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.

A therapy going through MAPPs will have demonstrated a level of efficacy, safety, and toxicity in Phase I and/or II that warrants and justifies the use of an adaptive pathway. Any therapy approved for MAPPs will be accompanied by a real world evidence package that will monitor the actual performance of the new therapy over the entire lifecycle of its use in many types of patients. This represents an increase in the level of evidence over current regulatory pathways. MAPPS will provide continuous risk/benefit information for better follow-on coverage decisions at the member state level, and give a more accurate safety profile to regulators, payers, and patients.

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.

Recent research published in the Journal of the American Medical Association (JAMA) investigated the effectiveness of oncology drugs released on the basis of a surrogate endpoint – a very challenging way to prove a drug actually works. The JAMA study showed that roughly 20% of these new therapies provided a “proven overall survival benefit.” ADAPT SMART will develop the tools to bring these medicines to patients earlier under MAPPs; it will facilitate the development of processes identification of responders and non-responders more quickly; and it would allow for the more effective analysis of the performance of new therapies in the real world and the removal of those that don’t provide a benefit to patients in partnership with patients, payers, regulators, industry, and practitioners.

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.

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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.

The application of MAPPs will allow for pharmacovigilance to occur simultaneously with evidence generation for effectiveness, with the use of real world evidence at the launch of the MAPPs process. Rather than the current scenario where therapies are systematically reassessed only after five years of marketing, evidence of how the new therapy is performing will be obtained from day one and as long as the product is licensed over its entire lifecycle as a licensed treatment.