Expect a wave of new drug treatments to receive Food and Drug Administration attention in the coming year, especially expensive orphan drugs designed to treat patients with rare medical conditions, according to pharmacy benefits manager OptumRx.
A unit of UnitedHealth Group, OptumRx predicts in its latest Drug Pipeline Insights Report that the FDA will evaluate more than 150 new drugs for approval. Currently, 64 total drugs have been filed with the FDA and have anticipated approval dates in 2020.
Eleven drugs are potential blockbusters with an expected $1 billion in U.S. sales, according to the report.
In particular, two categories will drive the greatest number of potential new drug entrants this year:
Oncology. A total of 44 drugs in the cancer space are in the pipeline slated for approval, according to the report -- which also predicts oncology will maintain its top spot for the foreseeable future, as it involves more than 200 disease variations requiring advanced research and different nuances in treatment.
Neurology. Drugs in the neurology space were second on the list of top drug categories, with 29 candidates in the pipeline for 2020. These include new therapies for common conditions (migraine, Parkinson's disease, epilepsy) and advanced therapies for rare conditions (spinal muscular atrophy, neuromyelitis optica).
Another major trend impacting the pipeline is orphan drugs for treating rare conditions that affect less than 200,000 people in the U.S.
The number of orphan drug indications has grown significantly during the past decade, and the focus on these drugs is expected to continue in 2020 and beyond.
In 2018, for the first time ever, the FDA approved more new molecular entities with orphan drug designation than non-orphan drugs.
The number of orphan drugs proportionate to all drugs approved is expected to remain the same, with orphan drugs representing an average of 44% of all drugs approved between 2016 and 2019.
Across all categories, oncology has the largest proportion of drugs with an orphan drug designation. Approximately 70% of anticipated oncology drug approvals this year will be orphan drugs focused mainly on very narrow populations with rare subsets of cancer.
WHY THIS MATTERS
Oncology's outsized presence in the drug pipeline is important because it's one of the costliest drug classes and one in which pharmaceutical companies have been successful in setting high prices for new drugs.
Likewise, drugs for neurological conditions could have significant financial impacts because of the size of the populations affected or the high unmet need for treatment options. In some areas of neurology, such as Multiple Sclerosis, these pipeline agents will be entering a relatively crowded marketplace, creating added competition that could lead to lower drug prices for available treatments.
THE LARGER TREND
FDA approval of drugs has remained robust in recent years. For example, the FDA's Center for Drug Evaluation and Research approved 48 novel drugs in 2019, down from its record of 59 approvals in 2018, but still the third largest approval class in the past 25 years.
CDER's approval average on a five-year rolling basis is now 44 new drugs per year, which is double its 2009 low-point of 22.
A similar trend also holds for generic drugs, as the FDA approved a record 1,171 generics in fiscal year 2019. That follows a record 971 approvals in fiscal year 2018 and a record 937 approvals in fiscal year 2017.
ON THE RECORD
"Looking ahead, specialty pharmacy care management will play a critical role in ensuring (oncology) drugs are being used appropriately, reducing waste, improving safety, and improving health outcomes for these patients," according to the Drug Pipeline Insights Report, which also noted that "Similar to the oncology space, orphan drugs are expensive, costing on average $147,000 a year or more, and will require utilization and specialty pharmacy care management to reduce costs for patients and clients. Given the return on investment, pharmaceutical manufacturers continue to prioritize development of orphan drugs for conditions where there is no other alternative option."
Mark Klimek is an independent writer and editor with 20 years' experience covering financial issues, healthcare and more.