Forget 1997: ASCO Modernizes Phase 1 Trials Policy

Nick Mulcahy

December 15, 2014

Phase 1 clinical trials now offer a greater potential for clinical benefit than in the past because molecular medicine has taken a lot of the guesswork out of picking optimal participants, according to the American Society of Clinical Oncology (ASCO).

And for that reason — and others — ASCO has issued a policy statement about the trials, which are also referred to as "first-in-human" studies of new agents.

ASCO's first policy update in this area of oncology since 1997 was published online today in the Journal of Clinical Oncology.

The forces behind the new effort were summarized by ASCO President Peter Paul Yu, MD, from the Palo Alto Medical Foundation in California.

"With new agents that target specific abnormalities in a patient's tumor, better tests to identify those abnormalities, and more sophisticated clinical trial designs, today's phase 1 trials in cancer offer patients a greater likelihood of benefit than ever before," he said in a statement.

An update is especially needed because "significant barriers discourage patient enrollment in research," according to an ASCO statement.

The policy has five broad recommendations aimed at modernizing the participation of the five major stakeholders in clinical trials.

These interested parties are payers, professional societies and patient advocacy organizations, clinicians, researchers, and the biopharmaceutical industry and other trial sponsors.

ASCO reminds its policy readers that phase 1 studies seek to determine recommended dose and schedule, observe therapeutic effect, and assess safety.

Additional phase 1b studies can evaluate new schedules of existing agents or combinations of new agents with established agents or radiation.

ASCO to Payers: Pay Up

Recommendation 1 states that "payers provide insurance coverage for these trials." That includes the biggest payer of all — Medicare. The Centers for Medicare and Medicaid Services only requires that "therapeutic intent" be a component of a trial in order for payment to occur. "Phase 1 trials in cancer meet this standard. Thus, these trials should be covered by Medicare," write Jeffrey S. Weber, MD, PhD, from the Moffitt Cancer Center and Research Institute in Tampa, Florida, and colleagues in the policy statement.

"State Medicaid programs should reimburse for routine patient costs associated with clinical trials, including phase 1 trials," they suggest.

Recommendation 2 states that professional societies should improve the ability of clinicians and researchers to discuss the purposes and risks and benefits of phase 1 trials in cancer. Both professional and patient advocacy groups need to develop better materials for patients.

Recommendation 3 is similar to recommendation 2, and states that professional societies need to develop better educational materials for clinicians and researchers about insurance coverage for trials, among other challenges to enrolment.

Recommendation 4 states that researchers and trial sponsors should specify the therapeutic intent of phase 1 trials and adopt trial designs that minimize the number of patients exposed to potentially subtherapeutic doses.

Recommendation 5 states that the biopharmaceutical industry should initiate pediatric phase 1 trials for agents directly relevant to childhood cancers during the late phase 1 or early phase 2 adult drug development program timeframe.

The majority of the policy authors have ties to industry.

J Clin Oncol. Published online December 15, 2014. Abstract


Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.