Regulatory science, the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of regulated medical products, has advanced over time due to a number of factors. The FDA, the EMA, and the Pharmaceuticals and Medical Devices Agency (PMDA) have recently formalized voluntary data submission processes for the regulatory “qualification” of novel tools and methodologies for use in drug development. While recognizing that other mechanisms exist within the research community for driving scientific consensus on novel tools and methodologies, this article focuses on the formal regulatory process that addresses a tool’s acceptability for incorporation by any sponsor into novel medical product development. Guidances, regulatory qualification opinions, and publications were reviewed to allow a systematic comparison of the process, content, and volume of submissions at the FDA, EMA, and PMDA. Qualification of new tools by regulatory agencies and subsequent adoption by drug developers are anticipated to speed therapeutic development for patients in need, build scientific consensus as to the usefulness and readiness of novel methodologies for understanding disease and therapeutic development, and decrease uncertainty between the regulators and sponsors regarding the appropriate application of new tools.
ScannellJWBlanckleyABoldonHWarringtonB. Diagnosing the decline in pharmaceutical R&D efficiency. Nat Rev Drug Discov. 2012;11(3):191–200.
2.
Institute of Medicine. Accelerating the Development of New Drugs and Diagnostics: Maximizing the Impact of the Cures Acceleration Network: Workshop Summary. Washington, DC: National Academies Press; August 2012.
3.
GoldmanM. The innovative medicines initiative: a European response to the innovation challenge. Clin Pharmacol Ther. 2012;91(3):418–425.
4.
Tralau-StewartCJWyattCAKleynDEAyadA. Drug discovery: new models for industry-academic partnerships. Drug Discov Today. 2009;14(1-2):95–101.
5.
SelkoeDJ. Resolving controversies on the path to Alzheimer’s therapeutics. Nat Med. 2011;17(9):1060–1065.
6.
LathiaCDAmakyeDDaiW. The value, qualification, and regulatory use of surrogate end points in drug development. Clin Pharmacol Ther. 2009;86(1):32–43.
7.
SouzaTKushREvansJP. Global clinical data interchange standards are here!Drug Discov Today. 2007;12(3-4):174–181.
8.
MarrerEDieterleF. Impact of biomarker development on drug safety assessment. Toxicol Appl Pharmacol. 2010;243(2):167–179.
9.
CoonsSJKothariSMonzBUBurkeLB. The patient-reported outcome (PRO) consortium: filling measurement gaps for PRO end points to support labeling claims. Clin Pharmacol Ther. 2011;90(5):743–748.
10.
GoodsaidFPapalucaM. Evolution of biomarker qualification at the health authorities. Nat Biotech. 2010;28(5):441–443.
11.
GoodsaidFMAmurSAubrechtJ. Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact. Nat Rev Drug Discov. 2010;9(6):435–445.
12.
WoodcockJBuckmanSGoodsaidFWaltonMZinehI. Qualifying biomarkers for use in drug development: a US Food and Drug Administration overview. Expert Opin Med Diagn. 2011;5(5):369–374.
13.
SistareFDDieterleFTrothS. Towards consensus practices to qualify safety biomarkers for use in early drug development. Nat Biotechnol. 2010;28(5):446–454.
WoosleyRLMyersRTGoodsaidF. The Critical Path Institute’s approach to precompetitive sharing and advancing regulatory science. Clin Pharm Ther. 2010;87(5):530–533.
21.
MattesMBWalkerEGAbadieE. Research at the interface of industry, academia and regulatory science. Nat Biotechnol. 2010;28(5):432–433.
22.
IchimaruKToyoshimaSUyamaY. PMDA’s challenge to accelerate clinical development and review of new drugs in Japan. Clin Pharmacol Ther. 2010;88(4):454–457.
23.
Nature Biotechnology (special issue The Predictive Safety Testing Consortium). 2010;28(5):431–494.
HarpurEEnnulatDHoffmanD; HESI Committee on Biomarkers of Nephrotoxicity. Biological qualification of biomarkers of chemical-induced renal toxicity in two strains of male rat. Toxicol Sci. 2011;122(2):235–252.
