OptumRx researchers are highlighting three more drug products that payers should be keeping an eye on in 2020.
Experts said in the pharmacy benefit manager’s second-quarter drug pipeline report echoed expectations from the first quarter that orphan drugs will be a major trend to watch as the year continues. Sumit Dutta, M.D., chief medical officer at OptumRx, wrote in the report these drugs will likely account for close to 40% of Food and Drug Administration approvals this year.
Dutta said Optum is seeing more drug manufacturers jump into developing orphan drug products, which are generally considered less appealing as their market—and thus financial value—is more limited.
“What is new is that we now are starting to see the development of orphan drugs become more competitive, increasing the potential for reduced costs and broader patient accessibility,” he wrote.
In 2018, the FDA approved more orphan drugs than non-orphan drugs for the first time. Optum’s first-quarter report also noted that these products are often pricey, as they target specific conditions. On average, orphan drugs cost $147,000 or more per year.
Of the three products highlighted in the second-quarter report, two are orphan drugs. Here’s more on what Optum’s analysts think payers need to know:
If the FDA gives risdiplam a thumbs-up, it would become the first oral therapy for spinal muscular atrophy (SMA), a rare group of severe neuromuscular disorders. SMA is one of the most common genetic causes for infant mortality and affects about 1 in 11,000 babies.
There are only two treatments for SMA that are currently approved by the FDA, meaning there’s a significant unmet need for therapies, particularly oral medications, Optum said. The other treatments available are Spinraza, which requires repeated spinal injections, and gene therapy Zolgensma, the world’s most expensive drug.
Risdiplam would be administered orally once a day, which would likely draw significant interest from patients and their families, according to the report.
“Practically speaking, the competitive advantage for risdiplam will rest mainly in its oral route of administration, and perhaps, a lower cost,” according to the report.
The analysts did caution that risdiplam is still in clinical trials and while results are promising, long-term outcomes associated with the drug are unclear.
Viltolarsen is in development to treat Duchenne muscular dystrophy (DMD), a rare genetic disease that impacts young boys. There is a large unmet need for drugs to treat DMD, Optum said in the report, as it's linked with significant sickness and death.
About 6,000 people in the U.S. have this disease, according to the report.
Vitolarsen is an “exon-skipping” drug that “short circuits” the genetic mutations that cause DMD. If approved, it would be the third such drug for the disease, and the second targeting a specific mutation that affects about 8% of those with DMD.
The drug has only been tested in small sample sizes, and there are limited safety data available, according to the report.
Trodelvy, the brand name of an antibody-drug conjugate (ADC) therapy aimed at metastatic triple-negative breast cancer, was approved in April.
Triple-negative breast cancers test negative for the three most common causes of cancer and are thus untreatable by many front-line therapies, though they are treatable by chemotherapy, according to the report. ADC products like Trodelvy combine genetically engineered antibodies and traditional chemotherapy drugs into one intravenous therapy.
Optum is highlighting Trodelvy as it expects ADC medications to be a trend to monitor in the near future, because they could be applied to conditions outside of oncology, according to the report.
“We can think of ADCs as a refinement or extension of precision medicine, which aims at maximizing therapeutic benefits while minimizing undesired side effects for an individual patient,” the researchers wrote. “As the field advances, we can look for new conjugate ‘payloads’ that will go far beyond hunting cancer cells.”
“Various manufacturers are exploring how to leverage the ADC approach to produce vaccines, radiological treatments, immunosuppressive, cardiovascular and more,” they wrote.