Using All Study Methods to See a Drug's Value

Jan 15, 2014
As the cost of specialty medications increases, observational studies supplement traditional clinical trials to help determine a therapy's real value.

The rising use and associated cost of specialty medications for complex, often life-threatening diseases has been the topic of discussion on this blog and many other forums. Our research shows that it is foremost among healthcare-related issues for plan sponsors as well. 

The concern about specialty medication cost is understandable and justified. These medications are expensive – from $1,000 to more than $50,000 per treatment. Treatment regimens for conditions such as cancer can run hundreds of thousands of dollars per year per patient.

Despite the high cost of these medications, some patients may not experience significant improvement in either their clinical outcomes or their health-related quality of life. As the number of these high-cost medications increase, so too, is the focus on what is the “value” of these drugs, as this article in Managed Care Magazine points out.

Bridging the Gap

Traditional randomized controlled trials to establish the efficacy and safety of pharmaceuticals and biological products are good for just that – determining whether a drug works and whether it is safe. However, alone, these methods are unable to provide sufficient evidence to demonstrate the value of the drug.

As a result, real-world observational studies have become increasingly important for bridging the results of randomized trials to the real world, and deriving important information about the value of a therapy. United BioSource Corporation, an Express Scripts subsidiary, conducts observational research in virtually every therapeutic area. Its findings have been published in leading journals and presented at many conferences. Our research enables healthcare providers and other stakeholders to better judge the value that medications, diagnostic tools and other healthcare products and services offer.

Why Clinical Studies Aren’t Enough

Clinical trials for drugs and biologics being tested for FDA approval are appropriately done under controlled settings. The physicians are generally in academic settings or clinical research sites; the participating patients tend not to have many concurrent medical conditions or be at risk of polypharmacy. This means that the patient population is often not representative of who will be prescribed the medication once it is approved and reaches the market. Additionally, the number of patients evaluated in controlled trials is relatively small, so that studies involving more patients followed over longer time periods also are needed.

The Importance of Observational Studies

Real-world observational studies can tell us a lot more about the presentation and course of disease as it is affected by actual diagnostic and treatment patterns. Two commonly used study methods are retrospective observational studies and prospective observational studies.

Retrospective Observational Studies: These studies use data obtained from either medical records or administrative claims data, and can tell us much about the association between use of different therapies and their key clinical outcomes and costs. They also can show us how important adherence with therapy is to improving clinical outcomes and avoiding the costs associated with suboptimal dosing.

Prospective Observational Studies: These studies allow the researcher to collect even more data than are contained in medical records or claims data. When compared with ROS, POS can offer more insight into clinical detail, allowing us to better define subcohorts who may respond differently (positively or negatively) to the therapy. POS can also include patient-centered outcomes, and data collected from patients as well as their caregivers. This increases the quantity and quality of data available to assess benefit versus risk, and ultimately value – both overall and in patient subgroups.

Randomized controlled trials and observational studies offer different views of medical conditions, treatments, patients and outcomes. These different types of research are complementary to each other – each with strengths and limitations – and when used in parallel, can help everyone involved make better decisions based on sound research and actionable data that lead to healthier outcomes.

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