Quantitative benefit–harm assessment for setting research priorities: the example of roflumilast for patients with COPD

European Medicines Agency – Human Medicines: European Public Assessment Report for Daliresp. 2011. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002398/human_med_001415.jsp&murl=menus/medi . Accessed 6 April 2015.

US Department of Health and Human Services. Food and Drug Administration. Approval package for: application number: 022522Orig1s000. 2011. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/022522Orig1s000Approv.pdf . Accessed 6 April 2015.

Chong J, Poole P, Leung B, Pn B. Phosphodiesterase 4 inhibitors for chronic obstructive pulmonary disease. Cochrane Database Syst Rev. 2013;11:CD002309.

Lone N, Oba Y. Roflumilast: a green signal is yet to come. J Thorac Dis. 2013;5:213–5.

Global Initiative for Chronic Obstructive Lung Disease. Updated 2014. Chapter 3: Therapeutic options, p. 25. http://www.goldcopd.org/Guidelines/guidelines-resources.html . Accessed 6 April 2015.

Puhan MA. Phosphodiesterase 4 inhibitors for chronic obstructive pulmonary disease. The Cochrane Library. 2011;8:ED000028.

United States Preventive Services Taskforce Grade Definitions. http://www.uspreventiveservicestaskforce.org/Page/Name/grade-definitions (accessed April 6, 2015).

Rocchi A, Miller E, Hopkins RB, Goeree R. Common Drug Review recommendations: an evidence base for expectations? Pharmacoeconomics. 2012;30:229–46.

Imberger G. Clinical guidelines and the question of uncertainty. Br J Anaesth. 2013;111:700–2.

Greenhalgh T. Less research is needed. http://blogs.plos.org/speakingofmedicine/2012/06/25/less-research-is-needed/ . Accessed 6 April 2015.

Kessler R, Glasgow RE. A proposal to speed translation of healthcare research into practice: dramatic change is needed. Am J Prev Med. 2011;40:637–44.

Krishnan JA, Lindenauer PK, Au DH, Carson SS, Lee TA, McBurnie MA, et al. Stakeholder priorities for comparative effectiveness research in chronic obstructive pulmonary disease: a workshop report. Am J Respir Crit Care Med. 2013;187:320–6.

Chalmers I, Bracken MB, Djulbegovic B, Garattini S, Grant J, Gülmezoglu M, et al. How to increase value and reduce waste when research priorities are set. Lancet. 2014;383:156–65.

Caron-Flinterman JF, Broerse JEW, Teerling J, Bunders JFG. Patients’ priorities concerning health research: the case of asthma and COPD research in the Netherlands. Health Expect. 2005;8:253–63.

Clavisi O, Bragge P, Tavender E, Turner T, Gruen RL. Effective stakeholder participation in setting research priorities using a Global Evidence Mapping approach. J Clin Epidemiol. 2013;66:496–502. e2.

National Institute for Health and Care Excellence (NICE). Research and Development. https://www.nice.org.uk/about/what-we-do/research-and-development . Accessed 6 April 2015.

Patient Centered Outcomes Research Institute. National Priorities and Research Agenda. http://www.pcori.org/content/national-priorities-and-research-agenda . Accessed 6 April 2015.

Fleurence RL, Torgerson DJ. Setting priorities for research. Health Policy. 2004;69:1–10.

Fleurence RL, Meltzer DO. Toward a science of research prioritization? The use of value of information by multidisciplinary stakeholder groups. Med Decis Making. 2013;33:460–2.

Wald HL, Leykum LK, Mattison MLP, Vasilevskis EE, Meltzer DO. Road map to a patient-centered research agenda at the intersection of hospital medicine and geriatric medicine. J Gen Intern Med. 2014;29:926–31.

Ginnelly L, Claxton K, Sculpher MJ, Golder S. Using value of information analysis to inform publicly funded research priorities. Appl Health Econ Health Policy. 2005;4:37–46.

Carlson JJ, Thariani R, Roth J, Gralow J, Henry NL, Esmail L, et al. Value-of-information analysis within a stakeholder-driven research prioritization process in a US setting: an application in cancer genomics. Med Decis Making. 2013;33:463–71.

Lozano R, Naghavi M, Foreman K, Lim S, Shibuya K, Aboyans V, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380:2095–128.

Vos T, Flaxman AD, Naghavi M, Lozano R, Michaud C, Ezzati M, et al. Years lived with disability (YLDs) for 1160 sequelae of 289 diseases and injuries 1990-2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380:2163–96.

Bill & Melinda Gates Foundation. Grant Opportunities. http://www.gatesfoundation.org/How-We-Work/General-Information/Grant-Opportunities . Accessed 6 April 2015.

Wennberg J. Tracking medicine: a researcher’s quest to understand health care. 1st ed. Oxford: Oxford University Press; 2010.

Phelps CE, Parente ST. Priority setting in medical technology and medical practice assessment. Med Care. 1990;28:703–23.

Fleurence RL. Setting priorities for research: a practical application of “payback” and expected value of information. Health Econ. 2007;16:1345–57.

Robinson KA, Saldanha IJ, McKoy NA. Development of a framework to identify research gaps from systematic reviews. J Clin Epidemiol. 2011;64:1325–30.

Li T, Vedula SS, Scherer R, Dickersin K. What comparative effectiveness research is needed? A framework for using guidelines and systematic reviews to identify evidence gaps and research priorities. Ann Intern Med. 2012;156:367–77.

Meltzer DO, Hoomans T, Chung JW, Basu A. Minimal modeling approaches to value of information analysis for health research. Med Decis Making. 2011;31:E1–22.

Yu T, Fain K, Boyd CM, Singh S, Weiss CO, Li T, et al. Benefits and harms of roflumilast in moderate to severe COPD. Thorax. 2014;69:616–22.

Guo JJ, Pandey S, Doyle J, Bian B, Lis Y, Raisch DW. A review of quantitative risk-benefit methodologies for assessing drug safety and efficacy-report of the ISPOR risk-benefit management working group. Value Health. 2010;13:657–66.

Puhan MA, Singh S, Weiss CO, Varadhan R, Boyd CM. A framework for organizing and selecting quantitative approaches for benefit-harm assessment. BMC Med Res Methodol. 2012;12:173.

Mt-Isa S, Hallgreen CE, Wang N, Callréus T, Genov G, Hirsch I, et al. Balancing benefit and risk of medicines: a systematic review and classification of available methodologies. Pharmacoepidemiol Drug Saf. 2014;23:667–78.

Gail MH, Costantino JP, Bryant J, Croyle R, Freedman L, Helzlsouer K, et al. Weighing the risks and benefits of tamoxifen. J Natl Cancer Inst. 1999;91:1829–46.

Mt-isa S, Goginsky A, Chan E, Downey G, Hallgreen CE, Hockley KS, et al. IMI-PROTECT Benefit-Risk Group Recommendations. Report recommendations for the methodology and visualisation techniques to be used in the assessment of benefit and risk of medicines. www.imi-protect.eu/documents/HughesetalRecommendationsforthemethodologyandvisualisationtechniquestobeusedintheassessmento.pdf . Accessed 6 April 2015.

Yu T, Vollenweider D, Varadhan R, Li T, Boyd C, Puhan MA. Support of personalized medicine through risk-stratified treatment recommendations – an environmental scan of clinical practice guidelines. BMC Med. 2013;11:7.

Boers M, Brooks P, Fries JF, Simon LS, Strand V, Tugwell P. A first step to assess harm and benefit in clinical trials in one scale. J Clin Epidemiol. 2010;63:627–32.

Chuang-Stein C. A new proposal for benefit-less-risk analysis in clinical trials. Control Clin Trials. 1994;15:30–43.

Martinez FJ, Calverley PMA, Goehring U-M, Brose M, Fabbri LM, Rabe KF. Effect of roflumilast on exacerbations in patients with severe chronic obstructive pulmonary disease uncontrolled by combination therapy (REACT): a multicentre randomised controlled trial. Lancet. 2015;385:857–66.

Berndt E, Cockburn I. Price indexes for clinical trial research: a feasibility study. Natl Bur Econ Res 2013, No. 18918. http://www.nber.org/papers/w18918 .