This Q&A aims to address some frequently addressed queries around MAPPs.
MAPPs, broadly, are a multi-stakeholder approach to developing an “RCT-plus” to evaluating new medicines. In order for outcomes-based healthcare to succeed, we need to know the performance of a treatment, both at a population level and an individual level. We need to expand our toolbox, and RCT data needs to be complemented with more information, notably more real world information.
In order to harness these large volumes of real world data, we also need rapid-cycle analysis providing the ability to process this data, almost in real time, in order to update decisions and minimize realized harm. This information can be used by regulatory decision-makers to adapt regulatory labels, as well as by payers to adapt reimbursement decisions.
Many initiatives are working towards achieving these points which bring us closer to realizing the goal of an “RCT-plus” toolbox. These include the Innovative Medicines Initiative projects such as ADAPT SMART, and the European Medicines Agency’s adaptive pathways pilot. The hurdles standing in the way of successfully implementing outcomes-based medicine are both technical and political, and collaborative initiatives like ADAPT SMART work to address both types of challenges.
The purpose of ADAPT SMART is to create new tools and procedures, which currently do not exist, to evaluate new medicines and determine their effectiveness. These new processes will also include instruments agreed by all stakeholders that insure the withdrawal of ineffective therapies and those demonstrating uncertain benefit/risk profiles.
These new tools will:
• Facilitate Real World Evidence and remote monitoring to effectively evaluate the actual performance of a new therapy;
• Develop processes that allow for the continuous reevaluation of a drug’s performance, ideally in real time and in the real world;
• Implement procedures in collaboration with patients, national health systems and HTA’s to ensure that the benefit/risk of new medicines is fully understood by all participants of MAPPs.
Once a new therapy is approved by the European Medicines Agency (EMA) it then needs to be reimbursed at the member state level to provide access to patients. In this way, it is often taken 2-5 years longer for patients to receive needed medicines at the member state level, regardless of the speed at which EMA approves a therapy.
Research published by the Genetic Alliance UK showed that 38% of patients across Europe are aware of a medicine they need, but they are not able to access it in their healthcare system.
This distinction is often not understood by those working outside of the medical sector, and is one of the core problems that ADAPT SMART will attempt address.
ADAPT SMART will develop as part of its programme, as well as in partnership with other IMI projects such as BD4BO and GetReal, MAPPs methodologies to help solve this problem in partnership and collaboration with payers, patients, practitioners, and industry.