Oncology Insights: 2023 Roundup

January 12, 2024

In 2023, Oncology Insights covered many hot topics in cancer therapy.  With the start of a new year, lets revisit them and highlight a potentially market changing end of the year US Food and Drug Administration (FDA) approval.


In 2023, Oncology Insights covered many hot topics in cancer therapy. With the start of a new year, let’s revisit them and highlight a potentially market changing end of the year US Food and Drug Administration (FDA) approval.

BiTE Combination Therapy is Imminent

Over the last five years, the FDA has approved 8 different, bi-specific T-cell engagers (BiTEs) as single agents to treat patients with a variety of cancers. BiTEs are high cost, effective immuno-oncology designed as an “off-the-shelf” treatment option. Most agents are indicated for hematologic malignancy and are administered either intravenously or subcutaneously. With the success of single-agent treatment, studies are ongoing to improve BiTE efficacy and outcomes through combination therapy. Many of these combinations are with newer, high-cost agents. For relapsed and refractory multiple myeloma, the estimated annual WAC for the phase 3 combination of teclistamab and daratumumab hyaluronidase-fihj is $679,132, significantly exceeding the drug cost of a CAR T therapy.


CAR T Evaluation: Items to Consider

Chimeric antigen receptor T-cell (CAR T) therapy has provided unprecedented opportunities in cancer therapy and treatment options when none existed. Due to improved response rates observed in heavily pre-treated patients CAR T therapy is considered a significant advancement in cancer immunotherapy and has led to multiple FDA approvals. Research in earlier lines of therapy have proliferated the pipeline, including over 100 trials in solid tumors. These novel agents are indicated for hematologic malignancies, and most have overlapping indications.

As institutions and payers consider treatment options that provide the best value, below are some items to consider.

Due to drug cost and therapy complexity, considerations such as administrative requirements, patient access, product availability, and overlapping indications may be evaluated to determine the agent that provides the best value for a given setting.


Cell and Gene Therapy: Changing the Cancer Paradigm

Developments in cell and gene therapy (CGT) have led to remarkable advancements in cancer drug therapy and improved outcomes. As evaluation of the long-term impact of gene therapy continues, new drug classes are moving through the pipeline. If the current trends observed with new to market drugs in cancer treatment continues, the price of newly approved CGT agents will likely be high and push current boundaries.


Evolution in First-Line Cisplatin-free Bladder Cancer Therapy

On April 3, 2023, the FDA approved Padcev® (enfortumab vedotin-ejfv) in combination with Keytruda® (pembrolizumab) as 1st line for the treatment of patients with locally advanced or metastatic bladder cancer not eligible for cisplatin-containing chemotherapy. Almost 50% of patients with metastatic bladder cancer are cisplatin-ineligible. Then in On December 15, 2023, FDA approved the same combination as first line for patients with locally advanced or metastatic urothelial cancer (la/mUC). For the first time, a non-platinum based combination therapy received approval as first line therapy.  Due to the high price of these 2 agents, the cost of therapy increases significantly as does the projected budget impact.


Precision Oncology Medicine 101

In 2023 alone, nearly 50% of all newly approved FDA cancer therapy indications and 73% of oncology drugs in the pipeline utilize precision medicine. Precision medicine, or personalized medicine, matches the cancer molecular mutation to the targeted drug. It uses genes or proteins to diagnose or treat disease and grew out of a need to improve and individualize patient treatments.

Molecular testing identifies changes in single/multiple genes and the whole exome/whole genome. Cancer, due to acquired mutations, is a genome disease. The testing steps and the different types and quality of genome sequences influence molecular testing costs. Listed from most to least expensive are WGS, WES, and panel testing of a select number of genes.  As genomic testing advancements lead to greater understanding and targeted therapy discoveries come to market, the utility of precision medicine will continue to expand.


Bispecific T-cell engagers taking a BiTE out of Cancer

BiTEs target specific tumor antigens and directly activate the patient’s T-cells to kill cancer cells without needing external manipulation of T-cells required by chimeric antigen receptor (CAR) T-cell therapy, allowing for the off-the-shelf use of these drugs. Currently, the annual cost of BiTE therapy is comparable to those of CAR T drugs. CAR T has shown improved efficacy; however, ease of administration and greater access to agents may provide an advantage for BiTEs.

BiTE development continues with structural enhancements that may lead to the creation of additional novel agents targeting multiple tumor antigens to enhance response and decrease toxicity.


The Emergence of Treatments for KRAS Mutated Cancer Provide an Unmet Need

Since the discovery of the Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) mutation in the 1980s, designing therapy to target this family of mutations was elusive until the FDA approval of Lumakras® (sotorasib) and Krazati™ (adagrasib) as the second line for treating non-small cell lung cancer (NSCLC) with the KRAS G12C mutation. The estimated annual therapy cost is $240,000 for each drug.

In lung cancer, G12C is predominant in NSCLC at 13-14% and lowest in CRC at 3%. G12C mutations weren’t found, but variant G12D is predominant and makes up 30% of pancreatic cancer cases.

With ongoing studies, and the possibility of combination therapy, new treatment options may be on the horizon for the KRAS G12 mutations currently without effective pharmacologic therapy.


Highlight of an FDA end of year approval

On December 22, 2023, the FDA approved Udenyca ONBODY™ which is an on-body injector for the Neulasta® (pegfilgrastim) biosimilar UDENYCA® (pegfilgrastim-cbqv). This marks the first long-acting colony stimulating factor biosimilar to receive approval using a self-injection system. Colony stimulating factors are administered to patients decrease the incidence of infection caused by anti-cancer drug induced febrile neutropenia.

With Udenyca ONBODY™ approval, greater utilization and adoption of long-acting colony stimulating factor biosimilars may follow.


With indication expansions pending with the FDA and novel therapy movement through the research and development pipeline, 2024 is sure to bring continued transformation to cancer therapy management.


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