Feb 6th, 2015 – by Magda Chlebus


While Europe is leading in the development of tools and methodologies in Medicine’s Adaptive Pathways (MAPPs) through the EMA live pilots and upcoming projects in IMI2, the key bottlenecks still holding back its implementation in Europe are gaining traction with the United States congress. This situation illustrates our need in Europe to move quickly and develop implementation strategies and foster political support for MAPPs whilst we still hold the drive and initiative.

There is much to like in the discussion draft released on Tuesday, January 27th, by the US Congress’ 21st Century Cures. Coming in at nearly 400 pages, the document is a detailed legislative proposal that has been tabled to outline how the US Commerce Commission hopes to redefine and accelerate healthcare delivery in the US, focusing particularly on the role of patient choice and input into the regulatory process.

The document articulates the need for, “the use of patient experience data to enhance structured risk-Benefit assessment frameworks,” and highlights a list of regulatory innovations that align with many of the desired goals inherent in MAPPs. These include standards for the review of surrogate endpoints, the approval of drugs for the use in a limited population of patients, a streamlined data review programme, and an expanded access policy as a condition of expedited approval.

As well, 21st Century Cures has also taken a very forward-looking position on the harnessing and application of real world evidence stating, “the Secretary shall establish a program under which a sponsor may submit real-world evidence for purposes including (1) to support the approval of the use of a drug for a new indication; and (2) to support or satisfy post-approval study requirements…the term ‘real-world evidence’ means data about the usage, benefits, or risks of a drug derived from sources other than randomized clinical trials, including from observational studies and registries, used to establish safety or effectiveness.” The use of observational data has not yet had a public endorsement such as the EMA’s adaptive pathways pilot project in the US, but the objectives of 21st Century Cures place it in alignment with the EMA, in principle.

Most closely aligned with MAPPs, the document outlines an overall comprehensive strategy that attempts to create tools, approaches and methodologies that harness patient outcomes data for the entire life-cycle of the treatment. Many of the current discussions in Europe around the implementation of MAPPs hinge on the ability of regulators, payers, patients and industry to agree on an evidence plan at launch, and rotating milestones for lifecycle evaluation. 21st Century Cures proposes a series of initiatives to design and prove processes that can make using multiple data streams possible, and determine the effectiveness of a new treatment in the real world.

In addition to 21st Century Cures embracing Adaptive Pathways, on January 30th President Obama also announced a $215 mil Precision Medicine Initiative that puts stratified healthcare at the centre of US policy. Both initiatives show that US regulators are addressing the need for more timely access to therapies that improve patient outcomes, and that they are fully engaged with facilitating the adaption of new methodologies. The US is making great strides ahead and it is vital that European regulators keep up.