
In the last decade, new therapies to treat rheumatoid arthritis (RA), a progressive and painful autoimmune disorder, have expanded patients’ treatment options and significantly improved outcomes, helping people with RA achieve disease remission, avoid disability and experience an improved quality of life. And since the approval of these therapies and biologics, clinical evidence has grown, and these treatments are demonstrating far greater benefits to patients than understood at the time of their initial approval.
According to new research from Boston Healthcare Associates, as new evidence regarding RA and RA treatments has emerged, our understanding of the value of these treatments has evolved significantly. Key findings from the research include:
- Individual patient characteristics can be important predictors of treatment response. For example, gene expression tests can identify as much as 60 percent of patients who will not respond to TNF-inhibitor therapy, a main class of biologic RA treatments. This allows providers and patients to select the appropriate treatment and helps avoid the use of ineffective medicines.
- Receiving treatment earlier in the progression of the disease can result in sustained disease remission. More recently, studies have shown that biologic therapy earlier in the treatment sequence substantially improves rates of remission and the durability of remission once achieved.
- Advances in treatment, resulting in better outcomes, have substantially changed the way we define clinical success of RA therapy. ACR20, a measurement developed in 1995, defines the treatment efficacy of a therapy as a greater than 20 percent improvement in tender and swollen joint counts. In 2008, the American College of Rheumatology updated the outcome measure to include 50 percent and 70 percent improvements, reflecting the additional value revealed by ongoing research that demonstrates that the use of biologic therapies in combination with synthetic disease-modifying anti-rheumatic drugs (DMARDs) and earlier in patients’ progression can deliver clinical benefits well beyond ACR20.
Conventional methods for assessing the value of new medical interventions capture only a point-in-time estimate of value, frequently devaluing new treatments by failing to recognize the potential benefits that emerge over time. Unlike the Institute for Clinical and Economic Review, which recently undertook an assessment of RA treatments, we need approaches to value assessments that can keep pace with changes in research and treatment options over time. Static, population-level assessments of the value of biopharmaceutical therapies that do not account for sustained remission and targeted treatment significantly undervalue the clinical and economic benefits of innovation in the treatment of RA.
As a result of the complexity of the disease and the evolving body of evidence on treatment options, patients and their physicians must have the autonomy to make the treatment decisions that best accommodate individual needs and preferences. That is why it is essential that tools to measure and assess the value of treatments, including value frameworks, capture how value evolves over time.
Read more at www.phrma.org/value-collaborative.
Lauren Neves
Lauren Neves is Vice President of Policy at PhRMA focusing on the value of cancer medicines, medication adherence, and payment and delivery reform. Prior to joining PhRMA, Lauren earned her law degree and worked as a market access and health policy consultant to health care and pharmaceutical clients.
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