This dataset is the result of an investigation characterizing nonefficacy benefits of newly approved molecular entities (NMEs) based exclusively on noninferiority trials. Qualifying NMEs were identified using the FDA’s Novel Drug Approval website, sponsor press releases at market entry, FDA Drug Trials Snapshots, and statistical and medical officer reports. A total of 18 were selected for analysis. All FDA and sponsor nonefficacy benefit statements were extracted and categorized. Additionally, the IBM Micromedex Red Book was consulted for average unit prices for drug cost comparisons. The dataset includes all information sources for the NMEs as well as the noninferiority data extraction sheet.

For two decades the standard drug therapy for patients with Crohn’s disease (CD) and ulcerative colitis (UC) has been anti-tumor necrosis factor agents (anti-TNFs) such as adalimumab (Humira), certolizumab pegol (Cimzia), infliximab (Remicade) and several others. More recently, vedolizumab (Entyvio) has entered the market as the first gut-selective integrin blocker that offers an alternative treatment for inflammatory bowel disease (IBD). Using the FDA Adverse Event Reporting System (FAERS) database, this study compared adverse event reports (AE) for the two types of therapies. The search retrieved 499 reports for vedolizumab and 119,620 for anti-TNFs for the years 1998 through 2015. Using the proportional reporting ratio (PPR) and the empirical Bayesian geometric mean (EBGM) algorithms, AE data were reviewed for signals of disproportionate reporting for the two types of drugs. This dataset includes statistical summary tables of characteristics (demographic) and a variety of adverse event comparisons of vedolizumab, anti-TNFs, and all other medications. Cleaned FAERS data was provided with permission by DrugLogic, Inc. Interested researchers may contact DrugLogic Inc. (Reston, VA) by calling 800-393-1313 or emailing swordham@druglogic.com

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, Amphotericin B, were summarized in the Amphotericin B: Summary Report accessible via the UMB Digital Archive.

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, boric acid, were summarized in the Boric Acid: Summary Report accessible via the UMB Digital Archive.

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, benzocaine, were summarized in the Benzocaine: Summary Report accessible via the UMB Digital Archive.

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, chloral hydrate, were summarized in the Chloral Hydrate: Summary Report accessible via the UMB Digital Archive.

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, cantharidin, were summarized in the Cantharidin: Summary Report accessible via the UMB Digital Archive.

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, coenzyme Q10, were summarized in the Coenzyme Q10: Summary Report accessible via the UMB Digital Archive.

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, clioquinol, were summarized in the Clioquinol: Summary Report accessible via the UMB Digital Archive.

The University of Maryland Center of Excellence in Regulatory Science and Innovation (M-CERSI), in collaboration with the Food and Drug Administration (FDA), conducted research and outreach to solicit input from the public, including medical specialists, to better understand the use of certain bulk drug substances nominated for use in compounding by outsourcing facilities under section 503B of the Federal Food, Drug, and Cosmetic Act (the Act). In particular, we were interested in how drugs compounded with these bulk drug substances were used historically, and how they are currently used in clinical practice. The research will assist the FDA in its development of a list of bulk drug substances that outsourcing facilities can use in compounding under section 503B of the Act. Research for each bulk drug substance included a systematic literature review, interviews with medical experts and a survey of healthcare practitioners. The data on findings for the nominated substance, citrulline, were summarized in the Citrulline: Summary Report accessible via the UMB Digital Archive.