Final answer:
Basket trials are used in phase I studies to assess safety and effectiveness of a drug across different mutations and cancers. They differ from compassionate use or expanded access programs and are not the same as bridging studies. Basket trials contribute to methodological approaches that improve the efficiency of drug evaluation.
Step-by-step explanation:
When researchers add small cohorts of a diseased population to phase I studies to assess the initial safety and effectiveness in the drug's targeted population, they typically use an approach known as a basket trial. Basket trials are designed to test how a drug affects different mutations across a variety of cancers. This approach contrasts with expanded access programs or compassionate use, which provide access to unapproved drugs for patients who do not meet the criteria for clinical trials. Bridging studies, on the other hand, are used to provide additional information on the efficacy or safety of a medicine.
Basket trials have become increasingly popular as they allow a more efficient evaluation of a drug's action on specific genetic mutations, regardless of the cancer's location in the body. It streamlines the clinical trial process by potentially reducing the number of patients needed and allows researchers to more quickly identify responsive sub-populations. The strategic incorporation of such adaptive study designs including a broad range of methodological approaches may help bridge the gap between research outcomes and patient access to potentially life-saving drugs.
It's crucial during phase I clinical trials to evaluate the pharmacokinetic properties of the drug, including its absorption and elimination. The trials must be strictly based on the guidelines of the ICH, EMA, and FDA. Their purpose extends beyond just determining safety but also includes assessing the correct dosing range to move forward with further development.