Watching the Drug Pipeline

Last year, the United States Food and Drug Administration (FDA) broke records with their list of new drug approvals-an astounding 59 new molecular entities (NMEs) and biologics. Included in that total were 34 products with an orphan drug designation and 19 first-in-class therapies.

David Calabrese, RPH, MHP, senior vice president and chief pharmacy officer of OptumRx, a pharmacy benefits firm that provides pharmacy care services for more than 65 million people, says this number is significant.

He says that what makes this year different from other years is a “shift from ‘me too’ therapies towards treatment options that:

• Approach conditions in different ways, such as novel mechanisms of action;

• Target unmet needs, such as orphan drugs; and

• Leverage advancements in technology to change the paradigm of pharmacotherapy, such as gene therapy.”

He adds that “we are also increasingly seeing drug companies go after disease states where there is no competition and opportunity to set higher drug prices.”

Related article: Seven Pharmacy Questions to Consider for 2020 and Beyond

When specialty pharmacy stakeholders, including providers, consultants, health plans, and PBMs were asked to identify which newly approved pharmaceuticals they were most excited about in the recent Managed Healthcare Executive’sManaged Care Pharmacy Survey, the majority (27.4%) selected Epidiolex, a first-in-class cannabinoid-based product for the treatment of rare-and severe-forms of epilepsy disorder in children.

This was closely followed by the new product, Erleada, with 26.2% of survey respondents saying they were excited about this once-daily oral treatment for non-metastatic castration-resistant prostate cancer. Aimovig, a first-in-class CGRP antagonist designed for migraine prevention (22.6%) and Biktarvey, a complete regimen for treatment of HIV-1 infection (15.2%) also received their share of votes.

“Responses here clearly reflect the strong desire amongst our healthcare colleagues for manufacturers to focus their research and development efforts in bringing about new products that represent true innovation in patient care versus small, incremental clinical advancements and/or more ‘me too’ type therapies,” said Calabrese.

In the survey, respondents also identified pharmaceutical products in the drug pipeline that they are most excited to see receive approval, 62.7% majority chose crenezumab, a monoclonal antibody that binds to amyloid-beta proteins to prevent and break up the aggregation of amyloid-beta plaques seen in Alzheimer’s disease, as the one to watch. Crenezumab was trailed by Bempedoic, a first-in-class non-statin oral therapy to reduce LDC cholesterol (16.8%) and AVXS-101, a gene replacement therapy to treat spinal muscular atrophy (11.8%).

The interest in crenezumab does not come as a shock to anyone in the specialty pharmacy industry. To date, there are only two types of FDA-approved medications for the treatment of this debilitating and costly neurodegenerative disorder: cholinesterase inhibitors like Aricept and Exelon and memantine. But while these drugs can help manage the symptoms of Alzheimer’s disease, they do not deal with the underlying pathology.

“The overwhelming response here in favor of crenezumab is not surprising,” said Calabrese. “For decades, we have struggled as an industry in identifying and bringing forth meaningful advancements in the treatment of Alzheimer’s in a time of growing disease prevalence. Unfortunately, with crenezumab, those struggles continue as the manufacturer, Roche, in early February announced it would discountinue its phase 3 clinical trials as an interim analysis found the drug was unlikely to reach its primary clinical endpoint.”

Not long after, a competing manufacturer, Biogen, announced that it was also halting two late-stage studies of a similar anti-amyloid compound, aducanumab, after interim analysis showed that it was also unlikely to work as expected. Calabrese said that, across the industry, hope continues as other nonamyloid approaches make their way through clinical testing, including products that target tau proteins, which also build up and cause tangles within the brain.

As for other trends, Calabrese said he and others in the industry are closely monitoring the development of oral therapies that can help patients, especially children, minimize risk of anaphylactic response to common allergens.

“Closest to market are products for patients with peanut allergies due to significant growth in this population over the last decade or more,” he said. “These products may have significant value for patients but will also represent a management challenge for managed care pharmacy as these products will be administered chronically in oral form and will likely be covered under most pharmacy benefits.”