Gulf · UAE Digital
At the annual European Society for Medical Oncology Congress , a new report brought forward yet another monumental moment in cancer science: A Phase 3 study showed that the “cancer vaccine” intismeran autogene reduced the risk of recurrence or death by 42% compared with pembrolizumab (Keytruda) alone. This is the first neoantigen and mRNA therapy to have met its Phase 3 endpoint in oncology and is part of a collection of well-conducted studies showing promise for cancer therapeutic vaccines . The question is no longer whether personalized cancer vaccines can work.
The question now becomes how to accelerate development and increase access for the patients who could benefit. The evolution of cancer genetics provides valuable insights as we consider cancer vaccine access. In 1990, a University of California, Berkeley, team led by Mary-Claire King identified the location of the BRCA1 gene, the first gene linked to hereditary breast cancer.
BRCA2 was discovered five years later, and by 1996, the first commercial test for these two mutations hit the market. For the first time, people could learn whether they carried an inherited cancer risk before the disease appeared, and take potentially lifesaving action with earlier and more frequent screening, preventative surgery or prophylactic medication. But patents and price kept testing expensive for years.
Guidelines required a family history before testing. And no one in the care system clearly owned the whole process. The science arrived in the 1990s, but the delivery system never caught up.
Thirty years later, the test now costs a few hundred dollars, but it’s still not reaching most patients whose cancers could have been caught earlier or prevented altogether. If we want the coming wave of cancer vaccines to reach patients faster than BRCA testing did, there are several challenges to address now. A regulatory framework that wasn’t built for n-of-1 therapeutics Today’s regulatory pathways were designed for drugs that are identical and for devices and tests that perform the same function for every patient.
Forcing personalized vaccines into a traditional drug pathway has consequences. Regulatory requirements and market pressure together push drug manufacturers toward narrow indications, one tumor type and one disease stage at a time. Each narrow program carries its own trial costs, and those costs eventually show up in the therapy’s retail price.
There are early signs of movement. A forthcoming $200 million public-private partnership from the National Cancer Institute and Foundation for the NIH, called the Therapeutic Cancer Vaccines Initiative, aims to build clinical trial networks, data standards, and infrastructure to accelerate evidence development for personalized cancer vaccines and bring them to patients within the decade. A framework that validates the neoantigen-selection pipeline and manufacturing platform, then allows faster extension to new cancers with rigorous post-market evidence, could lower costs and widen access without lowering the bar for safety.
Artificial intelligence can also cut cost and time at nearly every step in between, from tissue analysis to manufacturing quality control. A fragmented delivery model breeds middlemen Personalized cancer vaccines are not a conventional pill or drug that can be manufactured in bulk and shipped — each vaccine is made for a specific patient’s cancer. Consider what it takes to get one patient their vaccine: A tumor sample must be collected and preserved properly at surgery.
The tumor and a blood sample are then sequenced, and scientists use complex data pipelines to identify neoantigens and design the vaccine. The vaccine must then be manufactured, shipped, and administered on schedule, followed by years of monitoring. Today, each of those steps would belong to a different organization, with its own contract, incentives, and economics, with no one accountable for the full delivery chain on something that is n-of-1 to that patient.
If we think of cancer vaccines as a drug, the cost and inconvenience of the cancer vaccine experience will drastically dilute their potential. We need to define the “product” not as what is in the vial but as an integrated end-to-end model of care that delivers a full patient experience and ensures optimal health outcomes. Value to patients doesn’t align with payers For an individual patient, preventing a cancer recurrence is priceless.
But to a payer, cancer vaccines will look like a very expensive form of prevention, not a required therapy to face a current disease. A health plan that expects members to churn within one to two years has little financial incentive to pay for an expensive “benefit” that another plan will collect. Similar to cancer screenings, access becomes forced only through patient advocacy and eventual legislation.
Coverage that is forced through legislation means that the friction to access the specific therapy or tool is not proactively addressed, let alone removed. Yet the economics favor prevention of cancer recurrences. A recurrence that becomes metastatic disease can mean years of costly immunotherapy, imaging, and hospital care.
Multiyear or outcomes-based contracts could let payers share in those savings, and Medicare, which covers many patients for life, doesn’t face the same churn problem. Employers and direct-to-patient programs may open further paths, though each raises equity concerns. Guidelines can accelerate access or stall it In practice, clinical guidelines determine what gets ordered, covered, and reimbursed.
They are also slow. Many professional guidelines still have not caught up with the technological innovations that have come to market over the last 10-15 years, such as genetic and minimal residual disease testing. Personalized therapies will make guidelines even more challenging, as guideline bodies were built to evaluate a fixed product in a defined population.
But if guidelines treat each new indication with a multiyear review, the new therapies will keep outrunning the recommendations. Guideline organizations, professional societies, and payers should agree now on how they will evaluate platform therapies: what evidence is sufficient to extend a recommendation to a related setting, and how real-world data will be used to update it quickly. At the recent STAT Summit in Boston, Gary Keblish , a New York City public school teacher, described what he called his path to a successful outcome after a high-risk melanoma diagnosis in 2019.
After surgery, he enrolled in an earlier trial of intismeran autogene, and today, he is still teaching. The results presented at ESMO are not an abstraction for patients like Gary — they are the difference between planning around the next scan and planning for next year’s curriculum. There is still much to learn about how to optimize these vaccines, which cancers they will work in, and which technologies work best.
As that research continues, we must create a system within which these vaccines can reach patients as quickly as possible. Karen E. Knudsen, Ph.D., MBA, is a scientist, health care leader, and business executive who currently serves as CEO of the Parker Institute .
She is the former CEO of American Cancer Society and its advocacy affiliate the ACS CAN, and previously served as enterprise director of the Sidney Kimmel Comprehensive Cancer Center. Othman Laraki is a technology entrepreneur, an investor, and the co-founder and CEO of Color Health . He is a longtime investor and adviser to leading companies such as Pinterest, AngelList, Slack, Instacart, Gitlab, and others.
Source: STAT
