MFDS to incorporate patient experience data into GIFT reviews, introduces standardized application form

Patients’ experiences and perspectives will be systematically incorporated into designation reviews under the Global Innovative product on Fast Track (GIFT), an expedited review program for treatments for rare and serious diseases.

The National Institute of Food and Drug Safety Evaluation, under the Ministry of Food and Drug Safety, said on the 28th that it had revised its guidance for applicants, “Considerations for Applying for Priority Review Designation for Medical Products.”

GIFT (Global Innovative product on Fast Track) is a program that expedites reviews of globally innovative medical products with outstanding innovation, including treatments for life-threatening serious diseases or rare diseases.

The revision was pursued as part of the Food and Drug Safety Confidence Initiative. It is intended to enable the use at the GIFT designation stage of patient experience data on symptoms, functional status, quality of life and challenges during treatment among patients with rare and serious diseases—information that may be difficult to adequately capture from clinical trial results alone.

The main change to the applicant guidance is the addition of a patient experience data checklist to the GIFT designation application. A process has been established for companies to indicate the types of data and whether they are submitting them, and to submit patient experience data with their application. The guidance also adds a detailed explanation of “serious, severe diseases,” which are eligible for GIFT designation.

Patient Experience Data (PED) refers to data collected to understand patients’ experiences and perspectives, including symptoms, functional status, quality of life and treatment experiences related to a disease or its treatment. It includes clinical outcome assessment data, such as patient-reported outcomes (PROs); patient preference studies; qualitative research, such as interviews with patients and caregivers; and data collected through patient engagement activities.

Until now, in GIFT designation reviews, MFDS has assessed eligibility requirements primarily by considering the seriousness of the disease, whether alternative medicines are available, and whether the product offers improved safety and efficacy compared with available alternatives. Under the revision, if a company submits additional patient experience data, the information may be used as reference material in assessing the seriousness of rare and serious diseases and the benefits and risks of the product under application.

MFDS expects the revision to help companies better understand GIFT designation and improve the predictability of reviews. It also plans to systematically incorporate the experiences and perspectives of patients, who use the medicines, into GIFT designation reviews and support the rapid approval of treatments that patients with rare and serious diseases and their families can experience as beneficial.

The patient experience data checklist includes clinical outcome assessment data (patient-reported outcomes, observer-reported outcomes, clinician-reported outcomes and performance outcomes); patient preference studies; qualitative research (including interviews with patients and caregivers and focus group interviews); patient engagement activity data (including patient videos, transcripts and written comments); and other data (including natural history studies and survey studies).

More information is available on the MFDS website (www.mfds.go.kr) under “Laws and Data → Legal Information → Guidance for Public Officials/Guidance for Applicants.”