Recent guidance from the US Food and Drug Administration (FDA) calls for pharmaceutical companies to include more older people in cancer trials.
A central reason, according to the FDA, is that “older adults are underrepresented in cancer clinical trials despite representing a growing segment of the population of cancer patients,” and clinical trials need to more accurately represent the patient population likely to use the drug in clinical practice.
The guidance — one of three the agency released earlier this month — aligns closely with the goals of the reinvigorated Cancer Moonshot initiative, which President Joe Biden led during his last year as vice president and which aims to halve cancer deaths over the next 25 years.
Although cancer incidence increases with age, trial participation tends to skew toward younger, less medically complex patients. Currently, about half of patients with cancer are 65 or older, and just over 40% are 70 or older, but only about one quarter of clinical trial participants fall into that 70-plus age bracket.
That lack of representation poses a problem because drug efficacy can vary by age, and toxicity is often worse in older people, owing to comorbidities and other factors.
The FDA’s Inclusion of Older Adults in Cancer Clinical Trials guidance seeks to increase inclusion of adults aged 65 years or older, and particularly those above 75, in everything from phase 1 trials to postmarketing studies. The agency also said comorbidities should be considered in trial design because older adults often have additional health problems and may be taking medications that could influence the efficacy or side effects of a cancer drug.
The FDA notes that the guidance is not binding, but it stresses that understanding a drug’s benefit-risk profile as well as potential drug interactions early in development in older adults can “maximize the generalizability of the trial results” and help inform treatment decisions.
The FDA recommendations included two other guidance statements — one asks industry to design and conduct trials with multiple expansion cohorts, and the other proposes creating more “efficient” clinical trials that can evaluate more than one investigational drug or type of cancer in the same trials.
M. Alexander Otto is a physician assistant with a master’s degree in medical science. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape and is an MIT Knight Science Journalism fellow. Email: [email protected].