Payers better be prepared for orphan drugs.
The Food and Drug Administration (FDA) will evaluate more than 150 new approvals this year, OptumRx officials said in their latest quarterly report on the drug pipeline.
Already, there are 64 applications submitted to the agency with likely approval in 2020, the pharmacy benefit manager said. At least 11 of those drugs are set to be “blockbuster” products with over $1 billion in U.S. sales, Optum said in the report.
Optum has seen similar development activities over the past several years.
“It’s a little more than just an aberration—seeing three years in a row of high outputs is a trend, and something we think is going to continue at least for the foreseeable future,” Bill Dreitlein, senior director of pipeline and drug surveillance at OptumRx, told FierceHealthcare.
Oncology drugs are leading the way, according to the report, with 44 products in the pipeline for potential approval in 2020. Many of these products are orphan drugs, which pose a notable challenge for payers and PBMs, as they’ll need to strike the right balance between promoting access and managing costs.
Orphan drugs, which target specific rare diseases, have proliferated in the past several years, and now make up 40% of drugs across disease categories. In 2018, the FDA approved more orphan drugs than non-orphan drugs for the first time ever.
As these drugs target certain conditions, they’re often expensive, according to the report, costing on average $147,000 or more per year. Dreitlein said it’s critical to be proactive in tracking these products.
“We have these amazing technologies, but we need to use them for the right people at the right time so that we can still afford it for everybody,” he said.
The need to manage these high-cost and very targeted products is why health plans and PBMs need to be working together to build robust and effective specialty pharmacy programs.
Watching the pipeline closely can ensure that an insurer has enough time to prepare for emerging therapies, or to respond if a high-cost product goes through the FDA’s rapid approval process.
The report also flags three specific drug classes or treatments that payers should be monitoring in the early part of 2020.
1. Bempedoic acid
Bempedoic acid is a new—and potentially costly—class of drugs for patients with high cholesterol.
Dreitlein said that bempedoic acid is an interesting case, as it has a chance of being priced toward the higher end or lower end of the spectrum. As it’s an oral medication for high cholesterol, it’s likely to be quite attractive to both members and providers.
If it’s priced competitively with traditional treatments like statins, that opens the door for a new frontline option for some patients, he said. If it’s priced more highly, however, it’s likely to instead be a second- or third-line treatment option.
“If it comes in at a very low price point, it creates a very interesting and intriguing situation because of affordability,” Dreitlein said.
AR-101 is an immunotherapy designed to desensitize patients with a peanut allergy to prevent a major allergic reaction.
AR-101 is likely to rack up a hefty price tag, but is also a product that should draw significant consumer demand as managing a peanut allergy, especially for children, is quite exhaustive.
However, it’s not actually a curative treatment but instead meant to desensitize allergic patients enough that they can avoid a severe reaction to acute exposure. Patients will still be directed to carry epinephrine injectors in case of emergency, Dreitlein said.
The therapy is also administered several times for a significant cost—Optum estimates that the six-month initial dosing phase could cost between $6,000 and $8,000, with a year of maintenance costing between $4,000 and $5,000. Visits for treatment are intensive, and early dosing requires patients to come in as often as every two weeks.
“It’s not just the cost of the drug, it’s the cost on the medical side,” Dreitlein said.
3. Obeticholic acid
Obeticholic acid is a treatment that’s currently approved for a rare liver disease called primary biliary cholangitis but may be approved for the more common nonalcholic steatohepatitis, or fatty liver disease.
It's currently on the market at a significant price—about $85,000 per patient per year—because it’s an orphan drug for a rare disease. If it’s approved to treat NASH, a condition that 16 million Americans have, the manufacturer is likely to release it under another brand name in a different formulation to allow for a lower price tag.
Optum estimates that the drug would cost between $10,000 and $18,000 per patient per year to treat NASH, and would be most effective for those who can’t maintain weight loss to manage the condition.
“It sets up something called indication-specific pricing,” Dreitlein said. “That’s kind of unusual and notable.”