At the end of May, the pharmacy benefits manager Express Scripts announced a new program it said was aimed at “reducing the burden on patients with rare disease.”

Express Scripts unveiled its Rare Conditions Care Value program with the introduction of a support service known as Second Opinion. The program, which will begin January 1, 2019, will provide members whose plans are enrolled in Rare Conditions Care Value free access to the Second Opinion support service through a partnership with the health advisory firm PinnacleCare. PinnacleCare provides expert case review. The service offers a second opinion from a top specialist who will provide an expert assessment of the diagnosis and recommendations on the most effective treatment protocol.

Patients with a rare disease face a long diagnostic odyssey. On average, Express Scripts notes it takes 7.6 years to get a diagnosis and visits to seven different physicians. The program is intended to “reduce the emotional, physical, and financial burden on patients and plan sponsors associated with misdiagnosis, waste, and inappropriate treatments, which can cost millions of dollars and impact years of a patient’s life.” Among patients who have used PinnacleCare services, nearly 77 percent of engagements have resulted in a change of diagnosis, treatment or care, when working in coordination with a patient’s pharmacy benefit manager and medical benefit plan.

“The years spent going to different doctors, getting myriad medical tests, managing paperwork and experimenting with numerous treatments is an ordeal for patients and their health, as well as for those who love and care for them,” said Glen Stettin, senior vice president of clinical, research and new solutions at Express Scripts. “Getting to the right diagnosis and the right therapy is critically important for the more than 30 million Americans currently with a rare disease.”

Getting rare disease patients the right diagnosis and most appropriate treatments is a good thing. If the new Express Scripts program achieves that, more power to them. But it’s worth noting that Express Scripts customers are payers, such as health insurance plans. While Express Scripts extols the benefits it is providing rare disease patients with the offering, the program is designed to address the needs of payers and reflects the growing concerns payers have on containing the rising cost of caring for rare disease patients.

On its website where it pitches the new service, Express Scripts notes that the price of rare-condition medications has increased 54 percent in the last four years alone, with some drugs costing as much as one million dollars for a single year’s treatment. It also points out that one-third of new FDA-approved drugs have an orphan designation, suggesting more treatments coming to market will also represent new sources of costs to payers.

“The Rare Conditions Care Value program is addressing diseases that have recently seen significant increases in diagnosis and treatment utilization, such as hemophilia, Huntington’s disease, Gaucher’s disease, acromegaly, alpha-1 deficiency, hereditary angioedema and idiopathic pulmonary fibrosis,” the company said on its website. “Among these disease states alone, the number of newly approved drugs that can treat the disease or the symptoms associated with the disease will rise from 46 in 2014, to 76 in 2020. These treatments can be very costly.”

As an example, the company points to treatments for hereditary angioedema, a potentially life-threatening genetic condition. Treatment for one patient in a single year can cost $2.2 million.

“Rare disease trends are growing on all fronts – more patients, new drugs and higher costs,” said Miles Varn, chief medical officer of PinnacleCare. “In order to provide affordable access to these newer therapies, we need to make sure that the diagnosis is correct, and that the pharmaceutical treatment plan is based upon expert best practices. Working with Express Scripts, we’ll help patients achieve better health outcomes and ensure the care they receive is thoughtful and cost effective.”

The headline in the press release announcing the Rare Conditions Care Value program says “Express Scripts to Provide Patients with Faster Path to Rare Disease Diagnosis and Treatment,” but it’s not clear that is what they are doing. In fact, it appears they may be adding a layer to the diagnosis and treatment access. There’s nothing wrong with making sure a diagnosis and treatment are correct, especially for complex and costly-to-treat diseases. But it does seem clear that the intent here is to contain the cost of treating these patients and serve the needs of payers.

June 18, 2018
Glen Stettin, senior vice president of clinical, research and new solutions at Express Scripts