1. BIO believes that the public should have ready access to a comprehensive listing, in the form of a registry, of all confirmatory trials /1 that evaluate both safety and efficacy in patients who have the disease or condition to be treated, diagnosed, or prevented.
2. BIO supports the current requirements of ClinicalTrials.gov, which require information be provided about trials for serious or life-threatening conditions from product sponsors.
3. It is BIO's belief that ClinicalTrials.gov should be expanded beyond studies relating to serious or life-threatening conditions, to include the voluntary registration of all confirmatory trials that evaluate both safety and efficacy in patients who have the disease or condition to be treated, diagnosed, or prevented.
4. Information about studies described in Paragraph 3 should be supplied in summary format within 21 days after the start of patient enrollment. This should include information about whom to contact for further detail.
5. BIO is committed to the public disclosure, in a clinical trials database or otherwise, of the results of confirmatory trials for marketed drugs, including trials testing potential new indications, in an accurate, objective and balanced manner, regardless of whether the trial results are considered "positive" or "negative." The information disclosed will include (a) the results of the primary and secondary outcome measures that are specified in the study protocol, (b) safety information provided in summary form, and (c) a summary description of the trial design and methodology for each study. The content will be presented in a structure consistent with the standard summary format of the E3 Guideline issued by the International Conference on Harmonization (ICH) in July 1996, "Structure and Content of Clinical Study Reports," or, where clinical trial results have been published in a peer-reviewed journal, reference to such publication will be included by hyperlink or otherwise.
6. BIO supports public disclosure of the analysis of trial results for marketed drugs within one year of the completion of the analysis. If a study is under review by a peer-reviewed journal that prohibits pre-publication of results, an explanation should be placed in the database while publication of the manuscript is pending, and the results should be posted at the time of publication. In addition, in some instances there may be a delay in posting of complete trial summaries due to the need to seek intellectual-property protection or to comply with confidentiality provisions in agreements with other parties. In such cases, an explanation should be placed in the database. Notwithstanding the above, trial results pertinent to protecting the safety of the public should be made publicly available expeditiously.
/1 A confirmatory trial is an adequately controlled trial in which the hypotheses are stated in advance and evaluated and where the key hypothesis follows directly from the trial's primary objective, is always predefined, and is the hypothesis that is subsequently tested when the trial is complete. The rationale and design of confirmatory trials nearly always rests on earlier clinical work carried out in a series of exploratory studies which, in contrast to confirmatory trials, objectives may not always lead to simple tests of predefined hypotheses. Such trials cannot be the basis of the formal proof of efficacy, although they may contribute to the total body of relevant evidence.
(Adapted from the FDA Guidance entitled "E9 Statistical Principles for Clinical Trials," 63 Federal Register 49583-98 (Sept 16,1998)