New Program Aims to Provide Accelerated Diagnoses & Treatment to Rare Disease Patients

Yesterday, Express Scripts announced that a new Rare Conditions Care Value (RCCV) program is underway for rare disease patients with the introduction support service of Second Opinion in partnership with PinnacleCare.

The RCCV program will provide free access to Second Opinion, which provides a second opinion and expert assessment of the diagnoses and recommendations on the most effective treatment protocol from a top specialist in the field.

"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," commented Glen Stettin, MD, senior vice president of clinical, research and new solutions at Express Scripts in a recent statement.

"Getting to the right diagnosis and the right therapy is critically important for the more than 30 million Americans currently with a rare disease. Combining the benefits of PinnacleCare's Second Opinion program with the expertise and care delivered by the specialist pharmacists in our Accredo Specialty Pharmacy can make a tremendous difference in the patient's quality of life and substantially reduce wasteful spending,” he added.

Obtaining secure diagnoses and treatments will be addressed through the programs support services, which will assist in reducing the emotional, physical, and financial burdens on patients and plan sponsors. This service will help avoid, or at least, reduce misdiagnoses, waste, and inappropriate treatments, which can amount to millions of dollars in cost and affect a patient's life for years.

An observed change in diagnosis, treatment, or care in nearly 77% of engagements among patients who utilized PinnacleCare services when working in coordination with a patient's pharmacy benefit manager and medical benefit plan serve as reinforcement for the RCCV program’s need and projected success.

Additionally, personalized patient support from clinical pharmacists and nurses specializing in the care of patients with rare disease through the services offered by Accredo specialty pharmacy will enhance the RCCV program and Second Opinion service. One-to-one counseling and in-home nursing services (when a drug regimen so requires) to help patients better manage their disease and improve outcomes will be included in the support. Preparation and management assistance for the high costs of rare-condition treatments will also be provided through a variety of cost-containment tools and strategies.

The recent and significant increases in diagnosis and treatment utilization in rare diseases will also be addressed. (Serving rare disease examples include hemophilia, Huntington's disease, Gaucher's disease, acromegaly, alpha-1 deficiency, hereditary angioedema, and idiopathic pulmonary fibrosis).

The research behind the RCCV program shows statistics regarding rare diseases are on the rise, including prevalence, treatments, and costs. More specifically, an increase in the number of approved drugs for rare diseases is anticipated to rise from 46 in 2014, to 76 in 2020, and 1/3 of new FDA-approved drugs have an orphan designation.

In just the last 4 years alone, the price of rare-condition medications has also increased 54% with some drugs running a bill as much as one million dollars for a single year's treatment. Consequently, patient out-of-pocket costs for these conditions have also increased more than 41% since 2014 to an annual average of $1,790 in 2017. (This statistic does include manufacturer coupons or patient assistance programs and may continue to increase as additional pipeline therapies enter the market.)

"Rare disease trends are growing on all fronts — more patients, new drugs and higher costs," added Miles J. Varn, MD, 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."

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