Over the past decade, regulators and healthcare authorities have become increasingly aware of the important role that patients can play in developing new medicines and getting them to market. In light of this, in new legislation in the US, the 21st Century Cures Act, which was passed in December 2016, the FDA has been charged with incorporating more of the patient perspective and experience into the drug development and review processes.
Title III Section 3002 of the Cures Act requires the FDA to create new methods and approaches for capturing and measuring patient input. The agency will be required to share these new methods and approaches through one or more guidance documents over 5 years.
The FDA has announced that it intends to release four guidance documents over the given time period. The guidances will address topics of interest to stakeholders wishing to engage in, or promote, patient-focused drug development and regulatory decision making. Public workshops will be held, prior to the release of each draft guidance document, to allow the FDA to gather input from patients, caregivers, advocacy organizations, healthcare providers, medical researchers, industry, etc.
The first guidance document, to be released by the end of 2018, will describe approaches that can be used to collect patient and caregiver input on the burden of disease and current therapy. The second document, to be released by the end of 2019, will describe processes that can be used to help develop a holistic set of impacts that are most important to patients. The third document, to be released by the end of 2020, will describe approaches that can be used to develop an identified set of impacts which will help with the collection of meaningful patient input in clinical trials. The final document, also to be released by the end of 2020, will provide guidance on clinical outcome assessments, which, when finalized, can be used to revise or supplement the Guidance for Industry, Patient Reported Outcome Measures, which was released in 2009.
For more information, click here to access the FDA document detailing the agency’s plans for delivering on Title III Section 3002 of the Cures Act.