Authors
Nikhil Nair1*, Devea R. De2*, Jonathan W. Rick3, Jennifer L. Hsiao4, Vivian Y. Shi3 *Denotes co-first authors 1. University of Arkansas for Medical Sciences, College of Medicine, Little Rock, AR, USA 2. University at Buffalo, Jacobs School of Medicine and Biomedical Sciences, Buffalo, NY, USA 3. University of Arkansas for Medical Sciences, Department of Dermatology, Little Rock, AR, USA 4. University of Southern California, Department of Dermatology, Los Angeles, CA, USA
Introduction
Clinical trials are necessary to develop novel therapeutics and they represent a major investment of both time and money. Due to the high costs associated with bringing new drugs to the market, there has historically been a lack of diversity among drugs developed and targeted conditions in dermatology. Off-label use of medications is commonly used in dermatologic practice and the lack of Level 1 evidence for approved treatments makes clinical trials for new drug approval vital. In this study, we aim to analyze characteristics and patterns of the top ten pharmaceutical companies' investments in dermatologic clinical trials.
Methods
The top-earning ten pharmaceutical companies were chosen in June 2020 based on 2019 end of year revenues from the Yahoo Finance database. Dermatologic drug trials sponsored by companies were analyzed through the ClincialTrials.gov database. Data was collected on the dermatologic condition addressed, drug characteristics, study length and phase, method of drug administration, and participant characteristics. An ANOVA test was performed to evaluate if companies with higher valuations had more active/recruiting studies. P<0.05 was considered statistically significant.
Results
There were 344 dermatologic trials undertaken between 2010 and 2021. Most trials (82%) were in either phase 2 or 3 (phase 2, n=141; phase 3, n=140). Merck had the most ongoing (n=45) and Johnson & Johnson had the fewest ongoing (n=0) trials; Novartis had the highest overall number of trials (19 ongoing with 46 completed). The majority (81%) of trials included adult-only participants. 86% of trials (n=295) involved one of ten dermatologic conditions, with melanoma (n=74, 22%) leading, followed by psoriasis (n=74, 22%) and atopic dermatitis (n=61, 18%). 86% of trials (n=297) were dedicated to systemic medications and 14% (n=47) focused on topicals. Acne was the only condition that had more topical drug trials (66.7%) than systemics. Melanoma had the most systemic drug trials (n=75) while morphea had the least (n=1). Most companies had significantly more systemic drug trials than topical drug trials except Roche (p=0.06) and GlaxoSmithKline (p=0.52). Novartis had the most systemic medications (n=64) while Pfizer had the most topicals (n=20). The performed ANOVA demonstrated that companies with higher valuation had more active/recruiting studies (p=0.01).
Conclusion
Our results highlight that dermatologic clinical trials over the last decade focused on a select few conditions, adult populations, and systemic medications. Adult populations are often selected for initial trials of drug development due to limited vulnerability of the population itself and application of results to a more general cohort compared to pediatric trials. The focus on the ten conditions leaves numerous others conditions under-investigated. The overall paucity in diversity of clinical trials may be attributed to the high cost and time investment requirements. Research of underrepresented conditions is progressing and potential exists for new niches to be developed around diseases that have not yet been at the forefront of clinical trial attention.
References
Sertkaya A, Birkenbach A, Berlind A, et al. Examination of clinical trial costs and barriers for drug development. 24 July 2014. Office of the Assistant Secretary for Planning and Evaluation (ASPE). Accessed at https://aspe.hhs.gov/reports/examination-clinical-trial-costs-barriers-drug-development-0 on 5 October 2021.
Moore TJ, Zhang H, Anderson G, et al. Estimated costs of pivotal trials for novel therapeutic agents approved by the US Food and Drug Administration, 2015-2016. JAMA Intern Med. 2018;178:1451-1457. [PMID: 30264133] doi:10.1001/jamainternmed.2018.3931
Ali Z, Zibert JR, Thomsen SF. Virtual clinical trials: perspectives in dermatology. Dermatology. 2020;236:375-382. [PMID: 32126560] doi:10.1159/000506418
American Academy of Dermatology Association. Skin conditions by the numbers. American Academy of Dermatology. Accessed at www.aad.org/media/stats-numbers on 26 September 2021.
França K, Litewka S. Controversies in off-label prescriptions in dermatology: the perspective of the patient, the physician, and the pharmaceutical companies. Int J Dermatol. 2019;58:788-794. [PMID: 30168846] doi:10.1111/ijd.14222
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