Pharma industry sees US FDA guidance on patient-focused drug development to bolster faster treatment access

Bengaluru, July 13, 2022:

Pharma industry sees considerable relevance to the recent US FDA draft norms on patient-focused drug development: selecting, developing, or modifying fit-for purpose clinical outcome assessments.

This guidance, according to the global regulatory authority US FDA, is the third in a series of four methodological patient-focused drug development (PFDD) documents that portray how patients, caregivers, researchers and medical product developers, can collect and submit patient experience data to be used for medical product development and regulatory decision-making.

The pharma industry noted that draft guidance for which the comments need to be submitted to the regulatory authority will spur faster treatment access. This is because of the considerable importance given to clinical trial data.

Coming to the norms, it provides the methods to collect patient experience data that are accurate and representative of the intended patient population. The guidance also offers approaches to identifying what is most important to patients with respect to their experience as it relates to disease condition and burden of treatment. It also helps to selecting, modifying, developing, and validating clinical outcome  assessments (COAs) to measure outcomes of importance to patients in clinical trials. There are methods, standards, and technologies to collect and analyze clinical outcome assessments data for regulatory decision-making.

The global regulator in the guidance said that it encourages stakeholders to interact early and obtain feedback from its review division when considering collection of patient experience data related to the burden of disease and treatment. Further, it also recommends that stakeholders engage  with patients and other appropriate subject matter experts like qualitative researchers, clinical and disease experts, survey methodologists, statisticians, psychometricians, patient preference researchers when designing and implementing studies to evaluate the burden of disease and treatment. Such interactions also allow perspectives on treatment benefits and risks.

The framework discussed in this guidance, according to US FDA is for development of well-constructed measures based on developing evidence-based rationales. This is useful for discussing the broad range of clinical outcome assessments addressed by this guidance and helps to clarify evidence that may be appropriate to support the rationale for using a particular assessment.

Another type which is a proxy-reported outcome measure is discouraged by FDA. This measure is an assessment in which someone other than the patient reports on patient experiences as if the individual were the patient. FDA acknowledges that there are instances when it is impossible to collect valid and reliable self-report data from the patient. In these instances, it is recommended that an Observer-reported outcomes (ObsRO) measure be used to assess the patient’s behavior rather than a proxy-reported measure to report on the patient’s experience.

Besides the regulatory authority putting developments in perspective said that there has been a rapid evolution in digital health technologies (DHTs), which can be used to collect health care-related data from study participants in clinical trials. A DHT is a system that uses computing platforms, connectivity, software, and sensors for healthcare and related uses.  This may include use as a measurement tool for clinical outcome assessments in clinical investigations. Nevertheless the pharma industry sees US FDA guidance on patient-focused drug development to bolster faster treatment access and is a step in the right direction. Pharmabiz