Testerone level should be used as a surrogate endpoint in phase 3 trials of gonadotropin-releasing hormone (GnRH) analogs for treating advanced prostate cancer, the FDA says in a new draft guidance.
Sponsors should conduct single-arm trials that measure attainment and maintenance of castrate testosterone (T) levels, according to the draft guidance released last week. Trials should look at T levels maintained until the end of a dosing interval as well as immediately after later doses — not the first dose — of the study drug.
“To demonstrate these effects of the study drug on T levels, the treatment period should be at least twice as long as the dosing interval,” the agency advises. Products that act over shorter periods, such as one month, should have treatment periods extending over three to four dosing intervals.
Trial participants should have normal, age-adjusted T levels and metastatic disease in order to produce a more accurate safety profile of the intended population, the FDA says. It recommends documenting information about patients’ prostate cancer history, including the diagnosis date, current stage, prior therapies and the extent of metastatic disease at baseline.
Any indications besides treating advanced prostate cancer should be discussed with the agency before a sponsor begins a trial, the draft guidance says. Randomized designs for supporting efficacy and/or safety among GnRH analogs also should be discussed with the agency.
The draft guidance also provides recommendations for dose selection; trial procedures and timing of assessments; pharmacokinetics and pharmacodynamics; statistical suggestions and labeling considerations.
Read the guidance here: https://bit.ly/32zoyVU.
A joint clinical study of patients with heart failure is using wearable biosensors to evaluate the viability and significance of patient-centric endpoints such as physical function and quality of life.
The multicenter study is being conducted by the Mayo Clinic and Yale University with assistance from the FDA in what is called a CERSI (Center of Excellence in Regulatory Science and Innovation), a collaboration between the agency and academic institutions.
The study follows a draft guidance the FDA released in June that would allow endpoints based on a treatment’s effect on patient-reported symptoms and experiences even if the treatment has no favorable effect on survival or risk of hospitalization (CenterWatch Weekly, July 1, 2019).
Researchers will use two different mobile devices to monitor discharged heart failure patients for 60 days, then analyze the data to measure the correlation between patient-reported outcomes and clinical endpoints, such as lab tests and physical examinations.
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) is seeking public feedback on how the agency involves patients in its work, including in clinical trials.
The goal is to find out how patients would like the MHRA to communicate with them and how patients would prefer to communicate with the MHRA to raise concerns.
“We want to adopt a more systematic approach to listening to and involving patients — ensuring that the patient voice is heard when safety issues, concerning medicines or medical devices, are identified and in the licensing of new medicines,” the agency says.MHRA’s “consultation” survey will be open until Oct. 7.
Take the MHRA survey here: https://bit.ly/32rGemB.