The current regulatory environment for new medicines, with its formal procedures and emphasis on large-scale clinical trial data, is clearly designed to protect patients. However, it is not without its downside. The escalating cost of randomized clinical trials, often coupled with disappointing results, makes putting a new drug through regulatory approval an extremely high risk exercise. As a result, many potential new drugs are never put to the test, and for those that do make it onto the market the need to recover development costs means the market price is high.
It’s a problem that MAPPs (Medicines Adaptive Pathways to Patients) are designed to solve, and it’s one where Lygature already has a lot of key solutions knowledge and experience. Lygature director André Broekmans (pictured left), who has more than 25 years of regulatory experience in the pharmaceutical sector, was on the steering committee of TI Pharma’s Escher project – a public-private partnership initiative to develop a science-based approach to reforming Europe’s regulatory system in order to accelerate patient access to new drugs. Lygature program manager Pieter Stolk (pictured right) not only coordinated the Escher project, he also helped to transform its results into TI Pharma’s (now Lygature’s) Escher platform for regulatory innovation.
“In the Escher program, we addressed a number of regulatory topics from the regulatory science perspective – on the one hand looking at the development of new tools and methods for conducting clinical trials but also looking at how different research methods could be used to generate the evidence needed to inform regulatory decisions”, says Pieter Stolk. “In addition to engaging all the relevant stakeholders it trained more than 15 talented PhD students, many of whom have moved on to responsible positions in regulatory authorities and industry around Europe. It means there’s not only a community but also a real impetus to develop the necessary tools and methodologies to make MAPPs a reality.”
When the European Commission, through the Innovative Medicines Initiative (IMI), established the ADAPT SMART program (Accelerated Development of Appropriate Patient Therapies – a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes), Lygature was therefore well placed to take the lead in managing the program. ADAPT SMART is a coordinating and support action program in IMI2, aimed at developing an enabling platform for the coordination of European MAPPs activities.
MAPPs implementations do not attempt to tear down existing regulatory frameworks, but instead to work within them in a way that balances the needs of early patient access, public health and societal benefit. Subject to passing normal toxicity tests, drugs are authorized earlier than would normally be the case for use in patient groups with clearly defined safety and efficacy profiles. These patients are carefully monitored and the resultant evidence-based feedback is used to progressively adjust or extend the target population. With their emphasis on matching drugs to patients in order to maximize patient, healthcare system and societal benefit, MAPPs implementations require a high level of health technology assessment (HTA) together with a solid platform for collating, analyzing and reporting real-world data. They also require the end-to-end engagement of stakeholders – from drug research & development teams and licensing authorities to clinicians, patient organizations, hospitals, healthcare providers and HTA bodies. Within ADAPT SMART, Lygature will provide program management and an IT platform to gather, collate and analyze the necessary scientific evidence, and facilitate the necessary multi-stakeholder dialogue.
“To manage a consortium like ADAPT SMART and bring everyone on board, you need situational awareness of the policy requirements, the political environment, and the stakeholder challenges,” says Pieter.
However, as André Broekmans goes on to explain, different European countries have different models of healthcare provision and funding. As a result, the ADAPT SMART program will not attempt to create a one-size-fits-all solution for implementing MAPPs. Instead, it will generate a set of guidelines and best-practices that countries can refer to when developing solutions to meet their own specific requirements.
“Everybody has their own perspective on what adaptive pathways mean. For industry, it’s getting a safe and effective product to the market as quickly as possible. For the regulators, it’s maintaining the current high standards for approval in order to protect public health and address patient needs. For the HTA bodies, it’s gathering evidence about how effective a therapy is in clinical practice, especially compared to existing treatments, and for the payers it’s achieving a system that’s sustainable and affordable. The tensions occur where these different perspectives collide,” he says. “However, if we can collectively identify what’s needed to achieve a seamless pathway from regulatory approval, through HTA approval and approval by the payer, MAPPs could represent a real sea change in getting safe and effective therapies to the patients that need them in the shortest possible time.”
