ADAPT SMART Webinar Report – Putting Patients at the Centre
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.
The ADAPT SMART consortium (a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes) enables coordination of activities, supporting investigation of methodologies and dialogue to develop workable concepts for Medicines Adaptive Pathways to Patients, or MAPPs. According to the Journal The Pink Sheet, “Adaptive pathways would enable a drug to be approved earlier based on limited data for restricted populations where its benefit-risk balance could be favorable, and then real-world data would be used to support the product’s wider use. Discussions with HTA bodies, patients and other downstream stakeholders would have to take place early on during drug development so as to make sure their demands were also met.”
Whilst the MAPPs concept is being embraced internationally in Japan, Canada, and in the recent congressional testimony of newly appointed FDA Director Scott Gottlieb in the United States, in Europe, ADAPT SMART has seen both enthusiastic support as well as criticisms from payers and stakeholder groups that MAPPs will lower the safety bar for patients.
During the webinar, Dr Hans Georg Eichler, Senior Medical Officer at the European Medicines Agency responded, “the benefit-risk of a drug that we allow [in MAPPs] must be positive for the treatment-eligible population. This population may be smaller than for conventional development pathways, enabling smaller, faster studies that provide an earlier basis to conclude a positive BR.”
The webinar reserved 30 minutes for audience questions and many of the questions revolved around patient engagement and access in the project.
Christoph Thalheim of the European MS Platform asked, “who ultimately decides what makes a major difference to patients’ life? Is regular patient involvement foreseen in this decision [in MAPPs]?” Jacoline Bouvy of Nice responded, “Yes, absolutely. I don’t think that’s a question that can be answered suitably without patient engagement.” Andre Broekmans added, “We have the European Patient Form and Eurordis involved in the project, and they have an important impact on the project as to how patients should be involved and engaged in decision making.”
Jacqueline Bowman, Principal of Third-I, asked, “How do you ensure the involvement of patients is meaningful and how will you measure this”? Andre Broekmans of Lygature, and co-lead of Work Package 2, pointed out that, “The patient’s role is crucial in adaptive models…the patients will be sitting at the table during the decision models and will be an active part of the process.”
Paul Robinson of Merck, Sharp and Dohme asked, “With reimbursement and control of prescribing a national competency, is there a risk that MAPPs will make some medicines available to patients but not to others”? Hans Georg Eichler responded that, “We think MAPPs will help harmonize the different assessments in Europe. We’re hoping to get different people around the table from different EU member states, early on, and we think this process will help drive harmonization in the future.”
You can find the full archive of the webinar here, including downloads of the presentations that were made.
The ADAPT SMART Coordination and Support Action (CSA) acts as a neutral collaborative framework to establish the platform that will engage with all relevant stakeholders, including patients, industry, SMEs (Small and Medium sized Enterprises), regulators, health technology assessment bodies (HTAs), payers (national and European Networks), clinicians, governments/policy makers (national authorities as well as European Commission’s DG SANTE and DG GROW, and European Networks). In addition to engaging in a dialogue with relevant stakeholders, the ADAPT-SMART consortium aims to align understanding of the impact of MAPPs, to share findings between all stakeholders, and to allow the field to actively work towards MAPPs implementation.
Other questions asked during the webinar
Chen Inhua, “Given the heterogeneity of the assessment methods used by various member states in assessing value of a product, can the panel comment on how divergent these outcomes are across EU and how they relate to gaps identified within the MAPPs pathway?”
Answer: Do we hope MAPPs can help develop a more harmonized assessment across Europe? The answer is yes. MAPPs will help harmonize the different assessments in Europe for areas of unmet medical need and ultimately could help unify the access to therapies across the various EU member states.
Duane Schulthess: “MAPPs will be focused on transformative products, how will those therapies be selected and that criteria defined?”
Answer: A transformative product is more than simply a product that addresses an unmet medical need as there would also need to be a reasonable expectation of a substantial medical benefit from the new therapy. MAPPs will focus on products that can make a substantial positive impact on patients’ lives, and have a high probability of a positive net gain in quality of life while balancing benefit and risk.