Regulatory Focus™ > News Articles > PCORI: Industry Likely Skewing Trial Outcomes to Detriment of Patients, Regulators

PCORI: Industry Likely Skewing Trial Outcomes to Detriment of Patients, Regulators

Posted 26 July 2012 | By

It's an age-old question: Are life science companies influencing the outcomes of clinical trials? While it may not have a clear-cut answer, a newly-formed semi-governmental agency has announced it's looking to at least partially address it by focusing on how to make clinical trials more responsive to the needs of patients-a development with potentially huge implications for the pharmaceutical and medical device industries.

The Patient-Centered Outcomes Research Institute (PCORI), established by the Patient Protection and Affordable Care Act (PPACA), is a semi-private organization formed to conduct non-binding comparative effectiveness research. The organization is funded by the federal government, but is controlled and run by a private Board of Governors.

On Monday, 23 July the group sent out a call for comments on a draft methodology report focused on how to better tie clinical research to the needs of patients-a tie it said was potentially loose under the status quo.

"A pharmaceutical company that conducts studies of its own products will normally choose whom to study, what to compare, and what outcomes to measure so as to support an application for regulatory approval and ultimately success in the marketplace," explained PCORI in the draft report. "As a result, the apparent efficacy or safety of a medical treatment may depend on who is studying it and what they have to gain or lose by the results."

A Matter of Trust

This misalignment of incentives can have negative effects for not only patients, but for regulatory agencies and industry as well, argued PCORI. "Sometimes, rather than performing a comparison to the best alternative treatment to see which is better, investigators may choose, and sometimes regulatory agencies encourage, a comparison to a placebo, or an inferior alternative."

"Sometimes investigators are less inclined to publish results when the study shows no difference," continued PCORI. "Failure to fully publish the results of research also undermines trust and can create a false impression of the effectiveness and safety of treatments." This, in turn, can hurt regulatory authorities-as well as industry-by creating a perception that research is rigged from the start, and that regulatory assessments of healthcare products are not to be trusted.

Ultimately, PCORI said it wishes to allow patients to have access to more real-world data regarding the treatments they might take, including more data about the benefit:risk profile of a product, ways to improve the outcomes of a treatment, the delivery of the product, the care of the patient while taking the product and the full range of options available to a patient.

The first step in this process: "identifying the key problems in existing research from the perspectives of the patient and of these other stakeholders," writes PCORI.

The effect of all this will be to "promote and catalyze the development of evidence that is relevant at the time and place that people and their caregivers make health decisions," it said. The agency also said it hopes to eventually propose and drive adoption of methodological standards, per its legislative mandate.

Read more:

PCORI - Draft Methodology Report: "Our Questions, Our Decisions: Standards for Patient-centered Outcomes Research"

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