Abstract 84P
Background
Clinical trials (CTs) leading to FDA approvals should give an estimation of investigational drugs’ effect on enrolled patients (pts); however, enrollment criteria do not always accurately reflect a real-world population. As such, demographics and baseline characteristics of enrolled pts are essential to evaluating the applicability and safety of study drugs in the intended use population, particularly underrepresented minorities (URMs).
Methods
We searched “Drugs@FDA” to identify CTs that led to FDA drug approvals for gynecological cancers (GynC) between 2006 and 2024. We assessed the demographics and baseline characteristics, including ECOG Performance Status (PS), older adults (OA), race, and ethnicity in the published CTs.
Results
Out of 437 FDA approvals for solid tumors, 30 (4.6%) were for GynC based on 23 CTs: 73.9% phase 3, 21.8% phase 2, and 4.3% phase 1. Of note, 91.2% (21/23) of CTs led to approvals granted after 2014. ECOG PS was reported in 82.6% (19/23) CTs, with only 26.1% (6/23) CTs allowing the enrollment of pts with ECOG PS up to 2; the median proportion of enrolled pts with ECOG PS of 2 was 6.4 % (IQR 5.6-7.1%). The proportion of enrolled OA was reported in 30% (7/23) of CTs, all of which were published on or after 2014. The median proportion of enrolled OA was 36.8% (IQR 16.9-43.7%). Race was reported in 52.2% (12/23) of CTs, with a median proportion of 78.9% white pts enrolled (IQR 72.3-85.5). Ethnicity was reported in 30.4% (7/23) trials, but only in 13% (3/23) of CTs was reported separately from the race.
Conclusions
After years of limited therapeutic advancement, 2014 marked a new era for GynC treatment, with many FDA approvals. Despite the FDA’s recommendations regarding data collection, demographics and baseline characteristics are still underreported. Data on traditionally URMs (non-white race, Hispanic or Latino ethnicity, and pts with a non-optimal PS) are often lacking and deserve further inclusion in future CTs to assess the applicability of new drugs in the real world. Actionable first steps to achieve this goal include expanding eligibility criteria, establishing engagement and partnerships with communities and institutions, and prioritizing diversity.
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
E. Giudice: Financial Interests, Personal, Invited Speaker: GSK; Financial Interests, Personal, Other, Grant for travelling: MSD. D. Lorusso: Financial Interests, Personal, Advisory Board, Participation in Advisory Boards and Invited Speaker: GSK, Clovis Oncology, PharmaMar; Financial Interests, Personal, Advisory Board, Participation in Advisory Boards and Invited Speakers: AstraZeneca, MSD; Financial Interests, Personal, Other, Consultancy: PharmaMar, AstraZeneca, Clovis Oncology, GSK, MSD, Immunogen, Genmab, Seagen, Novartis; Financial Interests, Personal, Advisory Board, Invited member of advisory board and invited speaker: Seagen, Immunogen, Genmab; Financial Interests, Personal, Advisory Board, Invited member of advisory board: Oncoinvest, Corcept, Sutro; Financial Interests, Institutional, Funding, Grant for founding academic trials: MSD, Clovis Oncology, GSK, PharmaMar; Financial Interests, Institutional, Invited Speaker, ENGOT trial with institutional support for coordination: Clovis Oncology; Financial Interests, Institutional, Invited Speaker, ENGOT trial with institutional support for coordination: Genmab, MSD; Financial Interests, Institutional, Funding, Clinical trial/contracted research: AstraZeneca, Clovis Oncology, GSK, MSD, Seagen; Financial Interests, Institutional, Funding, Clinical trials/contracted research: Genmab, Immunogen, Incyte, Novartis, Roche; Non-Financial Interests, Personal, Principal Investigator, PI of several trials, no compensation received: GSK; Non-Financial Interests, Personal, Principal Investigator, PI of several trials. No personal compensation received: AstraZeneca, Genmab; Non-Financial Interests, Personal, Principal Investigator, PI in several trials. No personal compensation received: MSD; Non-Financial Interests, Personal, Principal Investigator, PI of clinical trial. No personal compensation received: immunogen, Clovis Oncology, Roche, Incyte; Non-Financial Interests, Personal, Principal Investigator, PI of several trials, no personal compensation received: Novartis; Non-Financial Interests, Personal, Principal Investigator, PI of clinical trial, no personal compensation received: Seagen; Non-Financial Interests, Personal, Principal Investigator, PI of clinical trials, no personal compensation received: PharmaMar; Non-Financial Interests, Personal, Member, Board of Directors: GCIG; Other, Personal, Other, Grants for traveling: AstraZeneca, Clovis Oncology, GSK. G. Daniele: Financial Interests, Personal, Invited Speaker: GSK, Gilead. G. Scambia: Financial Interests, Personal, Invited Speaker, Speaker: Baxter Healthcare, GSK, Intuitive Surgical Inc., AstraZeneca & MSD, Olympus Europa, GSK, AstraZeneca & MSD, Olympus Europa; Financial Interests, Personal, Expert Testimony, Trainer: Covidien AG (Medtronic company); Financial Interests, Institutional, Invited Speaker, ‘IsoMSLN’ in Ovarian Cancer and Malignant Pleural Mesothelioma: Kiromic; Financial Interests, Institutional, Invited Speaker, Roll-over study for patients who have completed a previous cancer study with olaparib and who the investigator believes can benefit from continued treatment - ROSY-O: AstraZeneca; Financial Interests, Institutional, Invited Speaker, CATCH-R: Roll-over study to provide continuous access to clinical therapy with rucaparib.: Clovis Oncology; Financial Interests, Institutional, Invited Speaker, Phase 3, multicenter, placebo-controlled clinical study comparing chemo-immunotherapy (paclitaxel-carboplatin-oregovomab) versus chemotherapy (paclitaxel-carboplatin-placebo) in patients with advanced epithelial ovarian, tubal cancer of fallopian or peritoneal (FLORA-5): Oncoquest Pharmaceuticals Inc.; Financial Interests, Institutional, Invited Speaker, Phase 2b randomized, open-label, active comparator, parallel-group, multicenter study designed to evaluate the efficacy and safety of three different doses of the P2X3 receptor antagonist (BAY 1817080) versus placebo and Elagolix 150 mg in women with symptomatic endometriosis: Bayer AG; Financial Interests, Institutional, Invited Speaker, Usability of ITE transducers for sending electric fields for tumor treatment (TTFields): Novocure Ltd; Financial Interests, Institutional, Invited Speaker, Phase III, multicentre, open-label extension trial to evaluate long-term safety and efficacy in patients with advanced cancers currently undergoing treatment or in follow-up in a pembrolizumab trial: Merck. V. Salutari: Financial Interests, Personal, Other, honoraria/consultation fees: AstraZeneca, MSD, GSK, PharmaMar, Novocure. All other authors have declared no conflicts of interest.