The US Food and Drug Administration (FDA) recently finalized four new final Guidance for Industry documents for cancer clinical trial eligibility criteria that describe ways that clinical trials can safely include patients who have historically been excluded. The guidance documents complement ongoing work by ASCO and Friends of Cancer Research (Friends) to eliminate overly restrictive eligibility criteria that can exclude patients from trials without scientific or participant safety justification. Clinical trials provide patients with cancer access to promising investigational therapies – which are sometimes the only treatment option available – and advance clinical care by producing safety and efficacy data about new therapies.
The FDA guidance documents, which are intended for use by trial sponsors, encourage broader eligibility criteria for cancer clinical trials related to:
- HIV, Hepatitis B, and Hepatitis C: This guidance document considers inclusion of patients with HIV infection, with subsections devoted to evaluation of immune function and considerations related to HIV therapy. It also provides insight on inclusion of patients with evidence of chronic HBV infection or history of chronic HCV or virologically suppression on HCV treatment.
- Organ Dysfunction, or Prior and Concurrent Malignancies: This guidance document provides information on inclusion of patients with renal, cardiac, and hepatic dysfunction, as well as inclusion of patients with prior or concurrent malignancies.
- Brain Metastases: This guidance document provides information for inclusion of patients with brain metastases that have been treated, are stable, or are active. It also includes patients with leptomeningeal disease, as well as recommendations for exclusion of patients with brain metastases in certain cases and guidance on baseline central nervous system screening.
- Minimum age: This guidance document examines inclusion of pediatric patients, including ethical and regulatory considerations and considerations for early phase versus late phase trials.
ASCO applauds these new FDA guidance documents, which build on the Society’s longstanding focus on expanding access to clinical trials. ASCO and Friends began their effort to develop and advance specific strategies to change the exclusionary nature of eligibility criteria in 2016. They published a Joint Research Statement in 2017 and that included specific recommendations. In 2018, ASCO and Friends submitted language to the FDA for the initial draft guidances that was based on the 2017 recommendations. ASCO has collaborated with the FDA and the National Cancer Institute for many years on this issue and will support efforts to encourage robust uptake of the new guidance by trial sponsors throughout the cancer community.