What Evidence is Essential for New Medical Products?
Implications for Patients and Health Policy

June 13, 2014
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Bibliography of Key Articles

Ardaugh B, Graves S, Redberg RF. The 510(k) ancestry of a metal-on-metal hip implant. New England Journal of Medicine 2013;368:97-100.

Avorn J. Approval of a tuberculosis drug based on a paradoxical surrogate measure. JAMA 2013;309(13):1349-1350.

Avorn J. Evaluating drug effects in the post-Vioxx world: there must be a better way. Circulation 2006;113(18):2173-2176.

Broder S. The development of antiretroviral therapy and its impact on the HIV-1/AIDS pandemic. Antiviral Research 2010;85(1):1-18.

Carpenter D, Chattopadhyay J, Moffitt S, Nall C. The complications of controlling agency time discretion: FDA review deadlines and postmarket drug safety. American Journal of Political Science 2012;56(1):98-114

Connor EM, Smoyer WE, Davis JM, Zajicek A, Ulrich L, Purucker M, Hirschfeld S. Meeting the demand for pediatric clinical trials. Science Translational Medicine 2014;6(227):227fs11.

Darrow JD, Avorn J, Kesselheim AS. New FDA breakthrough-drug category—implications for patients. New England Journal of Medicine 2014;370:1252-1258.

Dhruva SS, Bero LA, Redberg RF. Gender bias in studies for Food and Drug Administration premarket approval of cardiovascular devices. Circ Cardiovasc Qual Outcomes 2011;4(2):165-71.

Dhruva SS, Bero LA, Redberg RF. Strength of study evidence examined by the FDA in premarket approval of cardiovascular devices. JAMA 2009;302(24):2679-2685.

Djulbegovic B, Hozo I, Ioannidis JP. Improving the drug development process: more not less randomized trials. JAMA 2014;311(4):355-356.

Downing NS, Aminawung JA, Shah ND, Braunstein JB, Krumholz HM, Ross JS. Regulatory review of novel therapeutics—comparison of three regulatory agencies. New England Journal of Medicine 2012;366(24):2284-2293.

Downing NS, Aminawung JA, Shah ND, Krumholz HM, Ross JS. Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005-2012. JAMA 2014;311(4):368-377.

Fain K, Daubresse M, Alexander GC. The Food and Drug Administration Amendments Act and postmarketing commitments. JAMA 2013;310(2):202-204.

Kesselheim AS, Myers JA, Avorn J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA 2011;305:2320-2326

Ledford H. FDA under pressure to relax drug rules. Nature 2012;492:19.

Madigan D, Ryan P. What can we really learn from observational studies?: the need for empirical assessment of methodology for active drug safety surveillance and comparative effectiveness research. Epidemiology 2011;22(5):629-631.

Mayer M. Listen to all the voices: an advocate’s perspective on early access to investigational therapies. Clinical Trials 2006;3(2):149-153.

Moore TJ, Furberg CD. Development times, clinical testing, post-market follow-up, and safety risks for the new drugs approved by the US Food and Drug Administration: the class of 2008. JAMA Internal Medicine 2014;174:90-95.

Moore TJ, Furberg CD. The safety risks of innovation: the FDA’s expedited drug development pathway. JAMA 2012;308(9):869–870.

Prah Ruger J. Governing health. Harvard Law Review 2008;121:43-56.

Rome BN, Kramer DB, Kesselheim AS. FDA approval of cardiac implantable electronic devices via original and supplement premarket approval pathways, 1979-2012. JAMA 2014;311(4):385-391.

Schneeweiss S, Gagne JJ, Glynn RJ, Ruhl M, Rassen JA. Assessing the comparative effectiveness of newly marketed medications: methodological challenges and implications for drug development. Clinical Pharmacology and Therapeutics 2011;90(6):777-790.

Schwartz LM, Woloshin S. Communicating uncertainties about prescription drugs to the public: a national randomized trial. Archives of Internal Medicine 2011;171(16):1463-1468.

Svensson S, Menkes DB, Lexchin J. Surrogate outcomes in clinical trials: a cautionary tale. JAMA Internal Medicine 2013;173(8):611-612.

SYMPLICITY HTN-3 Investigators. A controlled trial of renal denervation for resistant hypertension. New England Journal of Medicine 2014;370(15):1393-1401.

Temple RJ. A regulatory authority’s opinion about surrogate endpoints. In <i>Clinical Measurement in Drug Evaluation</i> (WS Nimmo and GT Tucker, eds.). John Wiley & Sons, Ltd: 1995. Pg. 3-22.

Zuckerman DM, Brown P, Nissen SE. Medical device recalls and the FDA approval process. Archives of Internal Medicine 2011;171(11):1006-1011. <i>Author Reply</i>. Archives of Internal Medicine 2011;171(21):1959-1964.