Table 1.
Select FDA OWH Research: Regulatory Impact
| Research description | Study type | No. of participants/records | Outcome | Regulatory relevance |
|---|---|---|---|---|
| Developmental toxicity of toremifene11 | Animal study | Rat pups distributed in developmental groups: Postnatal days 1–5 (neonatal), 10–14 (infantile), and 20–24 (immature) | Indicated that toremifene is developmentally toxic | Contribution to labeling warning for toremifene |
| Predicting survival in oncology studies using tumor size12 | Observational | Four drug registration trials for nonsmall lung cancer treatments | Developed quantitative model for predicting survival | Quantitative tool for early drug development decisions and phase III trial design |
| Qualifying efficacy of HIV diagnostic tool in women13 | Observational | Multiple cohorts of men and women were included in the study | HIV-Selectest sensitivity in women scored 98.1% | Diagnostic tool for HIV-1 vaccine development |
| PBPK modeling for prediction of drug disposition14–16 | In silico study | CYP3A,14 CYP1A2, CYP2D6,15 CYP2C9, CYP2B6, CYP2C1916 | Development of PBPK model that can estimate the change in clearance of a drug during pregnancy | Tool for optimizing clinical trial design and drug dosing in pregnancy |
| Development of software codes17 and lesion phantom18 | In silico study | N/A | Development of dynamic lesion phantom and software code, penMesh | Tools for standardization and optimization of breast DCE-MRI and other imaging systems |
| Assessment of safety of hemostasis devices19–22 | Observational | Reports of serious injuries and deaths from CDRH Medical Device Reporting system from 1996 to 2000 and NCHS data19 | Identified relatively high rates of local vascular complications associated with VasoSeal, compared to Perclose, AngioSeal, and manual controls | Manufacturers voluntarily ceased marketing of VasoSeal following dissemination of research |
| 166,680 cardiac catheterizations20 from 2001, ACC-NCDR | ||||
| 13,878 cardiac catheterizations from 2003, ACC-NCDR21 | ||||
| Maternal exposure to ACEI in first trimester23 | Observational | 465,754 mother–infant pairs | Maternal use of ACE inhibitors in first trimester is not associated with greater risks of birth defects compared to the use of other antihypertensive medication or underlying condition of hypertension | Safety and efficacy of drugs during pregnancy |
| Vertebroplasty in osteoporotic women24 | Ex vivo study | 13 vertebral columns from adult white female cadavers | Identified potential lack of benefit for highly osteoporotic patients | Prognostic information can be used to inform enrollment and treatment criteria of clinical trials |
| Predictive prognostic markers for women receiving CRT-D25–28 | Observational | 144,642; 107,475 male and 37,167 female25 | Identification that women with LBBB have a greater benefit from CRT-D and at shorter QRSD | Significant sex difference in device performance from meta-analysis |
| 31,892; 20,350 males and 11,542 females26 | ||||
| 4076; 3191 males and 878 females27 | ||||
| 75,079; 51,335 males and 23,744 females28 | ||||
| Predicting liver toxicity in botanical extracts29 | In silico study | Four botanical extracts used by women for MHT: black cohosh, red clover, hops, and chasteberry | Identified structural features and botanical chemicals with toxicological potential | Computational tools used for prediction of hepatobiliary adverse events |
ACC-NCDR, American College of Cardiology National Cardiovascular Data Registry; ACEI, angiotensin converting enzyme inhibitors; CRT-D, cardiac resynchronization therapy defibrillator; DCE-MRI, dynamic contrast-enhanced magnetic resonance imaging; FDA OWH, Food and Drug Administration Office of Women's Health; LBBB, left bundle branch block; PBPK, physiologically based pharmacokinetic; QRSD, QRS complex duration.