ADAPT SMART Round Table – Thought leaders discuss objectives and approaches for success

On 27th October, thought leaders in the field of adaptive pathways met in Washington D.C. to discuss the global objectives of IMI ADAPT SMART and what the landmark project hopes to achieve.

ADAPT SMART (a Sustainable, Multi-stakeholder Approach from Research to Treatment-outcomes) aims to enable the coordination of Medicines Adaptive Pathways to Patients (MAPPs) activities within the Innovative Medicines Initiative. The D.C. session brought to the table three ADAPT SMART work package leads: Richard Barker, founding director Centre for the Advancement of Sustainable Medical Innovation (CASMI); Sarah Garner, Associate Director Science Policy and Research, National Institute for Health and Care Excellence (NICE); and Alicia Granados, Head Global Health Technology Assessment (HTA) Scientific Strategy Patients Outcomes and Medical Economics, Sanofi Genzyme.

ADAPT SMART is driven by science

MAPPs seeks to foster access to beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span in a sustainable fashion. Operating under the Innovative Medicines Initiative (IMI) ADAPT SMART will support IMI2 projects investigating MAPPs tools and methodologies, and support dialogue to prove and develop workable MAPPs concepts.

“What we’re trying to do… is to create a more collaborative approach,” said Sarah Garner, speaking on the need to progress from traditional models of medicines development.

Richard Barker said “We are moving into a new era of more adaptive development, one that is enabled by new science, particularly that of precision medicine. There is a need for the “ultimate personalised medicine”, one that is in fact motivated by the patients; the idea of MAPPs, he said, is to adapt the process so that it keeps up with the changes in science.

In discussions around Adaptive Pathways, the idea of earlier access often raises questions of uncertainty, patient safety and how to manage risk. However, Barker noted that smaller amount of well-characterised patients, and therefore shorter trials, does not necessarily translate to higher risk.

Coming from the rare diseases space, Granados championed the importance of a methodological approach using real world data as soon as possible. “Of course if there are issues we want to pick them up as early as possible,” agreed Garner – saying that this is in everyone’s interests.

A pan-European and multi-stakeholder initiative 

ADAPT SMART brings together patients, regulators, industry, health technology assessment (HTA) bodies, payers, and academics. Those present at the roundtable agreed that this full gamut of input is needed to address all legitimate questions and current uncertainties for Adaptive Pathways implementation to succeed. 

ADAPT SMART is trying to combine learnings from the many initiatives currently underway that aim to change the way research is done, said Granados. This includes IMI projects and beyond. “It’s a pan-European initiative,” said Barker, “It’s not just being driven by the UK or any other single country – it’s truly collective”.

“A lot of it is working out where the synergies and similarities are, and then working out the best way to capitalise on these,” said Garner. This, the roundtable participants agreed, is essential to help ensure that the results can be implemented later.

From new evidence to decision-making

Barker, Granados and Garner are leading or co-leading two of the three work packages of ADAPT SMART:

Work Package 1, “Evidence generation throughout the entire product life cycle”, is being led by NICE and AstraZeneca. It’s two core objectives are to analyse and monitor activities that occur both within and outside of IMI programmes that provide and generate evidence for MAPPs; and to develop a gap analysis of the current and future needs of evidence generation to support MAPPs.

Speaking on Work Package 1, Garner explained: “What we’re going to do is look through the different methodologies for evidence generation in different stages… and see where the opportunities lie in those distinct methodologies and to determine how we can maximise output.”

Work Package 3, “Decision-making, sustainability and their implications” is being led by the CASMI and Sanofi Genzyme. The Work Package aims to address the opportunities and obstacles to a successful, sustainable and ethically responsible implementation of adaptive decision making in research, regulatory, and medical practice As part of WP3 the collaborators are evaluating actual experience with managed entry agreements which are a range of mechanisms that allow pharmaceutical firms and payers to share some of the financial and clinical risk associated with the introduction of a new medicine. If any tools to manage some of the inherent uncertainties are missing, IMI or other mechanisms could be used to develop and test them.

Communication is key

“Open communication on the process and findings to patients and to the general public is essential to reassure people” Barker added.