The Cost of Drugs in Cancer Care: A Story of Misalignment, With Necessary (Positive) Changes!

Andre Goy, MD

Editor-in-Chief of Oncology & Biotech News

Chairman and Director Chief of Lymphoma Director, Clinical and Translational Cancer Research John Theurer Cancer Center at Hackensack University Medical Center

There is no question that we are currently living through one of the most exciting times in medicine, particularly in oncology, as I have frequently alluded to in this column. The new drugs that have emerged continue to transform the landscape and in some cases, will likely replace standard chemotherapy. The ongoing developments include advances with novel small molecules—such as kinase inhibitors in CML and lung cancer, and BCR targeted therapies in B-cell malignancies—as well as the enormous momentum seen (finally) in immunotherapy with checkpoint inhibitors, BITE antibodies, and cellular therapies (eg, CAR-T cells), which have shown sustainable activity across multiple tumor types.

These novel treatments are often reported as “breakthrough therapies” based on their impressive activity, even in patients refractory to chemotherapy, as well as their frequently being more tolerable than cytotoxic agents and often being administered conveniently as oral agents. Given these advantages, such novel therapies are, not surprisingly, widely adopted by the entire community. Even though some of these new anticancer agents do not induce complete remission, they provide durable responses, which translate into years of clinical benefit for patients and eventually carry the potential promise of making cancer “a chronic disease.”

Over the past few years, however, given the wellrecognized unsustainable cost of healthcare—particularly in cancer (with a predicted 40% increase in incidence by 2030!)—the cost of these new therapies has come under scrutiny. Though we have become accustomed to novel therapies being more costly for over two decades, their cost has continued to increase at an estimated average of $8,500 a year per patient for the past 15 years. Some extreme recent examples illustrate the current problem, with some breakthrough therapies costing up to $150,000 to $178,000 per patient, with copays that obviously then become prohibitive, leading large groups of oncologists to complain that it has gone too far.

Additionally, the next step with these emerging therapies will naturally be to combine biological agents, with the logical rationale that it will extend clinical benefit. This implies that in some cases, such as with indolent lymphomas, we will generate regimens that will cost over half a million dollars a year (with an endpoint to treat until progression). Needless to say, this becomes unfeasible and needs to be addressed. However, a number of factors might help read through and resolve this difficult equation:

• We need to look at the big picture for every patient and the cost of other procedures or strategies to treat every case. Reducing hospitalizations and complications with an ambulatory therapy can obviously offset a significant part of the cost of novel therapies.
• The use of combinations of new biologicals might help achieve deeper responses and hopefully help shift the field to minimal residual disease (MRD) as an endpoint and potentially be able to stop treatment or customize recommendations based on therapy “needs” through molecular monitoring.
• Nowadays, the FDA approval of a drug is based on proven/suggested clinical benefit in a population with unmet need, which typically refers to a disease subtype having failed X, Y, or Z prior therapies. However, most currently approved drugs in oncology will not work in about 30% to 70% of cases. Having the ability through molecular diagnostic/stratification to offer a given therapy to the right subset of patients is crucial and could save substantial dollars in addition to sparing risk and waste of precious lifetime for these patients. This effort of “precision medicine,” though palpable, still needs a huge amount of work and a stronger commitment from all players.
• In addition, the development of smarter—ie, molecularly relevant—trials would help identify biomarkers that could guide such decisions. They would also reduce the cost of drug development, which should translate into lower drug costs after FDA approval.
• The “me-too” drugs are sometimes looked at as another company “jumping” on the bandwagon; I would argue that they are critical to competitiveness, as recently shown with the hepatitis C treatment story.

Overall, the unprecedented, soaring price of anticancer drugs (and cancer care) is not sustainable and has truly not been addressed. This leads us to ambivalent situations: amazing stories of new compounds bringing patients back to life and generating appropriately huge hope for cancer patients around the world, while at the same time, the treatment is often accompanied by prohibitive financial toxicity.

I believe that in the pharmacoeconomics complex and deeply misaligned landscape, several forces will have to come forward together to improve the status quo. Countless initiatives are trying to look at outcome of cancer treatment or define the “best” pathways to treat patients. The goal is to identify where the real impact of a given drug or regimen is—ie, which agent, regardless of cost, gives the best mileage for a given patient? At that point, it is suggested that the “cost becomes almost relative.” I would argue that physicians as main prescribers need to be part of both the debate and solution. This solution needs to capture cost and outcome—as we do in COTA—in order to maintain quality for patients, while reducing variance in management by providing the appropriate rationale (evolving when needed) to support treatment decisions. This is the best way to render physicians responsible while allowing them to remain patient-centric—isn’t that the true core of medicine? And smarter medicine is better medicine!