Sec. 2. Findings
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/bill/118/hr/6288/ih/section-2A research copy — for the controlling text, always check the official state or federal source. Not legal advice.
Congress finds the following: Randomized clinical trials require the use of control groups to serve as the baseline for determining the effectiveness of a study treatment. Because of the rarity of some diseases, it is often hard to find enough study participants in a sufficient amount of time, making randomized clinical trials for these treatments infeasible. Couple this with the fact that the current standard of care for many rare diseases is ineffective (often leading to death), making administering the current standard of care unethical.
In these cases, data reflecting patient treatment in routine clinical practice can be used to develop external control groups for single‐arm trials. Clinical trials using external control cohorts can provide valuable benchmark results on potential comparator treatment efficacy. The Food and Drug Administration (in this section referred to as the FDA ) has distributed draft guidance on this issue commenting on considerations for the use of real-world data for the design and conduct of externally controlled trials.
Through collaboration with community advocates and industry partners with input from the FDA, researchers focused on atypical teratoid rhabdoid tumor (in this section referred to as ATRT ), an ultra-rare and poor prognosis pediatric brain tumor, have taken the lead in the design of real-world data sets. Because of the advancements in the research, ATRT has presented itself as the ideal candidate to pilot the development of real-world data sets for use in external control cohorts.