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OptumRx’s most recent pipeline outlook for 2020 reveals key therapeutic areas in the lineup.
The pipeline outlook for 2020 is strong, with over 150 drugs that could be approved by the FDA in the next year, according to an OptumRx report.
OptumRx’s Pipeline Drug Surveillance team monitors over 1000 new drugs and indications at any given time. The report, which is released quarterly, highlights the most notable drugs being reviewed by the FDA.
Among the most anticipated drugs in the pipeline is bempedoic acid, according to the report. If approved, this new treatment will represent a new class of drugs for high cholesterol.
“Since bempedoic acid is taken by mouth, we believe it will be favored over injectables like [proprotein convertase subtilisin/kexin type 9] inhibitors, a biologic drug that significantly lowers LDL-C levels,” the report indicated. However, since there are many drugs available for the management of high cholesterol, bempedoic acid will likely to be used as a second- or third-line option, according to the report.
The first oral immunotherapy drug, AR-101, for peanut allergy was also highlighted. In a clinical trial, AR-101 reduced allergic reactions from exposure to peanuts, but did exhibit limitations such as an approximately 6 month dose escalation period. AR-101 was also associated with a higher discontinuation rate due to adverse events and a higher rate of epinephrine use.
“However, the results of the pivotal trial demonstrate that AR-101 could substantially change the outlook for many patients who have a peanut allergy by potentially reducing the risk of severe allergic reactions from accidental peanut exposure,” the report said.
A large population of patients are affected by peanut allergy; however, there are limited treatment options. Viaskin Peanut, which is being developed by DBV Technologies, is another investigational immunotherapy being reviewed by the FDA with a target action date of August 2020.
Obeticholic acid is also a highly-anticipated drug listed in the report, as it would be the first treatment for nonalcoholic steatohepatitis (NASH). The drug is already on the market for primary biliary cirrhosis, but has not yet been approved for NASH. Approval is expected in 2020 based on positive results from the REGENERATE phase 3 study showing obeticholic acid improved liver fibrosis without worsening NASH at 18 months. However, the proportion of patients experiencing resolution of NASH was no different from placebo, the report noted.
“Given the large (and growing) target population in conjunction with the high unmet treatment need, it is projected that pricing for obeticholic acid for NASH will be between $10,000 to $18,000 per patient per year,” the report said.
Top Therapeutic Areas
Oncology continues to plow ahead in the lineup as the drug class leader among potential approvals in 2020. There are a total of 44 drugs in the pipeline slated for approval in the cancer space, according to the report.
A previous report published by the IQVIA Institute also highlighted oncology as the largest therapeutic area in biopharmaceuticals due to the large number of recently-launched therapies, significant clinical benefits, and robust pipeline. Results from the IQVIA report demonstrated extensive pipeline activity in this space over the years.
As such, oncology is also one of the most expensive drug classes, and is expected to increasingly account for more health care spending. “Looking ahead, specialty pharmacy care management will play a critical role in ensuring these drugs are being used appropriately, reducing waste, improving safety, and improving health outcomes for these patients,” OptumRx said in its report.
The number of orphan drug indications also dominate the 2020 pipeline. Within oncology, roughly 70% of anticipated approvals will be high-cost orphan drugs, with new drug developments focused on narrow populations based on biomarker or genetic mutation. As a whole, orphan drugs account for approximately 40% of drugs across disease categories. In 2018 alone, the FDA approved more new molecular entities with orphan drug designation than non-orphan drugs –– and orphan drugs continue to remain a priority moving forward, according to the report.
“In 2020, it is expected this emphasis on orphan drugs will continue, while the number of orphan drugs proportionate to all drugs approved is expected to remain the same,” OptumRx indicated. In addition to oncology, orphan drug designations also make up portions of the hematology, neurology, and endocrine/metabolic spaces, the report noted.
Drugs in the neurology space came in second on the list of top drug categories following oncology, with 29 drug candidates in the pipeline for 2020, according to OptumRx. Pipeline therapies cover a variety of conditions, such as migraine, Parkinson disease, and epilepsy. More novel therapies are also being investigated to treat rare conditions, such as spinal muscular atrophy and neuromyelitis optica.
The report noted that these drugs could have a significant clinical and financial impact due to the size of the population affected or because of the high unmet need for treatment options.
The top 5 therapeutic areas in the pipeline listed in the report include:
Looking ahead, the report also pointed to a current total of 64 drugs filed with the FDA that have anticipated approval dates and 11 potential blockbusters with expected $1 billion in US sales.