Elsewhere, a different type of immunotherapy known as CAR-T therapy is also exhibiting efficacy for cancer treatment -- at a cost of $475,000 for a one-time dose.7 While CAR-T is currently only approved for limited indications, that may change soon. Drug makers are hard at work on a wide range of potential applications for this costly technology.
Another feature of cancer drugs is that they are often used in combination therapy. In the case of Keytruda, a study found that when it was used in combination with chemotherapy it could extend the lives of people with advanced lung cancer by 51% compared to chemotherapy alone.8 So in addition to the high cost of Keytruda, we also have the cost of chemotherapy, which, while much less expensive, is still not cheap.
Biomarkers and targeted therapies
One long-established practice is to define cancers primarily by the area of the body in which they originate (i.e. lung cancer). This is now evolving as we increasingly sub-classify cancers according to their underlying biological or genetic factors.8
Improvements in genetic sequencing technologies are revealing a great deal of diversity between subpopulations of cancer cells within individual patients.9 Accordingly, the use of biomarkers is helping to redefine cancer more precisely, and leading to more targeted therapies and changes in the way drugs are developed.10 This has implications for both patients and payers.
Targeted therapies work because oncologists now can search for biomarkers in a person by biopsy, blood, or bodily fluids to get a better sense of the cancer they are seeking to treat. For example, about one in five breast cancers involve a gene mutation that makes an excess of the HER2 protein, which promotes the growth of cancer cells. Testing for that mutation can help determine treatment options that specifically target HER2, which are very effective.11 In fact, many insurers require such a test prior to treatment.11 Yet while the advantage to more precise profiling is more precise treatment, the disadvantage is a more fragmented market for cancer drugs.
We can see the effect of increased precision in the way it impacts how drugs are approved and priced. As the market for cancer is further subdivided, many of the drugs being developed are targeted toward much smaller populations. For example, of the 14 new active substances launched in the U.S. for oncology in 2017, 10 received Orphan Drug designation from the FDA.1 Orphan drugs are those that are aimed at diseases with fewer than 200,000 affected persons.12 We know that diseases with very low numbers of potential patients correlate strongly with high treatment costs.
In addition to Orphan designation, most of the new drugs approved in 2017 received the Breakthrough Therapy designation, which is designed to speed up the FDA approval process.1 While these expedited processes do get therapies to patients faster, it also means that many of these drugs are approved with limited evidence for their efficacy.13
This trend of expedited drug approvals can have real world consequences. In April of 2019, drug maker Eli Lilly said it was withdrawing its drug Lartruvo® (olaratumab) from the market.14 Designed to treat soft tissue sarcoma, a rare type of cancer impacting the connective tissue of the body, Lartruvo was approved in 2016 based on data from a single clinical trial involving 133 patients. Eli Lilly decided to pull the drug after a Phase 3 clinical trial demonstrated that Lartruvo did not improve patient survival compared with controls.11
While the case of Lartruvo is exceptional, it does illustrate the importance of prudence as costly new drugs enter the marketplace. Perhaps a more subtle threat from the flood of new cancer treatments is that they tend to drive out perfectly good existing treatments.
A recent study concluded that FDA approval doesn’t automatically make a new drug better than the treatments doctors are currently using.15 “Although anticancer drug approvals are increasing, a proportion of these drugs are reaching the market without proven superiority to what is considered the standard of care at the time of patient enrollment in pivotal trials,” the study states.
With these considerations in mind, what does the future of oncology treatment hold? One certainty is that the number of available oncology treatments will continue to grow. There are currently more than 700 oncology drugs in late stages of development.1 This number represents a 60% increase from a decade ago, as cancer drugs continue to represent a larger part of the overall drug pipeline.