Most ongoing diabetes trials do not include outcomes important to patients

Jun 03, 2008

An analysis of ongoing randomized clinical trials (RCTs) in diabetes finds that only about 20 percent have as primary outcomes results that patients consider important, such as illness, pain, effect on function and death, according to a study in the June 4 issue of JAMA.

Concerns about the safety and efficacy of diabetes interventions continue, in part because RCTs have not measured their effect on patient-important outcomes as quality of life and death, according to background information in the article. "Are future diabetes trials likely to be more informative to patients and clinicians?" the researchers ask.

Gunjan Y. Gandhi, M.D., M.Sc., and M. Hassan Murad, M.D., M.P.H., of Mayo Clinic, Rochester, Minn., and colleagues examined large public clinical trial registries to systematically determine the extent to which ongoing and future registered RCTs plan to measure patient-important outcomes in patients with diabetes. The researchers identified phase 2 through 4 RCTs enrolling patients with diabetes. Of 2,019 RCTs, 1,054 proved eligible, and 50 percent of these (527) were randomly sampled, and 436 trials registered since registration became mandatory in 2004 were selected. Of these, 6 percent (24) had not started enrollment, 25 percent (109) were actively enrolling, and 69 percent (303) had completed enrollment.

Reviewers collected study characteristics and determined the outcomes measured and their type (physiological outcomes [insulin, C-peptide levels], surrogate outcomes thought to reflect an increased risk for patient-important outcomes [such as cholesterol levels, worsening kidney function], and patient-important outcomes [illness, pain, function and death]).

The researchers found that primary outcomes were patient-important outcomes in 18 percent of the RCTs, physiological and laboratory outcomes in 16 percent, and surrogate outcomes in 61 percent of the 436 RCTs. Patient-important outcomes were reported as primary or secondary outcomes in 46 percent of the RCTs.

Independent predictors of patient-important outcomes were larger trial size and longer trial duration while trials of patients with type 2 diabetes were significantly less likely to report patient-important outcomes as a primary outcome. For primary and secondary outcomes, predictors of patient-important outcomes were trial length and trial phase (phase 3 or 4 vs. phase 2), with trials of patients with type 2 diabetes significantly less likely to report patient-important outcomes.

"… the importance of our findings is that (1) without pooling, the individual diabetes trials will largely fail in providing information about the effect of interventions on patient-important outcomes; and (2) trials that are planning to measure patient-important outcomes as secondary end points need to overcome the temptation to selectively report outcomes with statistically significant results as well as to report these findings transparently and carefully. Journals also may need to publish the less-than-interesting results to enable meta-analyses of these results to produce precise enough estimates that can guide practice," the authors write.

"We believe the time has come for a broad consensus on a standard set of important outcomes for patients in diabetes trials, similar to the Outcome Measures in Rheumatology (OMERACT) initiative. The OMERACT approach allows for the uniform measurement of outcomes in RCTs with emphasis on outcomes that experts—and ultimately patients—thought would better capture the experience of rheumatological conditions."

Source: JAMA and Archives Journals

Explore further: Burnout impacts transplant surgeons (w/ Video)

add to favorites email to friend print save as pdf

Related Stories

Sapphire talk enlivens guesswork over iPhone 6

7 hours ago

Sapphire screens for the next iPhone? Sapphire is second only to diamond in hardness scratch-proof properties, used in making LEDs, missiles sensors, and on screens for luxury-tier phones. Last year, the ...

The source of the sky's X-ray glow

10 hours ago

In findings that help astrophysicists understand our corner of the galaxy, an international research team has shown that the soft X-ray glow blanketing the sky comes from both inside and outside the solar system.

Recommended for you

Key element of CPR missing from guidelines

7 hours ago

Removing the head tilt/chin lift component of rescue breaths from the latest cardiopulmonary resuscitation (CPR) guidelines could be a mistake, according to Queen's University professor Anthony Ho.

Burnout impacts transplant surgeons (w/ Video)

Jul 28, 2014

Despite saving thousands of lives yearly, nearly half of organ transplant surgeons report a low sense of personal accomplishment and 40% feel emotionally exhausted, according to a new national study on transplant surgeon ...

User comments : 0