In 2023 the FDA approved 55 novel therapeutics, the second highest count in the past 30 years.1 This included 16 oncology approvals, including 4 novel bispecific antibodies, which contain multiple sites for 2 different antigens.1 New oncology clinical trial starts are at historically high levels.2 The next-generation biopharmaceuticals pipeline is robust and full of promise for patients with cancer, with many CAR T-cell, NK-cell, and gene therapies and cancer vaccines in development.2

The future of oncology research and development holds promising opportunities as well as challenges for payers. As key stakeholders responsible for managing health care costs and ensuring quality care, payers play a crucial role in shaping the trajectory of advancement in oncology and ensuring patient access to these potentially lifesaving therapies. While these innovations bring hope, they also come with a substantial price tag. Payers face the challenge of balancing the cost of these breakthrough treatments with their long-term benefits to ensure access for the patients that need them most.

Oncology development has focused increasingly on precision medicine, tailoring treatments based on genetic and molecular profiles. This allows for more targeted and effective interventions, while potentially minimizing adverse events and optimizing outcomes. Payers see the value of these treatments. At the same time, as the number of treatment options continues to grow and treatments are entering earlier stages of cancer, payers are challenged to ensure accessibility without compromising financial sustainability. They have started to manage the oncology category more tightly: for instance, with formulary exclusions for oral oncology agents and Medicare Part B step therapies for provider-administered cancer therapies. Therapies like next-generation CAR T-cell therapy for patients who already received prior CAR T-cell therapy and personalized therapeutic vaccines for larger patient populations with early-stage disease to prevent recurrence may seem daunting to payers and employers as they struggle with how to pay for these therapies.

Collaboration among stakeholders is paramount in navigating the future of oncology research and drug development. Payers, pharmaceutical companies, health care providers, and regulatory bodies must work synergistically to streamline processes, expedite approvals, and facilitate timely access to innovative therapies. The FDA has successfully launched programs to improve the speed of oncology approvals, such as the accelerated approval process, priority reviews, and real-time oncology review. Recently passed Pre-approval Information Exchange (PIE) legislation codifies requirements for early sharing of pre-approval information with access decision-makers, which can facilitate collaboration and can improve access by increasing the speed of payer review of new therapies and new indications.

One of the downfalls of expedited FDA approval processes is that therapies are often approved based on limited evidence. This presents a challenge to payers as they try to assess the value of therapies, especially in an increasingly complex treatment landscape. As health care moves more toward value-based models, payers are increasingly interested in exploring novel reimbursement strategies: for instance, linking payments to the actual performance of the drug or exploring other innovative payment models, which may help build additional evidence. Although this system is challenging to use in oncology, we have seen examples, such as Kymriah in pediatric acute lymphoblastic leukemia, for which an invoice is sent only if a patient has a response at 1 month, and Takeda and Pfizer warranty programs that refund the patient and payer if there is no response. Payers may also be open to incorporating real-world evidence (RWE) or other health care economic information (HCEI) into decision-making processes to complement traditional clinical trial data.

The future of oncology research and drug development holds immense promise for patients; however, the sheer number and speed of innovation may be daunting to payers. As manufacturers prepare to launch novel oncology therapies, they should ensure they are collaborating with payers by providing pre-approval information exchange, exploring the potential for innovative reimbursement models, and incorporating RWE/HCEI information into value propositions to shape a more effective, accessible, and sustainable landscape for oncology care.

  1. Dunleavy K. 2023 drug approvals: after a down year, FDA signs off on a bounty of new meds, including 7 from Pfizer. Fierce Pharma. January 2, 2024.
  2. IQVIA Institute. Global oncology trends 2023 outlook to 2027. May 2023.