Patient-Focused Drug Development: New FDA Draft Guidance Delves Into What’s Important

Regulatory NewsRegulatory News | 30 September 2019 |  By 

The US Food and Drug Administration (FDA) on Monday published the second in a series of four guidance documents on patient-focused drug development, with this latest draft helping sponsors identify what is most important to patients with respect to their experience with disease burden and treatment.

In determining what’s important, FDA recommends that sponsors use qualitative research methods, which can generate in-depth thoughts from patients in their own words through interviews or focus groups, quantitative research methods, which can use statistical methods to summarize the collective patient experience data, or mixed-methods research, which can combine qualitative and quantitative methods to understand the patient experience.

The 45-page draft discusses several ways to collect qualitative information, including via one-on-one interviews and focus groups, with hints on how to ask the right types of questions.

On quantitative methods, the draft explains different surveys and what to consider, including examples of what could potentially be leading or double-negative questions.

Examples of mixed-method study designs are also explained further, alongside questions researchers should ask themselves and reasons to use a mixed-method design.

In addition, the draft includes a section on specific populations, culturally diverse populations and considerations for using social media, which discusses the importance of keeping the various social platforms and users in mind.

“When possible, social media research should examine a variety of social media networks and communities to obtain data that can be most generalized to the population of interest,” the agency adds. The draft also includes six appendices, including a glossary of terms and different types of examples for each of the research methods.

The release of the draft guidance coincides with the Monday release of a new perspective article on engaging patients co-authored by both FDA and European Medicines Agency officials in Nature Reviews Drug Discovery.

The perspective focuses on initiatives from both regulators that have allowed for further patient or public engagement, and further engagement between them.

“Our responsibility is the same for all patients: to ensure that they receive the safest, most effective medicines and that, as much as possible, their needs and concerns are considered in the evaluation process,” EMA and FDA officials wrote. “Given the global aspects of medicine regulation, collaborating and sharing experiences, information and resources between agencies is also vital, and exchanges such as those seen through the collaboration between the EMA and the FDA are helping to improve patients’ trust in and understanding of the regulatory process.”

FDA, meanwhile, which previously released the first patient-focused drug development draft guidance in June 2018, under this new initiative created by the 21st Century Cures Act, will also release two other guidance documents dealing with approaches to identify and develop methods to measure impacts in clinical trials and methods, standards and technologies to collect and analyze clinical outcome assessment data for regulatory decision-making.

Patient-Focused Drug Development: Methods to Identify What Is Important to Patients Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders


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