In today’s blog post we talk to Mark H Mayer, Senior Advisor–Global Regulatory Policy and Intelligence, Eli Lilly and Company.
What’s an adaptive pathway?
“Adapt or perish” is a quote generally attributed to H.G. Wells, a British writer best known for his science fiction novels of The War of the Worlds, The Invisible Man and The Time Machine. Regardless of the context, I believe that the underlying message holds true; we must continue to explore new and better ways of doing things, because complacency can lead to insufficiency. In this case, ‘insufficiency’ could mean less efficient approaches for medicine development, suboptimal stakeholder engagement along the way, or even unnecessary delays in the availability of new medicines for patients. This is where the role of adaptive pathways comes in, to help speed up the process of earlier access for patients with unmet medical needs to a new and innovative medicine.
How can the concept benefit patients?
For patients who have serious, life-threatening diseases with no treatments yet available, having to wait for a medicine can have serious consequences. Therefore, there is a growing sense of urgency from patients to access promising therapies as early as a positive benefit-risk balance is sufficiently established. At this stage, an important question remains to establish the full therapeutic value of the medicine. Adaptive pathways give patients access to new and urgently needed treatments at the earliest appropriate opportunity by introducing earlier dialogue and exchange of information between pharmaceutical companies and decision makers. Early exchange of information enables stakeholders to adapt advice and optimize plans as new information is uncovered.
What are the opportunities and barriers?
The pace of innovation in life science and technology has never been faster. Today, there are many evolving trends such as more personalized medicines, use of medicine combinations, and advanced therapies that require novel ways of engaging across the healthcare space. We at Lilly believe that it is more important than ever to make sure that medicine development and clinical systems similarly continue to evolve to allow patient access to innovative medicine.
What is being done to progress the concept?
In 2015, we joined a coalition of 90 European healthcare stakeholders from over 30 organizations including patients, payers, regulators, Health Technology Assessment (HTA) bodies, academic institutions, health care professionals and industry to start the journey of exploring one such adaptive pathway approach called MAPPs (or Medicines Adaptive Pathways to Patients). From the start, the parties involved envisaged MAPPs as a way to encourage more efficient ways of developing and regulating medicines ultimately to provide patients with unmet medical needs to gain appropriate access to innovative therapies. This is done by developing tools that enable gathering, managing, and accessing as much information as possible on the new medicine to inform a decision about appropriate and safe early access. Appropriately, the project was given the name of ADAPT SMART and supported through the EU Innovative Medicines Initiative (IMI2).
Primarily, I’ve been involved in designing the MAPPs pathway. Our group was asked to chart the process and decision points within an adaptive pathway during medicine development and post-approval. To generate ideas for this effort, we hosted a multi-stakeholder workshop in London, which was an important step in working towards a model that is in line with the needs of patients, regulators, healthcare professionals and the industry.
Our project working group’s report has now been posted on the ADAPT SMART site welcoming comments and dialogue. This report contains a map of the key moments in which all the stakeholders that are part of the MAPPs process can share their respective views or take a decision to continue the development of a potential medicine. The tool will enable us to develop a workable adaptive pathway approach that shows the right timing for using the MAPPs pathway and the way all stakeholders will be involved in the process. Additional opportunities to share information, including a manuscript, about the outputs of ADAPT SMART are also in preparation.
With MAPPs’ goal to “foster access to beneficial treatments for the right patient groups at the earliest appropriate time”, Lilly has enthusiastically supported the initiative with our time and ideas. Indeed, patients are the focus as medicine development and clinical processes continually progress and ADAPT!
 Accelerated Development of Appropriate Patients Therapies, a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes.