The NRDD article, co-authored by Lygature’s André Broekmans (Director Escher), describes the evolution of the adaptive approach and of concepts developed by ADAPT SMART, a multi-stakeholder consortium funded by the European Innovative Medicines Initiative. The consortium (which completed its work in 2018), provided a platform to discuss bottlenecks and opportunities for making MAPPs a reality with a wider range of stakeholders, published analyses and offered advice on future research priorities.
The outcomes of ADAPT SMART do not propose a new regulatory pathway or legal framework, but a combination of ‘building blocks’, each of which are discussed in detail in the NRDD article:
- A focus on speedy access for patient (sub-)populations in urgent need
- Rapid increase of the evidence-to-uncertainty ratio
- Use of the full spectrum of study methods for evidence generation
- Adaptive pricing and reimbursement
- A focus on appropriate use
- Collaboration of all stakeholders
On a more practical note, the ADAPT SMART team also submitted an article to Clinical Pharmacology & Therapeutics. In this article, the authors summarise the MAPPs engagement process and critical questions to be asked at each milestone of the product life-span. The article was accepted for publication last month.
Are you interested in learning more? Both articles are available, open access, on the publishers’ websites:
- The evolution of adaptiveness: balancing speed and evidence, Nature Reviews Drug Discovery
- Medicines Adaptive Pathways to Patients: Why, when and how to engage?, Clinical Pharmacology & Therapeutics