Why Most Clinical Research Is Not Useful.

Practicing doctors and other health care professionals will be familiar with how little of what they find in medical journals is useful. The term “clinical research” is meant to cover all types of investigation that address questions on the treatment, prevention, diagnosis/screening, or prognosis of disease or enhancement and maintenance of health. Experimental intervention studies (clinical trials) are the major design intended to answer such questions, but observational studies may also offer relevant evidence. “Useful clinical research” means that it can lead to a favorable change in decision making (when changes in benefits, harms, cost, and any other impact are considered) either by itself or when integrated with other studies and evidence in systematic reviews, meta-analyses, decision analyses, and guidelines.

There are many millions of papers of clinical research — approximately 1 million papers from clinical trials have been published to date, along with tens of thousands of systematic reviews — but most of them are not useful. Waste across medical research (clinical or other types) has been estimated as consuming 85% of the billions spent each year (Macleod, et al., 2014). John P. A. Ioannidis (2005) has previously written about why most published research is false and how to make more of it true

Useful research is patient centered (Mullins et al., 2014). It is done to benefit patients or to preserve health and enhance wellness, not for the needs of physicians, investigators, or sponsors. Useful clinical research should be aligned with patient priorities, the utilities patients assign to different problems and outcomes, and how acceptable they find interventions over the period for which they are indicated. Proposed surrogate outcomes used in research need to closely correlate with real patient-relevant outcomes for patients in the clinic (Ioannidis JP, 2014).

There is currently a heightened interest in patient-centered research, as exemplified by the Patient-Centered Outcomes Research Institute (PCORI), which was launched in 2012 in the United States to foster research relevant to patient needs (Selby & Lipstein, 2014). Similar activities are ongoing in the United Kingdom and elsewhere. However, patients are still rarely involved in setting research priorities, despite the frequent mismatch between patient priorities and research agenda. Patients and physicians are frequently bombarded with information that tries to convince them that surrogates or other unimportant outcomes are important—such short-cuts either have commercial benefits or facilitate fast publication and academic advancement.


Ioannidis JPA. Why Most Clinical Research Is Not Useful. PLOS Medicine | DOI:10.1371/journal.pmed.1002049 June 21, 2016


Ioannidis JP. Why most published research findings are false. PLoS Med. 2005; 2(8):e124.

Ioannidis JP. How to make more published research true. PLoS Med. 2014; 11(10):e1001747. doi: 10. 1371/journal.pmed.1001747

Macleod MR, Michie S, Roberts I, Dirnagl U, Chalmers I, et al. Biomedical research: increasing value, reducing waste. Lancet. 2014; 383(9912):101–4. doi:10.1016/S0140-6736(13)62329-6

Mullins CD, Vandigo J, Zheng Z, Wicks P. Patient-centeredness in the design of clinical trials. Value Health. 2014; 17(4):471 5. doi:10.1016/j.jval.2014.02.012

Selby JV, Lipstein SH. PCORI at 3 years–progress, lessons, and plans. N Engl J Med. 2014; 370(7):592–5. doi:10.1056/NEJMp1313061

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