简明语言摘要的监管、伦理和患者参与考虑

Regulatory, E thical and P atient E ngagementConsiderations for P lain L anguage S ummaries While there is general consensus within thepharmaceutical industry on the importanceof providing plain language summaries (PLS)of clinical trial results to participants and thepublic as a whole, two of the largest regulatoryagencies ― the European Medicines Agency andthe Food and Drug Administration ― differ ontheir approach to the matter. trials in a format understandable to the generalpublic. In the US, the Multi-Regional ClinicalTrials (MRCT) Center of Brigham and Women’sHospital and Harvard, in collaboration withTransCelerate Biopharma, submitteddraftguidanceon PLS to the FDA in 2017. To date, theFDA has not required sponsors to submit PLS.The guidance remains open forpublic comment.Table 1 provides a side-by-side comparisonof the EU requirement and the proposed USguidance. In Europe, the EMA’s EU Clinical TrialsRegulation 536/2014(Article 37 EU CTR) requiressponsors to provide summary results of clinical Clinical Trials Information System (CTIS), whichlaunched in January 2022. Protocol synopsis(PLPS) are not a regulatory requirement in EEA/EU member states. However, ethics committeesin some countries require them, so they areconsidered a de facto requirement if not aregulation. The original EU CTR stipulated that laysummaries, more commonly referred to asplain language summaries, would be madeavailable in a new EU database once it becomesavailable. That time has come. As of Jan. 31,2023, all initial clinical trial applications inthe EU/EEA must be submitted through the Regulatory, E thical and P atient E ngagementConsiderations for P lain L anguage S ummaries For adult clinical trials, sponsors must posta plain language summary and a technicalsummary within 12 months of the end ofthe trial. For non-therapeutic Phase I trialssponsors have up to 30 months after the trialhas concluded, and for pediatric trials within sixmonths of the trial’s end. Green reminds that they must be free ofpromotional language. This ensures they reporton the results with no editorializing, even whencomparing one drug to another. The PLS writermust be someone who is objective, and “whoknows the science really well,” Green says. Tounderscore objectivity, some sponsors postPLS on a website unaffiliated with their owncompany, such asTrialSummaries.com. The EU regulations have not changed, theysimply became official when the regulation wentinto effect, explains disclosure and transparencyexpert Kimberly Green, founder of ClaritiDox. Allnew studies in the EU fall under the regulation,she says, adding that existing studies musttransition by the end of this year. She describesPLPS clause as “a wonderful and meaningfuladdition, although a bit of a surprise.” The factthat the EMA indicated these synopses will bemade public “will be really good for patients,”she says. Although the US has not made PLS mandatory,the FDA has recently emphasized the value ofusing language easily understood by the public.It encourages sponsors to use plain language inpresenting trial information, including informedconsent materials, to aid in the decision-makingprocess of whether to join a study. On Feb.29, FDA and the Office for Human ResearchProtections (OHRP) at the Department of Healthand Human Services (HHS) published draftguidance on best practices. Regulatory, E thical and P atient E ngagementConsiderations for P lain L anguage S ummaries It’s the right thing to do •Feeding the “info-demic” in medicalresearch literature Regulations aside, providing a PLS is simplythe ethical choice. However, according to onejournal article, even the PLS itself can spurethical debate. In the article referring to plainlanguage resources (PLR), which include PLS,the authors assert that, while PLR may addressmany ethical issues, they may potentiallyexacerbate the following: To avoid this, the authors recommend thereshould be standard guidelines for how PLR aredeveloped and shared. “I think it’s important to note that, evenwhen the product didn’t do what youhoped it did, it’s still advancing researchand important to share that,” Green says.Whether the study was deemed a successor not, “there’s no failure here.” •Challenges to fair balanced presentationand interpretation of medical research•Existing positive publication bias•Challenges to equitable access toinformation•Ambivalent outcomes of patients’autonomy Here’s an example of how PLS can take thecomplex and make it understandable for all: Clinicaltrials.gov PLS ICH E3 The patients in your studyall had a type of lymphoma.They had all receivedtreatment for cancer at leastonce before, but their cancerhad returned. Everyone in thestudy received the same kindof medicine. The patients sawtheir doctors every week tosee how the medicine worked. This is a Phase II, open-label, single-arm study ofdrug in approximately 65patients with disease. Theprimary efficacy endpoint isORR (CR + PR) in determinedby IRC. Efficacy t