Abstract
Patient recruitment and retention are arguably the greatest challenges to the timely execution of clinical trials. This is particularly true in the case of trials involving biosimilars and those focused on rare diseases. For biosimilars, recruitment success typically hinges on difficulty of access to reimbursement for the originator product and may be hindered by competition from studies with other biosimilars and those with new chemical entities. For rare diseases, recruitment success depends not only on finding enough patients but also on retaining them for the duration of the trial. Historical success with patient recruitment—in addition to site qualifications—needs to be considered in parallel with current market competition and results in an ever-changing patient recruitment environment. Multiple companies supporting the biopharmaceutical industry, such as Contract Research Organizations, have begun to leverage sophisticated data modeling tools and techniques to find additional patients for biosimilar and rare disease trials and/or to find patients more quickly. In addition, these companies seek to better understand the population of interest and refine their statistical assumptions when conducting clinical trials or real-world analyses. The largest and most well-established companies that support the biopharmaceutical industry now have unparalleled access to big data from clinical trials, electronic health records, medical claims, laboratory tests, and prescriptions. This paper will discuss how big data can be harnessed to aid patient recruitment with a focus on clinical trials for biosimilars and orphan rare diseases.
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References
The Center for Biosimilars Staff. Infographic: biosimilars and rare diseases. 28 Feb 2019.
The Center for Biosimilars Staff. Russian drug maker gains world’s first approval for biosimilar eculizumab. 11 Apr 2019.
Huml RA, Dean M. Designing effective rare disease registries. Drug Discovery and Development, https://globalgenes.org/2017/04/23/designing-effective-rare-disease-registries/. Accessed 10 Dec 2019.
Isaacman D, Iliach O, Keefer J, Campion DM, Kelly B. Registries for rare diseases: a foundation for multi-arm, multi-company trials. IQVIA White Paper (IQVIA.com). Feb 2019.
Heads of Medicines Agencies (HMA)-European Medicines Agency (EMA) Joint big data taskforce summary report. Accessed 13 Feb 2019.
Hansen AJ. Health data provide insights into rare diseases, say panelists at big data in precision health. Scope, Stanford University. 29 May 2018.
Raghupathi W. Data mining in health care. In: Kudyba S, editor. Healthcare informatics: improving efficiency and productivity; 2010. p. 211–223.
Raghupathi W, Raghupathi V. Big data analytics in health care: promise and potential. Health Inf Sci Syst. 2014;2(1):3.
FDA Draft Guidance: Rare Diseases. Rare diseases: natural history studies for drug development guidance for industry. March 2019.
Legal Information Institute Website. Cornell Law School: 21 U.S. Code § 355 g—Utilizing Real World Evidence. Accessed 15 May 2019.
International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use; ICH Harmonised Tripartite Guideline: Choice of Control Group and Related Issues in Clinical Trials. E10 Guidance. 20 July 2000.
Gaasterland CMW, Jansen MC, van der Weide MJP, et al. The patient’s view on rare disease trial design—a qualitative study. Orphanet J Rare Dis. 2019;14:31.
FDA website; Rare diseases: natural history studies for drug development guidance for industry. Mar 2019.
Augustine EF, Adams HR, Mink JW. Clinical trials in rare disease: challenges and opportunities. J Child Neurol. 2013;28(9):1142–50. https://doi.org/10.1177/0883073813495959.
FDA website: Real-world Evidence, https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence, 9 May 2019. Accessed 2 Jun 2019.
FDA website: Morales JP. The use of registry data to support regulatory decisions. FDA Division of Cardiovascular Devices CRN for DAISI Workshop. 3 Feb 2017.
FDA web page: An Introduction to FDA MyStudies: an Opensource, Digital Platform to Gather Real World Data for Clinical Trials and Research Studies. 9 May 2019.
FDA website: Framework for FDA’s Real-world Evidence Program. Dec 2018.
Patel D. IQVIA blog: securing market access with single-arm trial data: why external comparators could be the answer (IQVIA.com). 14 Mar 2019.
Parent Project Muscular Dystrophy (PPMD) website: Wave Life Sciences Duchenne Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program. 3 Jan 2019.
FDA website: Complex Innovative Trial Design Pilot Program. Food and Drug Administration. 20 Dec 2018.
Gliklich RE, Levy D, Karl J, Leavy MB, Taylora T, Campion DM. Registry of patient registries (RoPR): project overview. Agency for Healthcare and Research Quality. May 2012.
Contract Pharma website: MDA Selects Quintiles for Disease Registry. 8 Oct 2103.
Muscular Dystrophy Association (MDA) website: MDA Collaborates with IQVIA to Expand Disease Registry into the New MOVR Data Hub. 12 Mar 2018.
European Union Clinical Trials Register. A randomized, double-blind, active-controlled phase 3 study evaluating the efficacy and safety of ABP 959 compared with eculizumab in adult subjects with paroxysmal nocturnal hemoglobinuria (PNH). https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-001418-27/ES. Accessed 15 May 2019.
Kos IA, Azevedo VF, Neto DE, Kowalski SC. The biosimilars journey: current status and ongoing challenges. Drugs Context. 2018;7:212543. https://doi.org/10.7573/dic.212543.
Dowlat HA. The opportunities and challenges of biosimilar orphans. Expert Opin Orphan Drugs. 2016;4(6):563–6. https://doi.org/10.1517/21678707.2016.1171142.
Pharma Intelligence website: Orphan Biosimilar: Soliris is One Target. 14 Jun 2017.
FDA website: Guidance Document—Rare Diseases: Common Issues in Drug Development Guidance for Industry. Jan 2019.
ClinicalTrials.gov website: A Study Evaluating the Efficacy and Safety of ABP 959 Compared with Eculizumab in Adult Participants with PNH (DAHLIA). US National Library of Medicine. Accessed 15 May 2019.
Acknowledgements
The authors would like to thank Dr. Oxana Iliach, Senior Director of Regulatory Affairs in IQVIA’s Biosimilars Center of Excellence, and Denise Messer, Engagement Manager from IQVIA’s Design Analytics and Next Generation Evidence Driven Design group, for their editorial comments and insights.
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Huml, R.A., Dawson, J., Lipworth, K. et al. Use of Big Data to Aid Patient Recruitment for Clinical Trials Involving Biosimilars and Rare Diseases. Ther Innov Regul Sci 54, 870–877 (2020). https://doi.org/10.1007/s43441-019-00009-1
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DOI: https://doi.org/10.1007/s43441-019-00009-1