Improving Outcomes Research Protocols

Pharmaceutical Commerce, Pharmaceutical Commerce - January/February 2010,

Today, the need to make better coverage and spending decisions throughout the healthcare arena is a universal goal. In addition to efforts throughout the pharma industry, there are also a number of government and academic initiatives under way to help further the cause when it comes to generating patient outcomes data.

Through its Sentinel Initiative program, FDA is planning to use data on 100 million patients to conduct all sorts of post-marketing surveillance, to proactively look for adverse events signals. “This additional FDA scrutiny has the potential to uncover some interesting trends, but it will also likely generate a lot of false-positives (because real world data are inherently noisy), and these will require follow-up studies to determine if the apparent problem is real or not,” says Tierce of IMS. “Drug companies need to be aware and prepare for this, because they will likely be held accountable for any apparent problems that arise any they could see fallout in terms of prescriber and patient perception of the product suffering until the truth can be sorted out.”

The Critical Path Initiative (C-PATH;, a consortium involving industry, government regulators, and academics, is working to identify and develop new patient-reported outcome measures and endpoints to incorporate into FDA Phase 2 and 3 clinical trials. “This is very exciting because it represents a new, cooperative model for instrument development,” says says Dennis Revicki, PhD, SVP for UBC’s HEOR Center (Bethesda, MD). “By including FDA in the development process, will increase the likelihood of developing a successful instrument.”

Working with outcomes scientists from seven institutions, the National Institutes of Health (NIH) formed the cooperative Patient-Reported Outcomes Measurement Information System (PROMIS) program (, which aims to revolutionize the way patient-reported outcome tools (such as measurement instruments for assessing physical function, fatigue experience and interference, pain impact and behavior, social function, anxiety, depression and other outcomes) are selected and carried out in clinical research and practice evaluation. The group is also working to establish a national resource for the accurate and efficient measurement of patient-reported symptoms and other health outcomes in clinical practice and develop computer adaptive testing to increase the efficiency and precision of patient- reported outcome assessments.

The Foundation for the NIH, a public-private partnership including FDA, NIH and “Distributed Partners,” such as SDI, are conducting a major two-year project, partly funded by participating pharmaceutical manufacturers. According to the group, the project “will assess the feasibility and utility of using ‘observational’ healthcare data, chiefly healthcare claims and electronic patients, to identify and evaluate associations between drugs and health-related outcomes.”

Another industry-academia-government consortium, called the Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials (IMMPACT;, is working to improve the design, execution and interpretation of clinical trials for treatments related to acute and chronic pain.

Another public-private initiative, spearheaded by UBC, is the EXACT-PRO Initiative (, whose focus is on developing a single, validated, patient-reported outcome measure to evaluate exacerbation-related outcomes of treatment in drug development trials of chronic bronchitis and COPD.

“All of these initiatives are designed to improve the quality of health-outcomes measurement either directly for assessing the effectiveness of new treatments being developed by the health products industry, or indirectly by developing and making available new and improved instruments for assessing health outcomes for a variety of research applications,” says Revicki of UBC. PC