The US Food and Drug Administration (FDA) has published two new draft guidance documents that will provide clarity to medical product developers on the agency’s recommendations for researching and developing the next generation of individualised therapies.
Called ‘Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease’, the first draft guidance addresses the important topic of finding treatments that address the underlying molecular changes that often cause or contribute to diseases.
The guidance proposes a new approach for drug developers to enrol patients based on the identification of rare mutations into clinical trials for targeted therapies.
It also discusses the evidence that is required to demonstrate effectiveness for various molecular subsets within a particular disease.
This is expected to lead to consistent development and approval of targeted therapies for patients.
The second draft guidance, ‘Investigational IVD Devices Used in Clinical Investigations of Therapeutic Products’, discusses the way of determining if an in-vitro diagnostic (IVD) device used in a study must undergo its own FDA review.
Once finalised, it is set to clarify the appropriate regulatory pathway for investigational in-vitro diagnostic (IVD) devices used in clinical trials for therapeutic products.
Building on the IVD draft guidance, FDA is also considering methods to streamline the review of oncology therapeutic products and the IVDs used with them.
FDA said in a statement: “These draft guidances are just a few examples of the FDA’s ongoing efforts to advance the development of innovative, targeted drugs and foster the availability of individualised treatment approaches.
“Finally, we specifically addressed a more efficient pathway for developing drugs targeted to the rare pediatric disorder Gaucher disease.”