Speeding drugs to market using adaptive pathways

For parents of children with serious disorders where there is no approved treatment, or physicians who have tried everything for their patients with terminal diseases, the wait for approval of innovative drugs is a long one. The European Medicines Agency’s adaptive pathways pilot project aims to allow patients and their physicians to access life-changing or life-saving medications before approval. The adaptive pathways pilot project was launched in March 2014.

The adaptive pathways process

The aim of the adaptive pathways process isn’t to create new regulatory tools, but to use the existing regulatory tools, including the full, conditional and under exceptional circumstances approvals frameworks to create a more flexible and faster approvals process.

The three principles behind the process are:

  • Iterative development
  • Gathering evidence
  • Early involvement of patients and health technology assessment bodies

Iterative development

The process begins with an iterative development plan, with the drug moving into a gradually expanding target population from an initial and clearly defined subpopulation with a high unmet need as more data becomes available. Alternatively, the drug may get a conditional approval based on surrogate endpoint data, bearing in mind the balance of the benefit and the risk, which can be ‘converted’ to a full approval. Both of these approaches mean that patients can gain access to drugs more quickly, but both physicians and patients need to be aware that there is a degree of risk involved as the drugs haven’t been through the entire clinical trial process, or have only been studied in small groups of patients.

Gathering the evidence

An essential part of the adaptive pathways process is the collection of real-world data to provide evidence in addition to that gleaned from randomised controlled trials. This comes from the everyday use of the drug in the selected populations, and adds to the battery of safety, efficacy and cost-effectiveness data. It can be used to support the step from conditional to full approval, inform label updates, and help to build a value story for communication with stakeholders.

Engaging with stakeholders

It’s important to communicate with stakeholders for all drug development projects, but it is especially important when working with adaptive pathways. This is because physicians and patient organisations play a key role in understanding unmet needs and finding patients for the studies, and also because gaining advice from regulators, HTAs and payers smooths the path to approval.

The outcomes of the pilot

In May 2016, a group of nine scientists sent a letter to the European Medicines Agency. They said that the concept had potential benefits, but were concerned about the levels of evidence behind a number of the assumptions made, and their potential operational applications. They suggested “a few changes and slight modifications”, based on experience in the rare diseases area, including clear and shared criteria for selection of drugs submitted to the adaptive pathway, plans for generation of evidence, and publication of all documents. The EMA responded, agreeing with many of the points, including the requirement for openness, and confirming the role of the pilot as a time for feedback and debate. For further details of the assumptions and ideas as outlined by the scientists, and the EMA’s response, download the pdf from the EMA website.

The aim of the pilot is to gain feedback and develop the process, and then integrate its findings into the existing drug approval pathway. These changes have potential to ensure that patients can access innovative drugs at an earlier stage, improving physician choice and helping improve outcomes in the groups most at need.

To find out how Valid Insight can shed light on the adaptive pathways process and help in defining product value and opportunity and in generating evidence for early stage innovative products, contact us at

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