2020 could be a blockbuster year for drug approvals

2020 could go down as a record year of drug approvals, with the highest number of approvals coming in the oncology, neurology, and endocrine/metabolic infectious diseases categories. This forecast comes from OptumRx’s Drug Pipeline Insights report, which shows that over 150 new drug approvals, including new molecular entities and other approvals will be evaluated by the Food and Drug Administration this year.

Sixty-four drugs have already been filed with the FDA and have anticipated approval dates in 2020. Eleven of these drugs are potential blockbusters with sales expected to exceed $1 billion.

The OptumRx report also includes the following revelations:

Oncology will continues to be a top therapy class indicating that pharmaceutical manufacturers continue to invest heavily in this space.

Within oncology, roughly 70% of anticipated approvals in 2020 will be high cost orphan drugs; this means that new drug development is primarily focused on very narrow populations with rare subsets of cancer, as defined by a biomarker or genetic mutation.

"As previously noted, oncology is one of the most costly drug classes. Pharmaceutical companies have been successful in setting high prices for new drugs, which we expect to continue for 2020 and beyond. Looking ahead, specialty pharmacy management will play a critical role in ensuring the drug is being used appropriately, reducing waste, improving safety, and improving health outcomes for these patients."

Treatments for neurological conditions are second on the list of top drug categories and the pipeline of products treat a wide array of conditions within that class. Perhaps the most dramatic example of why this class ranks so high is the recent spinal muscular atrophy drug, Spinraza, with its current list price of $750,000 for the initial year and $375,000 per year thereafter.

The current development pipeline includes new therapies for more common conditions such as migraine, Parkinson’s disease, and epilepsy, as well as novel therapies that treat rare or ultra-rare conditions, such as spinal muscular atrophy and neuromyelitis optica.

New drugs for rare and ultra-rare conditions could have significant financial and clinical impact because of their small target populations and high unmet treatment needs. In some cases, these pipeline agents will be entering a relatively crowded marketplace. This raises the possibility for added competition and potentially lower drug prices for available treatments.

Orphan drugs make up nearly 40% of drugs across disease categories – and will continue to remain a priority. Orphan drugs treat conditions that affect relatively small numbers of people sometimes only a few thousand. These drugs are critical for patients with rare conditions where previous treatments may not exist.

In the past ten years, the number of orphan drug indications has risen dramatically. In 2017, the FDA approved 80 new orphan indications, up from 40 in 2016 and just 15 in 2010. In 2018, for the first time ever, the FDA approved more new molecular entities with orphan drug designation than non-orphan drugs.

"Given the return on investment, pharmaceutical manufacturers will continue to prioritize development of orphan drugs for conditions where there is no other alternative option. Therefore in 2020, we expect this emphasis on orphan drugs to continue, with orphan drugs representing an average of 44% of all drugs approved," the report said.

Looking across all categories, oncology has the largest proportion of drugs with an orphan drug designation. Remaining orphan drugs are diverse across the categories, including hematology, neurology and endocrine/metabolic, according to the report.