Evidence-based medicine aims to put best evidence into practice. Another less-publicised application EBM and epidemiology is the shedding light on the best use of resources or the “biggest” health and public health issues. The National Institute for Health and Clinical Excellence (NICE) aims to judge which treatments and technologies are most “cost-effective” in order to advise policymakers of the treatments which give most “bang-for-buck”.

We are bombarded with priorities, goals and commitments. The United Nations’ Millennium Development Goals are an example of how different stakeholders can be galvanised towards a common set of targets, which may be as important as achieving the goals. Research priorities have begun to be set in the same way. The Grand Challenges in Global Health have highlighted the areas of greatest need in global health research. These are high profile initiatives with scientific financial backing from key players including the Gates Foundation and the Wellcome Trust. Both journals and funders have continual “calls for proposals”, whether it is the Lancet’s latest call for health research in China, or the strategic goals of the Wellcome Trust.

How good are these goals at picking the right priorities? At first glance, they may be wide open to multiple biases and conflicts of interest depending on the interests, both financial and scientific, of stakeholders and the goal-setters. The Delphi method tries to avoid this problem by using the “collective intelligence” of many experts in several questionnaire-led rounds. For example, this method has been used to set the Grand Challenges in Mental Health.

At the World Congress of Epidemiology last week, during a keynote speech by Ivor Rudan, I learned about the Child Health and Nutrition Research Initiative, or CHNRI method, designed to suggest the “best bets” for health research priorities on the basis of evidence and several stages of scoring of different options.

There will inevitably be some wastage in research funding but it is surely a good sign that institutions, whether local or global, want to channel resources towards areas of greatest need and potentially greatest benefit, and that we are developing better tools to set these priorities.

I’ve created a list of what I think are the top ten the most influential epidemiological studies – I’m looking for feedback to see what folk think.

Therefore, if you believe there should be something different on the list then can you post a comment. Rules are it has to be a clinical study that involves people, which excludes basic sciences. In addition the study has to have subsequently influenced the field significantly.

1954
Doll Richard, Bradford Hilly A (June 26, 1954). "The mortality of doctors in relation to their smoking habits. A preliminary report". British Medical Journal 1 (4877): 1451–55. PMID 13160495

1994
Antiplatelet Trialists' Collaboration. Collaborative overview of randomised trials of antiplatelet therapy--I: Prevention of death, myocardial infarction, and stroke by prolonged antiplatelet therapy in various categories of patients BMJ. 1994 Jan 8;308(6921):81-106. PMID 8298418

1989
Cardiac Arrhythmia Suppression Trial (CAST) Investigators. Preliminary report: effect of encainide and flecainide on mortality in a randomized trial of arrhythmia suppression after myocardial infarction. N Engl J Med. 1989;321:406-412. PMID 2473403

1948
MRC Streptomycin in Tuberculosis Trials Committee. Streptomycin treatment of pulmonary tuberculosis. BMJ. 1948;ii:769–783. James Lind summary

1954
Thomas Francis, Robert Korn, et al. "An Evaluation of the the 1954 Poliomyelitis Vaccine Trials." American Journal of Public Health 45 (1955), 50 page supplement with a 63 page appendix. The salk vaccine trials

1994
Randomized trial of cholesterol lowering in 4444 patients with coronary heart disease: The Scandinavian Simvastatin Survival Study Lancet 1994; 344 1383-1389 PMID 7968073

1998 (now retracted)
The Editors Of The Lancet (February 2010). "Retraction--Ileal-lymphoid-nodular hyperplasia, non-specific colitis, and pervasive developmental disorder in children". Lancet 375 (9713): 445. doi:10.1016/S0140-6736(10)60175-4. PMID 20137807.

1976
Bonadonna G, Brusamolino E, Valagussa P, Veronesi U. Adjuvant study with combination chemotherapy in operable breast cancer. Proc Am Assoc Cancer Res Am Soc Clin Oncol 1975;16:254-and N Engl J Med. 1976 Feb 19;294(8):405-10. PMID 1246307

1950
Thomas R. Dawber, M.D., Gilcin F. Meadors, M.D., M.P.H., and Felix E. Moore, Jr., National Heart Institute, National Institutes of Health, Public Health Service, Federal Security Agensy, Washington, D. C., Epidemiological Approaches to Heart Disease: The Framingham Study Presented at a Joint Session of the Epidemiology, Health Officers, Medical Care, and Statistics Sections of the American Public Health Association, at the Seventy-eighth Annual Meeting in St. Louis, Mo., November 3, 1950.
Framingham Heart Study

1855
On the Mode of Communication of Cholera by John Snow, M.D.
London: John Churchill, New Burlington Street, England, 1855 On the Mode of communication of cholera

Look forward to seeing what you think
you can post it to me on twitter
@cebmblog

Cheers Carl

I see epidemiology as the study of health and disease in populations. It can be observational (e.g. cohort studies) or experimental (e.g. clinical trials). Last week, I blogged about the increase in research funding and how more “bang-for-buck” may be expected from public- and private-funded research in hard economic times. Moreover, the best kind of health research is the kind that influences practice, or even better, translates into the policy sphere. However, shortcomings in design of research on the one hand, and clinical practice and policy on the other, mean that much epidemiology is not used or useful.

Clinical trials attempt to tell us whether a drug has a benefit in a particular clinical setting. Cost-effectiveness studies go further, allowing us to assess the relative cost of a drug versus the benefit gained (often by quality-adjusted life-years, QALYs). This epidemiologic research is vital because drugs make up a huge proportion of healthcare spending, for both the individual and the health system. Amazingly, there is no system in the NHS (or for that matter in the world) for directly correlating the amount of money spent or profit made by a certain drug with the health effects of that drug. The pharmaceutical industry has enjoyed the unique status among other manufacturing industries where sale of its products is not determined by their value. This aspect may contribute to the many conflicts of interest, scope for manipulation and lack of clinical data.

There have been many calls for changes in physician-pharma relationships and in the way pharma deals with its intellectual property rights, but few calls for change in the way pharma interacts with health systems. Well that is about to change. The UK Department for Health this week ended a 3 month consultation about value-based pricing (VBP). The government is hoping to roll out this scheme in 2014 and plans to incentivise innovation in drug research by rewarding pharma companies based on the “value” of their products.

A non-profit organisation, Incentives for Global Health, to which I am a medical advisor, is promoting a similar proposal on a global scale: the Health Impact Fund. This global fund aims to incentivise drug development and distribution based on the health impact of the drugs.

Both value-based pricing and the Health Impact Fund must face scrutiny, improvement and testing. However, not only do they both represent opportunities to change the way in which epidemiologic data is collected and used in this research area, they have the potential to radically improve the way in which health systems use limited resources on the expensive resources which are drugs. Have a look at my podcast on IDEAS-LAB about value-based pricing