New Project to Improve Timely Access to MedicinesСентябрь 2015
EURORDIS is partnering in the recently launched ADAPT SMART* project, which will establish a platform that enables the coordination of Medicines Adaptive Pathways to Patients (MAPPs) activities.
MAPPs can help to ensure access to new treatments for patients with unmet medical needs, such as rare diseases, at the earliest appropriate time in the entire life cycle of a new treatment (from development of a treatment during clinical trials, to licensing, to patient access to the medicine, which involves health-technology-assessment (HTA) bodies, as well as payers and pharmaceutical companies).
The ADAPT SMART project will engage all relevant stakeholders in dialogue with the aim of investigating MAPPs tools/ methodologies to develop workable MAPPs concepts. The work of ADAPT SMART focuses on wide concepts aiming to identify opportunities that could be leveraged and obstacles that need to be addressed to enable MAPPs in the future.
ADAPT SMART is a 30-month project that started in July 2015 under IMI2, which is the second phase of the Innovative Medicines Initiative (IMI) under the EU Horizon 2020 framework. IMI2 is a public-private partnership of the European Federation of Pharmaceutical Industries and Associations (EFPIA) and the European Commission.
The ADAPT SMART Consortium, led by the European Medicines Agency, is made up of stakeholders including patient groups, regulators, HTA bodies, payers, researchers, academics and companies. Dialogue between such a range of partners is crucial to ensure that all relevant stakeholders are aligned on how to effectively develop new medicines. The challenge for ADAPT SMART is to develop a MAPPs model that aligns the needs of all these stakeholders and to ensure that this model is sustainable.
EURORDIS will bring the rare disease patient perspective to this project; the rare disease community faces great challenges in the development of medicines due to small patient populations and rarity of patients, experts and knowledge, making necessary adaptive and innovative approaches to medicines development.
What are Medicines Adaptive Pathways to Patients?
Patients are demanding faster access to new medicines. The concept of Medicines Adaptive Pathways to Patients (MAPPs) reflects the need for a more flexible, adaptive approach to the medicines development pathway.
In the current access system, for both marketing authorisation and reimbursement of a medicine, a «yes or no» decision takes place after completing a series of research experiments that can take as long as 10 years.
MAPPs refer to flexible development and access pathways within the current regulatory framework that aim to ensure that medicines can reach patients at an earlier stage than they do in the current system. They start with an early authorisation of a product focused on a well-defined and targeted population (such as a rare disease patient population) for who benefits are likely to outweigh risks. The target population is adjusted as evidence on the safety and efficacy of the medicine expands.
MAPPs may then integrate adaptive clinical trial design, patient-centric benefit/risk assessments and continuous re-evaluation as new evidence becomes available throughout the entire life cycle of a medicine.
* ADAPT SMART = Accelerated Development of Appropriate Patient Therapies: A Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes.
This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 115890. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.
This article reflects EURORDIS’ view and that neither IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained therein.
Eva Bearryman, Junior Communications Manager, EURORDIS