MFDS Adds Patient Experience Data to GIFT Reviews, Introduces Checklist

The Ministry of Food and Drug Safety (MFDS) will systematically incorporate patients’ experiences and perspectives into expedited reviews of treatments for rare and serious diseases.

The National Institute of Food and Drug Safety Evaluation, under MFDS, said on the 28th that it had revised the “Considerations for Applying for Priority Review Designation of Medical Products (Guidance for Civil Petitioners)” to allow patient experience data to be used in the Global Innovative Product on Fast Track (GIFT) program.

GIFT is a program for expedited review of innovative global medical products, including treatments for life-threatening serious diseases and 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 real-world patient experience data on symptoms, functional status, quality of life and difficulties 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 guidance is the addition of a patient experience data checklist to the GIFT designation application. The revision establishes a process for companies to indicate data types and whether they are submitting them, and to submit patient experience data alongside the application. It also adds a detailed explanation of “serious, severe diseases,” which are eligible for GIFT designation.

Patient experience data refers to information 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.

In GIFT designation reviews, MFDS has so far assessed eligibility requirements primarily by considering disease severity, the availability of alternative medicines, and whether the product offers improved safety and efficacy compared with available alternatives. Under the revision, when companies submit additional patient experience data, it may be used as reference material in assessing the severity of rare and serious diseases and the benefits and risks of the product under review.

MFDS said it expects the revision to help companies better understand GIFT designation and improve the predictability of reviews. The agency 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 can make a tangible difference to patients with rare and serious diseases and their families.

The newly established patient experience data checklist includes patient-reported outcomes, observer-reported outcomes, clinician-reported outcomes and performance outcomes as clinical outcome assessment data. It also covers patient preference studies; qualitative research, including interviews or focus group interviews with patients or caregivers; patient engagement activity materials, such as videos, recordings and written comments; and other data, including natural history studies and survey research.

More information is available under Laws and Resources on the MFDS website (www.mfds.go.kr).