In January 2020, the United States (US) Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) published Advancing Health through Innovation: New Drug Therapy Approvals 2019. This report provides a summary of a number of approvals and highlights the “game-changers” approved in 2019, solidifying the upward trend in approval volume initially seen in 2017. Compared to 2017 and 2018, in which CDER approved 46 and 59 new drugs, respectively, 48 novel agents were approved in 2019. This number does not include new and expanded uses of already approved drugs, new formulations, new dosage forms, vaccines, blood products, cellular or gene therapy, or the 10 biosimilar approvals. This far exceeds the average of 38 novel approvals per year in the past 10 years. Figure 1 outlines approvals over the past 10 years.

In 2019, the FDA continued its strategic initiatives, with additional approvals of biosimilars and generics (both first-time agents and those off-patent with limited competition), as well as a focus on the expedited approval process, particularly for rare diseases and agents supported by positive patient-reported outcomes. Notably, the FDA hosted a public meeting in April 2019 to receive input on how to tackle barriers associated with the development of treatments for rare diseases, and held another meeting to celebrate “Rare Disease Day” in February 2020. Moreover, this trend is likely to continue, with the FDA releasing several guidances on gene therapy. The new pathway for insulin approval under biologic license applications planned for March 2020 and the launch of internet-based repositories or apps to improve access to information for both providers and patients (e.g., CURE ID in 2019, antiretrovirals in 2020) also highlight strategic initiatives of the FDA.

Last year, all 48 novel drug approvals met their Prescription Drug User Fee Act (PDUFA) goal dates, cementing this as a priority for the Agency. In 2019, 42% were considered first-in-class, and 44% were approved for rare diseases (Orphan Drugs). Priority Review was granted to 58% of novel drugs, 19% received Accelerated Approval (more than doubling the rate of 7% last year), 27% were designated as Breakthrough Therapy, and 35% garnered Fast Track designation. Overall, 60% of all drug approvals in 2019 used expedited development and review methods. In addition, 90% were approved in the first review cycle, and 69% were approved in the US prior to receiving approval in other countries. A breakdown of the types of drugs approved in 2019 is illustrated in Figure 2, with approvals once again dominated by drugs in the expansive oncology spectrum.

Some of the notable 2019 approvals included new advances for the treatment of cystic fibrosis (CF), with the first triple combination therapy and expanded indications of existing CF therapy available to younger patients, and the first new medications in several years for sickle cell disease. Additionally, several approvals targeted rare conditions, such as erythropoietic protoporphyria, neuromyelitis optica spectrum disorder, tenosynovial giant cell tumor, Duchenne muscular dystrophy, and systemic sclerosis-associated interstitial lung disease. Beyond treatments for rare disease and cancer, the FDA also approved agents for more common disorders, including multiple sclerosis, novel treatments for depression, migraine and cluster headaches, epilepsy, several autoimmune conditions, age expansions for diabetes therapy, and a drug-sparing regimen and new prophylaxis option for human immunodeficiency virus-1 (HIV-1). Although down slightly from 2018, the large number of approvals from 2019 represent a continued trend by the FDA to expedite and increase the quantity of annual approvals.

The costs associated with cancer care continue to rise, and many new therapy developments are on the horizon. When looking specifically at medical benefit drug spend, which has historically gone unmanaged, oncology medications make up one-third of total per-member-per-month spend, with an average cost per claim of over $2,300 for commercial plans.¹  With estimates that overall oncology spending will reach $220-$250 billion dollars over the next five years², there is a critical need to provide better management for this spend category.

We asked Rebecca Borgert, Pharm.D., Senior Director of Clinical Oncology Product Development at Magellan Rx Management, for her thoughts on what strategies payers should consider for patient-focused, holistic oncology management.

When it comes to tried-and-true cost containment strategies, like utilization management guidelines, what innovations do you see in the oncology space that are making an impact for payers and their members?

The need to ensure patients are receiving evidence-based cancer treatment continues to be of paramount importance. In the era of precision medicine , treatments are often personalized and based on the patient’s specific genomic profile; payers want to ensure their members are receiving the best treatment for their particular cancer. Due to the extremely high cost of most cancer medications, interventions aimed at unit cost savings can improve value and decrease waste. For example, waste often occurs as part of the drug compounding process due to limited vial size availability. Compendia guidelines endorse rounding doses to within 10% of the calculated dose in order to optimize vial utilization and decrease waste. Additionally, other classes of drugs may be candidates for dose optimization strategies. These increased efficiencies can account for thousands of dollars of savings per dose while decreasing overall waste in healthcare.

While a focus on the patient is critical, there’s also the need to engage providers and provide solutions that allow for cohesive workflow. Enhanced claim edits and appropriate network/fee schedules can also be effective management strategies. At Magellan Rx, we stay up to date on the latest trends and have more than 15 years of experience in providing our customers with flexible interventions, like the ones just discussed, to manage both medical and pharmacy oncology spend. It’s important to understand that a one-size-fits-all approach will not work in today’s dynamic healthcare environment.

In 2018, the U.S. Food and Drug Administration set an all-time record with the highest number of drug approvals in the last 23 years (59 total). Several new medical pharmacy drugs were approved for oncology, including 6 biosimilars. How can payers and providers be better prepared for this evolution and ever-expanding pipeline?  

Unlike the pharmacy benefit where formulary management is an industry standard practice, formulary management under the medical benefit is a relatively underused strategy. The introduction of multiple oncology biosimilars presents a perfect opportunity to execute a medical pharmacy formulary strategy in this space.

Currently there are a number of oncology supportive care biosimilars already in the market and, most recently, we have seen the launch of two oncology therapeutic biosimilars. In late 2019 and early 2020, we anticipate the launch of several more of these FDA-approved oncology biosimilars which will create true competition in the marketplace. While discounts for biosimilars compared to their reference products are in generally only in the range of 10%-15%, due to the high cost of these therapies and their prevalent utilization, it is possible for payers to achieve large cost savings with these agents by adopting a thoughtful biosimilar formulary strategy.

At the center, we have patients and their caregivers who are trying to navigate the complex journey from diagnosis to treatment through survivorship. Is this an area of focus that can have a positive effect on outcomes and mitigate rising oncology spend?

Receiving a diagnosis of cancer often results in a total upheaval of the patient’s life as well as the lives of their family members. Providing patients with consistent support and personalized assistance can help to ensure they are able to be compliant with their prescribed treatment. This also helps patients understand the anticipated side effects of treatment and how to proactively manage those side effects, avoiding the need for unplanned acute care.

1. IQVIA. 30 May 2019. Global Oncology Trend Report 2019. https://www.iqvia.com/insights/the-iqvia-institute/reports/global-oncology-trends-2019. Accessed October 15, 2019.