A revised guidance aims to modernize clinical trials and ensure the safety and efficacy of novel treatments.
Photo has been resized. FDA photo courtesy of Wikimedia Commons.
The U.S. Food and Drug Administration (FDA) today released guidance on strategies to modernize clinical trials to support the development of precision medicines, according to an announcement.
“The advent of precision medicine is challenging the entire medical research ecosystem to develop more efficient approaches to testing and developing diagnostics and therapeutics…” Scott Gottlieb said in a statement.
Precision medicine has the potential to use science to reduce the suffering, death and disability caused by human illnesses.
With more diseases being redefined based on genomic subtype, there are more novel targets and opportunities for researchers to precisely modulate or repair the basic biological drivers of illness.
Precision medicines can demonstrate strong efficacy signals in early clinical trials that could allow earlier regulatory assessment of benefit or risk. If the FDA can make a positive approval decision, patients can gain earlier access to important new therapeutic options.
While the FDA has worked with stakeholders to identify trial designs and help validate novel endpoints that can generate evidence, the agency still sees a reluctance to adopt innovative approaches among sponsors and clinical research organizations.
Guidances were released for strategies to support the development of precision medicines and on risk-based monitoring.
In Enrichment Strategies for Clinical Trials to Support Determination of Effectiveness of Human Drugs and Biological Products Guidance for Industry, the FDA lists three strategies to enrich the study population: strategies to decrease variability, prognostic enrichment strategies and predictive enrichment strategies.
Enrichment strategies rely on the selection of patients for clinical trials based on one or more characteristics intended to demonstrate the safety or effectiveness of the drug or biologic in selected populations.
These strategies allow signal detection in smaller clinical trials. The information gathered can guide clinical practice to help doctors get the right treatment to the right patient at the right time.
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