Innovation Driving New Drug Development in Oncology

There are currently greater than 700 new therapies in development for people with cancer, representing a trend that is being driven by increased innovation, according to a presentation at the Asembia Specialty Pharmacy Summit 2019.

The new therapies being researched are spread across three key areas: gene/cellular therapies, precision medicine and immunotherapy, according to the presentation. Of the agents in development, one-third are geared toward specific patient populations defined by a biomarker. The impressive results brought about by this innovation coupled with improved overall survival has led to Food and Drug Administration (FDA) approvals based on early phase clinical trial data, rather than the large phase 3 results that traditionally led to a regulatory decision.

“The movement through the approval pathway has been incredible,” said co-presenter Cheryl Allen, 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 that we’re monitoring that are progressing rapidly, and we’re seeing offerings that are coming to market that are improving overall survival.”

Chimeric antigen receptor (CAR)-modified T cell therapies have recently reached the market, representing a milestone in innovation. These therapies involve extensive manufacturing, as they require immune cells to be extracted from the patient and genetically modified. As such, they carry high price tags, especially when considering side effect management and hospitalization costs, Allen noted.

The next generation of CAR-T cell therapies in development, namely lisocabtagene maraleucel (JCAR017) and bb2121, could potentially ease some of these costs by allowing for outpatient administration and fewer associated supportive care medications, suggested Allen.

As a frontrunner in the recent oncology expansion, immunotherapy has come to define the innovation seen 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 the 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.

“If you look at these therapies, the thing I would emphasize is that while 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,” said co-presenter Florencio Calderon, vice president of Managed Care for Diplomat Pharmacy.

Precision medicine involves matching treatment to an individual's genetic makeup and represents the final area of innovation driving growth in oncology. The recent approval of Vitrakvi (larotrectinib) for patients with any type of solid tumor expressing an NTRK gene fusion highlights this trend, Allen noted. The approval of Balversa (erdafitinib) 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.

With increased innovation comes increased costs, however. Spending on cancer medications and supportive care in the United States reached $50 billion in 2017, with estimates predicting continued growth, said Allen. This amount represents a 50% increase over spending in 2012, she noted, and will continue to climb rapidly toward $100 billion by 2020. New payment models and added awareness are needed to address the growing costs, the presenters said.

“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 Calderon. “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.”