FDA Issues Draft Guidance on Conducting Multiregional Oncology Trials

Clinical regulation helps enable consistent study results

Oncology trials are becoming more global. While 22% of active, industry-sponsored oncology trials are multicountry, this percentage rises to 45% when looking at registrational trials alone.* The advantages are obvious from faster patient recruitment and expedited regulatory approvals to reduced operational costs—to name a few.

With this trend, however, there is a notable decline in the number of US patients being represented in these trials. With cancer presentation and treatment standards differing across regions, the US Food and Drug Administration (FDA) has become extra vigilant to ensure global study results are not being generalized across the US population and that the effects observed in the overall study population are consistent with those being seen in US study participants.

Responding to this shift, the FDA issued draft guidance “Considerations for Generating Clinical Evidence from Oncology Multiregional Clinical Development Programs” aimed at helping the industry conduct global cancer clinical trials more effectively. The aim is to provide sponsors with recommendations when planning, designing, and conducting multiregional clinical trials in support of marketing applications for cancer drugs in the US.

There are several important key points raised in the draft guidance.

Encouragement of multiregional clinical trials (MRCTs)

The FDA promotes the use of MRCTs but emphasizes the importance of ensuring that clinical trial data is applicable to the US population and aligns with US standard medical practice. There is a need to consider regional differences in prevalence, causes and presentation of disease when interpreting data in the context of the US population.

The FDA is recommending that sponsors conduct a MRCT across continents as opposed to a single country or geographical area. Companies may be required to conduct additional trials if the data is not representative of the diverse US population.

Considerations for site selection and data analysis

Included within the guidance are recommendations on the selection of clinical trial sites, as well as incorporating regional information on the disease and standard of care in the data analysis. A rationale for the sponsor’s geographical approach needs to be included. Sites should be selected to ensure diversity within the patient population and expanded beyond tertiary and academic centers into community hospitals.

Addressing decreasing US participation

The decline in the proportion of US participants in oncology MRCTs could impact the ability to interpret the data. Regional allocation of trial participants should align with disease prevalence while evaluating regional differences.

Improving generalizability and applicability

The draft guidance aims to help sponsors improve the planning, design, conduct, and analysis of MRCTs to ensure the results are relevant to US patients. This highlights the importance of sponsors outlining their diversity action plan early in their development plans, ideally as early as Phase I.

Public comment period

The FDA is accepting comments on the draft guidance. Comments must be submitted in the Federal Register within 60 days after publication, which is November 18, 2024, to Regulations.gov to ensure the agency considers them.

Once finalized, these guidelines are designed to streamline the process and ensure that global clinical trials produce data that can be effectively used for FDA approvals in the US.

We can help

Catalyst Oncology is an oncology and biotech specialty CRO with a robust pipeline of global early- and late-phase cancer trials. With over 1,000 staff across North America, Europe, and the Asia-Pacific region, Catalyst Oncology is well-positioned to run MRCTs as encouraged by this draft FDA guidance. Reach out to discover how we can support your global oncology program.

* Statistics from Citeline Trialtrove database