Oncology Innovation Fuels Rapid Pipeline Growth

The oncology drug pipeline has experienced rapid growth over the past decade, driven by innovations in cell therapies, immunotherapy, and precision medicine, according to a specialty pipeline update presentation at the Asembia Specialty Pharmacy Summit 2019.

“The movement through the approval pathway has been incredible,” said co-presenter Cheryl Allen, MBA, BSPharm, RPh, vice president of industry relations at Diplomat Pharmacy. “Seven of the 14 drugs that were approved in oncology in 2017 were approved on phase 1/2 or phase 2 data. We have breakthrough data we’re monitoring that are progressing rapidly, and we’re seeing offerings that are coming to market that are improving overall survival.”

Spending on cancer medications and supportive care in the United States reached $50 billion in 2017, with estimates that predict continued growth into the future, according to Allen. This amount represents a 50% increase over spending in 2012, she noted, and will continue to climb rapidly toward $100 billion by 2020. This increase is driven by innovation and the launch of new medications, with the top oncology drugs comprising 80% of the total spend. There are currently more than 700 late-stage agents in the drug development pipeline, according to Allen. Of these treatments, one-third are geared toward specific patient populations defined by a biomarker, representing a trend toward increasingly targeted groups.

“Payers need to be attuned to how fast these approvals are coming and start planning effectively before these products make it to the market,” said co-presenter Florencio Calderon, PharmD, BCPS, vice president of managed care for Diplomat Pharmacy. “We’re seeing a change in how payers are looking at a partnership with providers. We’ve heard more about value-based contracting, but we’re seeing more integrated benefit management and focus on the total cost of care.”

Chimeric antigen receptor (CAR)-modified T-cell therapies recently reached the market, representing a milestone in oncology drug development. However, these therapies also carry high price tags, especially when considering the full spectrum of patient care, Allen noted. The next generation of CAR T-cell therapies in development, specifically lisocabtagene maraleucel (JCAR017) and bb2121, could ease some of these costs by allowing for outpatient administration and lower use of supportive care medications, Allen said.

As a frontrunner in the continued oncology expansion, immunotherapy has come to define innovation in the cancer pipeline. The blockade of the immune checkpoints PD-1, PD-L1, and CTLA-4 has led to unprecedented success across several types of cancer, even earning a tumor-agnostic approval for those with microsatellite instability—high tumors. With immunotherapies now firmly established across several indications, the next step in the development pipeline is to explore ways to optimize their use. Molecular characterizations could represent a method for building on this success, according to the panel.

“If you look at these therapies, the thing I would emphasize is that [although] we’re seeing good results, these are accompanied with opportunities for next-generation sequencing to look not only at biomarkers that show the efficacy of these treatments but also opportunities for these treatments to be almost the backbones for other treatments,” Calderon said.

Precision medicine represents the final area of innovation. The recent approval of larotrectinib (Vitrakvi) for patients with any type of solid tumor expressing an NTRK gene fusion highlights this trend, Allen noted. The approval of erdafitinib (Balversa) for patients with FGFR2/3-mutated metastatic bladder cancer and the pending FDA decision for quizartinib for FLT3-ITD—mutated acute myeloid leukemia further points toward growth in biomarker-based strategies.

“We are starting to get data [on] the NCCN guidelines that are telling us where testing makes sense, to find these therapies that are going to be more effective,” Calderon said. “If you create hurdles in terms of the testing you allow, that creates a risk for inappropriate [use]. There has been more thoughtfulness about how we create more value in the current treatment paradigm."