Getting the Right Evidence After Drug Approval

Overview

Journal Front Pharmacol

Date 2020 Oct 5

PMID 33013409

Citations 5

Authors

Rick A Vreman

Hubert G M Leufkens

Aaron S Kesselheim

Affiliations

Soon will be listed here.

Abstract

To generate comparative evidence in a timely fashion for drugs without restricting or delaying access, value-based pricing and reimbursement could be conditioned on a prospective, post-approval evidence generation plan.

Citing Articles

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Added benefit and revenues of oncology drugs approved by the European Medicines Agency between 1995 and 2020: retrospective cohort study.

Brinkhuis F, Goettsch W, Mantel-Teeuwisse A, Bloem L BMJ. 2024; 384:e077391.

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Synergy between health technology assessments and clinical guidelines for multiple sclerosis.

Hogervorst M, Vreman R, Zawada A, Zielinska M, Dawoud D, de Jong B Clin Transl Sci. 2023; 16(5):835-849.

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Real World Data in Health Technology Assessment of Complex Health Technologies.

Hogervorst M, Ponten J, Vreman R, Mantel-Teeuwisse A, Goettsch W Front Pharmacol. 2022; 13:837302.

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An urgent call to raise the bar in oncology.

Schnog J, Samson M, Gans R, Duits A Br J Cancer. 2021; 125(11):1477-1485.

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References

Hatswell A, Baio G, Berlin J, Irs A, Freemantle N . Regulatory approval of pharmaceuticals without a randomised controlled study: analysis of EMA and FDA approvals 1999-2014. BMJ Open. 2016; 6(6):e011666. PMC: 4932294. DOI: 10.1136/bmjopen-2016-011666. View

Eichler H, Enzmann H, Rasi G . Added therapeutic benefit and drug licensing. Nat Rev Drug Discov. 2019; 18(9):651-652. DOI: 10.1038/d41573-019-00068-x. View

Yeung C, Shadman M . How to Choose the Best Treatment and Testing for Chronic Lymphocytic Leukemia in the Tsunami of New Treatment Options. Curr Oncol Rep. 2019; 21(8):74. DOI: 10.1007/s11912-019-0819-x. View

Eichler H, Barker R, Bedlington N, Bouvy J, Broekmans A, Bucsics A . The evolution of adaptiveness: balancing speed and evidence. Nat Rev Drug Discov. 2018; 17(12):845-846. DOI: 10.1038/nrd.2018.90. View

Tafuri G, Pagnini M, Moseley J, Massari M, Petavy F, Behring A . How aligned are the perspectives of EU regulators and HTA bodies? A comparative analysis of regulatory-HTA parallel scientific advice. Br J Clin Pharmacol. 2016; 82(4):965-73. PMC: 5137821. DOI: 10.1111/bcp.13023. View

Morant A, Vestergaard H . European Marketing Authorizations Granted Based on a Single Pivotal Clinical Trial: The Rule or the Exception?. Clin Pharmacol Ther. 2017; 104(1):169-177. DOI: 10.1002/cpt.900. View

Augustovski F, McClellan M . Current Policy and Practice for Value-Based Pricing. Value Health. 2019; 22(6S):S4-S6. DOI: 10.1016/j.jval.2019.04.1918. View

Vivot A, Jacot J, Zeitoun J, Ravaud P, Crequit P, Porcher R . Clinical benefit, price and approval characteristics of FDA-approved new drugs for treating advanced solid cancer, 2000-2015. Ann Oncol. 2017; 28(5):1111-1116. DOI: 10.1093/annonc/mdx053. View

Chen E, Raghunathan V, Prasad V . An Overview of Cancer Drugs Approved by the US Food and Drug Administration Based on the Surrogate End Point of Response Rate. JAMA Intern Med. 2019; 179(7):915-921. PMC: 6547222. DOI: 10.1001/jamainternmed.2019.0583. View

10.

Skydel J, Luxkaranayagam A, Dhruva S, Ross J, Wallach J . Analysis of Postapproval Clinical Trials of Therapeutics Approved by the US Food and Drug Administration Without Clinical Postmarketing Requirements or Commitments. JAMA Netw Open. 2019; 2(5):e193410. PMC: 6512282. DOI: 10.1001/jamanetworkopen.2019.3410. View

11.

Kang S, Bai G, DiStefano M, P Socal M, Yehia F, Anderson G . Comparative Approaches to Drug Pricing. Annu Rev Public Health. 2019; 41:499-512. DOI: 10.1146/annurev-publhealth-040119-094305. View

12.

Cipriani A, Ioannidis J, Rothwell P, Glasziou P, Li T, Hernandez A . Generating comparative evidence on new drugs and devices after approval. Lancet. 2020; 395(10228):998-1010. DOI: 10.1016/S0140-6736(19)33177-0. View

13.

Bloem L, Mantel-Teeuwisse A, Leufkens H, De Bruin M, Klungel O, Hoekman J . Postauthorization Changes to Specific Obligations of Conditionally Authorized Medicines in the European Union: A Retrospective Cohort Study. Clin Pharmacol Ther. 2018; 105(2):426-435. DOI: 10.1002/cpt.1169. View

14.

Naci H, Davis C, Savovic J, Higgins J, Sterne J, Gyawali B . Design characteristics, risk of bias, and reporting of randomised controlled trials supporting approvals of cancer drugs by European Medicines Agency, 2014-16: cross sectional analysis. BMJ. 2019; 366:l5221. PMC: 6749182. DOI: 10.1136/bmj.l5221. View

15.

Kim C, Prasad V . Cancer Drugs Approved on the Basis of a Surrogate End Point and Subsequent Overall Survival: An Analysis of 5 Years of US Food and Drug Administration Approvals. JAMA Intern Med. 2015; 175(12):1992-4. DOI: 10.1001/jamainternmed.2015.5868. View

16.

Hoekman J, Klamer T, Mantel-Teeuwisse A, Leufkens H, De Bruin M . Characteristics and follow-up of postmarketing studies of conditionally authorized medicines in the EU. Br J Clin Pharmacol. 2016; 82(1):213-26. PMC: 4917806. DOI: 10.1111/bcp.12940. View

17.

Naci H, Smalley K, Kesselheim A . Characteristics of Preapproval and Postapproval Studies for Drugs Granted Accelerated Approval by the US Food and Drug Administration. JAMA. 2017; 318(7):626-636. PMC: 5817559. DOI: 10.1001/jama.2017.9415. View