Europe faces many challenges in bringing new medicines to patients in an efficient and timely manner. The reasons for this include the high attrition rates in medicine development, a regulatory environment lagging behind rapidly evolving science, and research and development (R&D) and price/reimbursement models that are no longer financially viable.
Medicines adaptive pathways to patients (MAPPs) refers to a concept that seeks to provide patients with timely access to beneficial medicines, starting from a small group of well-identified patients (e.g. those with no alternative treatments). As evidence accumulates on the benefits and risks of a medicine, access may be extended to other groups of patients. MAPPs relate to a flexible pathway covering the entire life cycle of a medicine, from development, through licensing, to patient access (pricing/reimbursement and healthcare delivery).
By bringing together representatives from key European stakeholder groups – regulatory agencies, health technology assessment (HTA) bodies, pharmaceutical companies, payers, patients and health care professionals – ADAPT-SMART created a platform where the conditions and feasibility of MAPPs implementation within the EU regulatory/legal context could be discussed openly. As a result, the project contributed to an improved understanding among stakeholders of the issues promoting early access of medicinal products to patients.
Embracing the lifespan approach
Thanks to ADAPT-SMART, there is now a wide acceptance among different stakeholders that medicines development should take the lifespan approach. Whereas in the past the prevailing idea was that a drug should be developed for 5 to 10 years, then launched and left out there for patients to use, this project reinforced the idea of continuous evaluation and use. The project came up with recommendations on how the system could be improved at the various decision points in the product lifespan, including before and after licencing. This means that patients who are running out of time could get access to new drugs more quickly, and that evaluation continues even after the product launch.
Other top project outputs include:
- an overview of conditions which need to be met for entering the MAPPs/adaptive pathway;
- a definition of the building blocks for early access, appropriate use and the continuous evaluation of medicines;
- a model for a seamless pathway for early access and continuous evaluations;
- a recognition that there are no legal constraints in implementing an adaptive pathway approach for medicines development, approval and reimbursement;
- a glossary of terms to be used in the debate of adaptive pathways and early access;
- recommendations for potential future IMI research projects which will be important in progressing the implementation of MAPPs.
For the benefit of patients, industry and regulators
Industry has learned the value of interacting with regulators, HTA bodies and patient organisations throughout the drug development process. Different industry partners have also learned that it is possible to work with each other constructively in the pre-competitive space.
Regulators such as the European Medicines Agency also benefitted from their interaction with different stakeholders, especially with HTA bodies and payers. Among other things, having a debate with these downstream decision makers could help them avoid situations where they authorise products which are later rejected for pricing and reimbursement, and never reach patients.
ADAPT-SMART also involved two patient organisations as partners in the project. They helped to set the project goals and get results that are meaningful for patients. Furthermore, the recommendations that the project brought forward acknowledge the important role patients should play in the development, approval, reimbursement and appropriate use of medicines.
The most important results of the project have been published in scientific journals. Additionally, the project published a comprehensive online infographic to help stakeholders to access all the relevant documents. Finally, recommendations and conclusions brought forward by ADAPT-SMART will continue to be debated by different groups of stakeholders, and developed by different groups of researchers, including those involved in future IMI projects.