Originally published by our sister publication Specialty Pharmacy Continuum

Fully 1,600 cancer treatments and vaccines are in development, according to speakers at an AMCP Nexus 2024 presentation, in Las Vegas. The median cost for new oncology drugs in 2023 was $298,628, they noted, adding that 72% of newly initiated trials in 2023 were for rare cancers and 82% were of potential treatments for solid tumors.

This is an exciting time for oncology therapeutics, said Kaelyn Boss, PharmD, a clinical consultant pharmacist at the University of Massachusetts Chan Medical School, in Worcester. For example, “we have the first subcutaneous checkpoint inhibitor approved,” Dr. Boss said, referring to atezolizumab (Tecentriq, Genentech). 

In September 2024, the FDA approved subcutaneous atezolizumab for all indications previously approved for its IV formulation—non-small cell lung cancer (NSCLC), small cell lung cancer, hepatocellular carcinoma, melanoma and alveolar soft part sarcoma. The new formulation “gives patients more treatment flexibility,” Dr. Boss said.

The costs of many current or potential cancer treatments, particularly one-time therapies, gave the audience pause. The answer to a Likert poll question with the prompt, “increasing number of high-cost, one-time therapies pose a significant challenge for my practice” was 3.9 of 5, indicating agreement bordering on strong agreement.

As an example, the FDA granted accelerated approval to the one-time T-cell receptor therapy afamitresgene autoleucel (Tecelra, Adaptimmune) in August, for treating unresectable or metastatic synovial sarcoma. Treatment cost is estimated at $727,000, Dr. Boss noted.

Although these cost challenges are formidable and will require creative insurance solutions and formulary management, both Dr. Boss and speaker Sita Bhatt, PharmD, BCOP, stressed that there is hope for previously challenging types of cancer.

“There’s a ton of options coming out and potentially cures down the line,” said Dr. Bhatt, a clinical pharmacy specialist at Boston Medical Center.

One example Dr. Bhatt cited, fruquintinib (Fruzaqla, Takeda), was approved by the FDA in late 2023 for people with metastatic colon cancer that has not responded to multiple lines of therapy.

‘Chatter’ About NSCLC Drug

In advanced NSCLC, ivonescimab (SMT112; Summit Therapeutics) outperformed pembrolizumab (Keytruda, Merck) in phase 3 results from the HARMONi-2 trial (AK112-303; ClinicalTrials.gov Identifier: NCT05499390), which was presented at the 2024 IASLC World Conference on Lung Cancer, in San Diego. Although ivonescimab, a programmed cell death protein 1/vascular endothelial growth factor (PD-1/VEGF) bispecific antibody, has not received regulatory approval, Dr. Bhatt noted that the results from the HARMONi-2 generated “a lot of chatter” at the conference.

Specifically, at a median follow-up of nearly nine months, the median progression-free survival (PFS) of patients treated with ivonescimab was 11.14 months compared with 5.82 months for those treated with pembrolizumab (P<0.0001). Additionally, the nine-month PFS rate with ivonescimab and pembrolizumab was 56% (95% CI, 47%-64%) and 40% (95% CI, 32%-48%), respectively.

“This is the first randomized phase 3 study to demonstrate a clinically significant improvement in efficacy with a novel [PD-1/VEGF] drug compared with pembrolizumab in advanced NSCLC,” Caicun Zhou, MD, PhD, lead author of the study and the director of the Department of Oncology at Shanghai Pulmonary Hospital, said while presenting the data at the conference.

High-risk neuroblastoma, another challenging condition, also has at least one noteworthy drug in development, according to Dr. Bhatt. In late 2023, the FDA approved eflornithine (Iwilfin, USWM) to reduce the risk for relapse in adult or pediatric patients with high-risk neuroblastoma. The FDA granted both breakthrough and orphan designations for the drug, partly because neuroblastoma is a rare form of cancer.

That rarity makes clinical trial recruitment difficult, so in this case the FDA did not require control and treatment arms to be in the same trial. Instead, the treatment data in the trial were compared with those derived from controls from previous clinical trials, a study design the FDA allows in exigent circumstances.

Dr. Bhatt noted that approval based on an external control is new in oncology, and hopes that this approach will be used again to spur study of treatments for other rare cancers.

The speakers reported no relevant financial disclosures. The session was supported by grants from Coherus Biosciences and Janssen Biotech.