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No Randomized Controlled Trial Data? Try RCD with RWE Instead

July 12, 2016
Posted by: Joe Martinez, RPh, PDE, PPC

While the demand for Comparative Effectiveness Research (CER) data is increasing and will continue to increase for the foreseeable future, managed care decision-makers need both clinical and economic data to make informed formulary and coverage decisions. If the company doesn’t provide this type of information in the form of randomized controlled trial (RCT) data, the plan payers and decision-makers will use what data is available for their decision analysis. Unfortunately, this situation can create uncertain results.

An often-overlooked source of compelling information to remedy this is the Retrospective Claims Database (RCD) analysis which utilizes real-world evidence (RWE) gathered from respected medical practices that treat patients as part of their daily practice and not with professional clinical patients. Though it has been nicknamed the poor man’s RCT, it has been known to make a positive difference. The RCD analysis may provide a wealth of valuable information to payers and decision-makers when it is executed with a thoughtful and pragmatic protocol design.

Regulatory groups around the globe (FDA, NICE, CADTH, PBAC) are recognizing the value of RCD data as a supplement to, and sometimes even in place of RCT data. (1) Potential pathways to generate acceptable clinical and medical outcomes data for a product or procedure include: literature review and analyses, meta-analyses, economic modeling, database studies, retrospective claims database and electronic medical record (EMR) reviews.

Payers are increasing their acceptance of quality RCD data to evaluate medication utilization and prescribing trends when RCT data are not available.

In addition, RCD data can be preferable to RCT data because RCD data represents a heterogeneous population with real-world comorbidities and potentially a longer duration of patient follow-up. As an added bonus to the pharma, biotech, device or medical procedure company, the associated cost and time for these retrospective analyses and reviews is usually much less and dramatically shorter.

An RCD analysis should be well designed and include consideration for:

  • What is the primary question to be answered and who is the audience (health plan, integrated delivery network, accountable care organization, etc.)?
  • Where is the impact on health outcomes and overall health resource utilization (disease-related hospitalizations, emergency room use)?
  • What type of data is available for collection – clinical and/or procedural outcomes coding?

In any RCD project, there are also limitations and nearly all can be overcome. These include:

  • Lack of complete clinical parameters – where the use of proxy measures may be appropriate when clinical data is not available.
  • Treatment and treatment selection bias – potential statistical analysis and therapy discussion may provide important insights when more intense treatment may be selected for severe patients that may bias toward a selected product or procedure.

Companies and manufacturers should consider the retrospective claims database and the medical record analysis as cost-effective and time-efficient tools to generate meaningful and compelling data for health decision-makers, especially in the absence of RCT data.


1. ISPOR Real World Task Force Report Using Real World Data for Coverage and Payment decisions: [12/11/2006] http://www.ispor.org/workpaper/RWD_TF/RWTFManuscript.pdf

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