As biological and targeted synthetic disease-modifying antirheumatic drugs (DMARDs) have proliferated, the cost of care has begun to inflate with a questionable associated benefit to patients. This “bubble” is unsustainable, according to one expert, and rheumatologists should work to help deflate it.

“By putting a high price on newly approved biological and targeted synthetic DMARDs, manufacturers implicitly promise added value in terms of health and patient experience outcomes,” wrote Prof Robert Landewé, of Amsterdam University Medical Centre, in a Viewpoint in The Lancet Rheumatology. “Do these highly priced, novel drugs in rheumatology really fulfil this promise?”

At his centre in the Netherlands, 40% of all patients have rheumatoid arthritis, psoriatic arthritis, or axial spondyloarthritis, 27% of whom are treated with biological or targeted synthetic DMARDs. In total, 44% of the centre’s entire budget was allocated for those DMARDs, meaning it went to only 11% of all patients while the other 56% was left to care for the remaining 89% of patients.

Among the ways this skewed level of spending has evolved, Prof Landewé wrote, is with the increased focus on patient satisfaction as an important outcome in itself. A patient’s perception of pain or fatigue despite treatment with a DMARD could convince a physician to change medications.

“Efficacy across biological and targeted synthetic DMARDs, as assessed in clinical trials, is reported to be roughly similar,” according to Prof Landewé. Approximately 20 such agents are now available in various countries, but he pointed out that clinical trials generally compared the novel agents against placebo rather than best possible standard-of-care – which suggests the more expensive agents may not offer substantially more value than conventional DMARDs such as methotrexate.

“Reasoning from a value-based health-care perspective, the biological and targeted synthetic DMARDs that have obtained market authorisation should in fact have a niche position: they should only be used in patients who have not responded adequately to standard-of-care treatment—the population in which these drugs might add value when administered in addition to, or after, optimal standard-of-care treatment,” he wrote. “But these DMARDs should not be prescribed to any other patients.”

Despite the perfusion of biological and synthetic DMARDs, prices have stubbornly remained very high, thanks in various part to profuse marketing of the agents, effective lobbying from pharmaceutical companies, regulatory uncoupling of drug approval and pricing and reimbursement, and other factors.

Some countries including the UK do regulate based on evidence-based principles of cost-effectiveness. “In the UK, a country with a publicly funded health-care system, this regulation has led to a situation in which only the patients with the most severe and active disease can be treated with biological and targeted synthetic DMARDs,” Prof Landewé wrote.

This unfortunately comes at the expense of patients with moderate disease activity, who can face poor quality of life and progression of damage. “Access regulation via cost-effectiveness analysis is apparently not a panacea for the problem of expanding DMARD costs.”

Prof Landewé offers four suggestions for rheumatologists to help begin countering the bubble of DMARD pricing. These include:

• More prudent diagnoses of chronic inflammatory diseases. “Every patient journey, which might include treatment with expensive drugs for many years, starts with a formal diagnosis.”
• A change in prescribing practices, where only patients who have not responded adequately to proper standard-of-care treatment are offered biological and targeted synthetic DMARDs.
• An increased reticence of inappropriate drug cycling, “which too often does not help the patient substantially makes health care more expensive.”
• Better communication with patients. “Take their important and informed questions seriously, and convince them of the fact that more expensive is not synonymous to added value.”

Prof Landewé reports receiving personal fees from AbbVie, Gilead, Eli Lilly, and GlaxoSmithKline, along with grants and personal fees from Galapagos, Pfizer, Novartis, and UCB.