is a welcome step towards connecting healthcare decision-makers to expedite patient access to new medicines – but what sets it apart from other mechanisms with similar aims? The Conditional Marketing Authorisation (CMA) and Accelerated Assessment (AA) texts were also adopted by CHMP last week. With the PRIME text, as well as CMA and AA, to be published in March 2016, it is important to discuss what sets PRIME apart – and what elements are needed to make PRIME a success, not only in principle, but also in practice. In an article for EFPIA, Susan Forda, Vice President, International Regulatory Affairs Eli Lilly and Company, examines the potential of PRIME.
After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014. Although it is a significant achievement to see the first asset being placed into human trials under an adaptive pathway, there is much to be learned regarding the multinational and multi-stakeholder effort that has driven the growing acceptance of MAPPs as a methodology and concept, as well as the need for continued and increasing international collaboration to foster the wider adoption of MAPPs. Changes in available science and technology, as well as a number of challenges in the current system, outlined in this paper, are transforming approaches to medicines development and approval. It is these challenges that have led directly to the groundbreaking MAPPs collaboration between the Massachusetts Institute of Technology Center for Biomedical Innovation’s New Drug Development Paradigms Initiative, the EMA, patient, payer and health technology assessment groups, the European Federation of Pharmaceutical Industries and Associations, and the Innovative Medicines Initiative—a European public-private partnership. This article examines the development of MAPPs, from inception of the concept, to the establishment of this trans-Atlantic initiative, and examines challenges for the future.
The concept of adaptive licensing (AL) has met with considerable interest. Yet some remain skeptical about its feasibility. Others argue that the focus and name of AL should be broadened. Against this background of ongoing debate, we examine the environmental changes that will likely make adaptive pathways the preferred approach in the future. The key drivers include: growing patient demand for timely access to promising therapies, emerging science leading to fragmentation of treatment populations, rising payer influence on product accessibility, and pressure on pharma/investors to ensure sustainability of drug development. We also discuss a number of environmental changes that will enable an adaptive paradigm. A life-span approach to bringing innovation to patients is expected to help address the perceived access vs. evi- dence trade-off, help de-risk drug development, and lead to better outcomes for patients.
The UK government’s Accelerated Access Review is planning to bring big and meaningful changes to the way drugs are developed and brought to UK patients and is not just a tweak to the system.
The recent launch of a pilot scheme for ‘adaptive licensing’ by the European Medicines Agency could spell a new era in drug development. Rather than focusing on the purely regulatory aspect of the system, the proposals challenge several aspects of existing development processes, including the conventional roles of sponsor, regulatory and reimbursement agencies, payers and patients. They also exploit a number of scientific advances not available when the existing development path was laid out. Like all other innovations the new model of adaptive development also carries significant challenges, in areas as diverse as clinical management, economics, intellectual property, ethics and public communications. This paper outlines these changes to thinking, summarizes the benefits the model offers and proposes how some of the challenges should be met. The perspectives in the paper are drawn from the authors’ own involvement with this European project and are informed by discussions at several workshops and conferences held over recent months.
From 27th-28th October, international thought leaders in new healthcare models met in Washington D.C. to discuss means of collaborating to create better patient outcomes in the emerging world of precision medicine. The two-day conference included a series of moderated panel discussions, participation from the audience, and live participation from the public via Twitter. EFPIA was a conference supporter, and EFPIA MAPPs was represented in the conference by Sabine Atzor, Director Regulatory Affairs, part time seconded from F.Hoffmann-La Roche to EFPIA.
On 30th September, thought leaders in the field of personalised medicine from Europe, the United Kingdom and the United States met in Bad Gastein, Austria to discuss how Medicine’s Adaptive Pathways to Patients (MAPPs) and breakthrough designation will impact patients. In a moderated discussion with the experts, the audience and live participation from the public via Twitter, the panel examined the potential long-term implications of these new models for personalised medicines, and what their adoption means for regulators, payers, patients and society as a whole.Interview with Sabine Atzor: Examining the Building Blocks of MAPPs
To truly understand Medicines Adaptive Pathways to Patients (MAPPs) from a regulatory perspective, it’s essential that we take a closer look at the building blocks – both old and new – that are paving the way for its implementation, says Sabine Atzor, Director Regulatory Affairs, part-time seconded from F.Hoffmann-La Roche to EFPIA. Among these are Conditional Marketing Authorisation and Accelerated Assessment guidelines, as well as the European Medicines Agency’s (EMA) recently announced PRIME (Priority Medicines) scheme.Interview: US Personalized Medicine Coalition – Exploring Pathways Towards Patient-Centred Healthcare
Adaptive pathways are among the possibilities being examined for incentivising medicine and future innovation in the United States, says Amy M. Miller, Ph.D., Executive Vice President of the Washington DC-based Personalized Medicine Coalition. But some key hurdles remain, not only from a scientific view, but also from a policy perspective. Dialogue and an exchange of ideas between the US and the EU can help spur progress.MAPPs and New Benefit Risk Models: IMI2 Leads with Patient Engagement
by Magda Chlebus
The Innovative Medicines Initiative (IMI) yesterday launched its 5th Call for proposals under the IMI 2 programme (http://www.imi.europa.eu/content/stage-1-16). Topic 1 addresses one of the key enablers of adaptive pathways – developing methods to incorporate the input of patients into the assessments of benefits and risks of new medicines at all stages of the drug development life cycle.Event Report and Video – MAPPs: Saving Time, Saving Lives
While progress is often slow and laborious when trying to gain traction for new ideas in the ‘Brussels Bubble’, there was much to be optimistic about in the Medicine’s Adaptive Pathways to Patients (MAPPs) meeting held on Thursday June 4 in Luxembourg. Organised as part of EFPIA’s general assembly, a broad cross section of stakeholders attended the MAPPs session to hear what had been accomplished over the last twelve months and discover how the initiative is being moved forward in partnership with the second Innovative Medicines Initiative (IMI2) and the European Medicine’s Agency (EMA).
A full event report, including video, is available here.
Presentations from the event can be downloaded here.
- Hans-Georg Eichler, Senior Medical Officer, European Medicines Agency (EMA)
- Karin Van Baelen, Head Global Regulatory Affairs at Janssen, J&J
- Luk Maes, Executive Director Regulatory Europe, Bristol-Myers Squibb
- James Anderson, Head of Corporate Government Affairs, GlaxoSmithKline, UK
- Sue Forda, VP-Global Regulatory Affairs – International, Eli Lilly
- Nathalie Seigneuret, Senior Scientific Project Manager, Innovative Medicines Initiative
- Sarah Garner, Associate Director Science Policy and Research, NICE
- Jeremy Haigh, EFPIA, moderator
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, says Professor Sarah Garner, Associate Director for Science Policy and Research at NICE.New MAPPs Group Launches on LinkedIn
A new MAPPs group has been launched on LinkedIn. Join the group to get the latest updates on MAPPs, and to take part in the conversation.Paper: “Partnering With Patients in the Development and Lifecycle of Medicines – A Call for Action”
Therapeutic Innovation & Regulatory Science – the official journal of DIA
Excerpt: “Progress toward a universal framework for patient involvement requires a joint, precompetitive, and international approach by all stakeholders, working in true partnership to consolidate outputs from existing initiatives, identify gaps, and develop a comprehensive framework. It is essential that all stakeholders participate to drive adoption and implementation of the framework and to ensure that patients and their needs are embedded at the heart of medicines development and lifecycle management.”Interview with Top Drug Regulator: Adaptive Licensing is a “New Paradigm” – for Everyone
by Gary Finnegan
Reinventing how medicines come to market demands new thinking from regulators, payors, patients and industry, says Professor Luca Pani, Director General of the Italian Medicines Agency.‘Patients need medicines – not to die in safety’: Interview with Bettina Ryll, Founder, Melanoma Patient Network Europe
by Gary Finnegan
Her husband’s diagnosis with melanoma led biomedical scientist Bettina Ryll to change how she thinks about drug development and riskKUL Survey Adaptive Pathways Survey
Please take the KU Leuvent survey on Adaptive Pathways – your opinion counts! Completing this questionnaire will only take 10 minutes of your time.Commission Expert Group on Safe and Timely Access to Medicines for Patients (“STAMP”)
European Commission Website
The first Meeting of the STAMP Expert Group took place on 27 January 2015. The STAMP expert group is set up to provide advice and expertise to the Commission services in relation to the implementation of the EU Pharmaceutical legislation, as well as programmes and policies in this field. The STAMP will exchange views and information about the experience of Member States, examine national initiatives and identify ways to use more effectively the existing EU regulatory tools with the aim to further improve safe and timely access and availability of medicines for patients. Expert presentations and information about the scope and objectives of STAMP can be found on the European Commission’s website.Fast-tracking medicines innovation: a question of uncertainties
Excerpt: “Among the most challenging questions raised by earlier launch are how to ensure that faster does not mean riskier, how far existing legislation can allow for it, how soon adequate structures can be put in place to capture and analyse data gathered after product launch, how current conflicts between data privacy and data access may be resolved, how to handle the risk that early disclosure of an innovation could erode its data exclusivity period, and how to link medicines authorisation with authorisation of the accompanying diagnostics that targeted therapies depend on.”Building on our own potential: A UK pathway for regenerative medicine – A report from the Regenerative Medicine Expert Group
UK Government Website
Excerpt: “The Expert Group recommends that the developers of regenerative medicines give serious consideration to seeking marketing authorisation through the adaptive licensing pilot scheme where appropriate.”Event Report: MIT’s NEWDIGS Launches New Program in Healthcare Data
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. Through discussion, participants joined in formulating a research agenda with associated pilot programs to develop the tools, methods and policies needed to leverage real world evidence for better decision-making.Behind the scenes of medicines regulation
The Pharmaceutical Journal
Sir Michael Rawlins talks about how the MHRA intends to speed up approval of innovative medicines, involve pharmacists in reclassification and address drug shortages and trade in counterfeit medicines. Excerpt: “…the Adaptive Pathways Pilot is encouraging greater dialogue between regulators, industry, health professionals and other stakeholders at an earlier stage of drug development.”Regulators streamline drug approvals
Excerpt: Hans-Georg Eichler, senior medical officer at the EMA, says pressure for fast-track processes is coming from patients as well as industry.“Patients are saying: ‘Why do we have to wait years for this treatment? I’m expected to die next year; I don’t care what drugs will come to market in five years’ time’. That line of argument is coming to Europe and we are listening and responding.” Adaptive licensing aims to find a balance between the competing demands of speed and safety in approving drugs, says Dr Eichler.Conference Report: Better Science, Better Health: New Trial Pathways and Better Patient Data
A report of an international conference held at the Royal College of Physicians, London, 21 October 2014Accelerated Access to Innovative Medicines for Patients in Need
Clinical Pharmacology & Therapeutics advance online publication 13 August 2014; doi: 10.1038/clpt.2014.145, L G Baird, R Banken, H-G Eichler, F B Kristensen, D K Lee, J C W Lim, R Lim, C Longson, E Pezalla, T Salmonson, D Samaha, S Tunis, J Woodcock and G Hirsch.Better Clinical Trial Designs Could Lower Development Costs And Yield More New Drugs
Forbes Pfizer, Roche join consortia of adaptive trial sponsors
Outsourcing-Pharma FDA simplifies approval process for innovative devices lacking a predecessor
FierceMedicalDevices Pfizer hands in an early application for blockbuster contender
FierceBiotech ALTTO Fallout Focuses Debate on the Meaning of pCR
Medscape Colorado first state to Pass “Right to Try”