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Exclusive Drug Labeling Rights as a New Incentive for Contribution to a Communal Biomarker Resource

Published online by Cambridge University Press:  06 January 2021

Extract

Biomarkers are an important tool in modern drug development. The FDA has posited that increased use of biomarkers in clinical trials can accelerate pharmaceutical industry productivity, ushering new drugs to market. Accordingly, the FDA has created two pathways for evaluation of biomarker utility. Biomarkers incorporated into clinical trials, the traditional pathway, are effectively private to a therapeutic sponsor and to the scope of the trial. By contrast, in Biomarker Qualification (“BQ”), the second pathway, a biomarker is certified as a publicly available tool. The FDA has hoped that academic, non-profit, and industry stakeholders would work together in consortia to qualify biomarkers, cumulatively generating a common resource of broad utility. In practice, utilization of Biomarker Qualification has been paltry. Incentives for BQ that align with the interests of industry resource holders are necessary to fuel increased utilization of biomarkers in clinical trials and create the communal biomarker toolkit envisioned by the FDA. A blanket extension of exclusivity for any drug successfully paired with a companion biomarker would diminish public access to medicine by encouraging spurious biomarkers and correspondingly narrowed clinical trials. As a measured alternative, an exclusive right to include a qualified companion biomarker on an FDA drug label would balance public access externalities. This exclusivity would waylay label approval, and thus marketability, of later drugs relying on the qualified biomarker for clinical safety or efficacy. Accordingly, sponsors would find no incentive to portage an ineffective or unnecessary biomarker through clinical trials, as there would be no benefit to securing exclusive rights in a tool others saw no value in using.

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Copyright © American Society of Law, Medicine and Ethics and Boston University 2018

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47 21 C.F.R. § 314.125(b)(2), (b)(5) (2017).

48 21 C.F.R. § 314.105(c) (2017).

49 Id.

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51 21 C.F.R. § 314.105(c) (2017).

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67 Id. at 6.

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69 Id. at 11; 21 C.F.R. §§ 201.56-201.57 (2017).

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78 Id. at 7-13.

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81 Id. at 7-13.

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83 Biomarkers Consortium Evidentiary Standards Writing Group, supra note 76.

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85 Id. at 3.

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87 Id. at 8, 20. In somewhat more technical terms, the guidance identifies precision, accuracy, limit of detection, limit of quantitation, specificity, linearity and range, ruggedness and robustness as fundamental assessment factors. Id. at 18.

88 Id. at 13, 14. A unique feature of this guidance is that it establishes three tiers of risk/benefit (favorable, intermediate, and challenging) with increasing evidentiary demands. Id. at 16,17. Here, the FDA has provided tables identifying evaluative factors and what would satisfy “minimal” or “high” evidentiary criteria. This scheme maintains flexibility while also providing guidance that goes well beyond that available in statute, CFR, or other relevant guidance documents. Id. at 23.

89 21st Century Cures Act § 3011, 21 U.S.C. § 357 (2016).

90 Id.

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103 Id. at 66, 72-73, 87.

104 Id.

105 Id. at 79-80.

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107 Id.

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131 Id.

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142 Id.

143 Id.

144 Id.

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146 Beach, supra note 43, at S5-S6.

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148 FDA 2017 Case Study, supra note 25, at 4.

149 Id. at 5.

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151 U.S. Dep't of Health and Human Servs., Food and Drug Admin., Drug-Diagnostic Co-Development Concept Paper; Draft – Not for Implementation 7, 15 (2005).

152 21 U.S.C. § 355(d) (2017).

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158 Id. at 2; 21 C.F.R. § 314.108 (2017).

159 CDER Small Business and Industry Assistance (SBIA), Patents and Exclusivity 2 (2015); 21 C.F.R. § 316.31 (2017).

160 Nate Aumock et al., McKinsey Center for Government, Do Incentives Drive Pediatric Research? (2013), file:///Users/Home/Downloads/Do_incentives_drive_pediatric_research.pdf.

161 Id.

162 Table of Pharmacogenomic Biomarkers, supra note 16.

163 By contrast, a regulatory scheme under which development could only begin after the exhaustion of exclusivity would create further, substantial time delays for second comers.

164 Already, the FDA has indicated that Qualification submissions and assessments will be public in accordance with transparency provisions of the 21st Century Cures Act. U.S. Food and Drug Admin., Drug Development Tool Qualification Process: Transparency Provisions (Nov. 17, 2018), https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm561587.htm. [https://perma.cc/VF7Y-SK99].