The head of the National Cancer Institute, Dr. Norman “Ned” Sharpless, will be temporarily appointed as acting commissioner of the Food and Drug Administration amid the news of Dr. Scott Gottlieb stepping down for personal reasons. Dr. Sharpless oversees billions in grants for research and federal funding at his current position. Time will tell if the appointment of this new commissioner or his successor will open up more dialogue regarding and addressing funding for research, expense of diagnostic and genetic testing, drug development, treatment regimens.
Cancer testing and cancer therapy continue to evolve at a rapid pace in hopes of helping as many patients as possible rid this terrible monster. Millions of people are affected by cancer and according to the International Agency for Research on Cancer, each year 18.1 million cancer new cases are diagnosed worldwide and approximately 9.6 million deaths occur from the disease. This number will only continue to rise with increased lifespan, improved diagnostics, and environmental influences. By 2030 these numbers are expected to grow to 21.7 million new cases and 13 million cancer deaths. This devastating illness continues to affect patients and their families in several ways. While these patients are struggling for their lives, at the same time many of them are struggling to pay the bills for their treatments and make ends meet. A typical cancer patient’s treatment can exceed $100,000 dollars a year with medical office bills, prescriptions, cancer treatments, and testing, in addition to the other miscellaneous items.
Billions of dollars globally are donated to cancer research yearly. However, only approximately 2% of this money will actually be used on developing a treatment that can be used in practice. In addition, much of the money that is spent on cancer research in pursuit of promising new treatments is unfortunately spent pursuing dead ends. In May 2016 the largest study of clinical development success rates showed that only about 5.1% of the treatments will even pass clinical trials. Although not all research will at first succeed, and this part of the process, what remains concerning is funding for projects that may make outlandish claims without scientific evidence to back them up.
This then raises another economic question – are eccentric assertions of the success of tests and treatments being made for financial gains for further additional funding? Recently a company participated in an interview where they have claimed to find the “cure for cancer” in one year. Although this may grab headlines, this statement is likely premature and does not have the backing yet for such a claim. Is it possible that the goal of such statements is to receive further funding for continued research and development and is this the best use of the money without more evidence? False positives and exaggerated results have reached alarming rates. There will have to be further due diligence and accountability prior to shelling out these research funds.
Lately, the development of a more personalized approach with genetic and mutation-specific testing has been implemented to determine if particular treatments will be beneficial to a patient. This precision testing has proved to be effective for certain cancers that may have germline and somatic alterations that can be targeted. In certain situations, approval of off-label drugs can be approved to treat the patient. While this can many times save a patient from unnecessary chemotherapy that may not benefit them or point them in a direction towards a regimen that will be more effective, it is unfortunately expensive to develop, run, and process and there is no guarantee that there is a treatment available that is specific to the patient’s disease.
Currently, the development of new cancer drugs that target specific pathways, molecules that interrupt signal transmission, activation/inhibition of proteins, antibody therapy, immunotherapy, and other cellular therapy have been rising. These therapies can stop cancer cells from multiplying, induce cellular destruction, or lead to a dynamic immune response to attack. Companies and organizations are investing more and more in these complex drugs that will hopefully decrease the need for older cytotoxic drugs that are designed to kill rapidly proliferating cells with little expense to the type of cell. This archaic (yet many times still effective) non-specific method may lead patients to experience severe adverse effects nausea, diarrhea, fatigue, and decreased cell counts requiring transfusions and hospitalizations.
The affordability of these new drugs that are being released raises another concern. Just because a new drug passes clinical trials doesn’t mean that it will be approved to be immediately administered, and cost plays a large part in this process. Analysis of the new cancer drug approvals by NICE shows that only about 51% of approved therapy is affordable for routine use and patients may not be benefitting from them. Ideally, physicians would be able to administer a drug that they believe would give their patient the best chance of survival with the least amount of suffering. However, this is not the case and the financial logistics concerning this can be improved just as much as the therapies we currently have to treat cancer.
At the end of the day, patients are living longer as their diagnosis is being made earlier and the treatments are improving. For good reason, money is being poured into the research and development of better tests and management. Virtually every person knows a person who has been affected by cancer, and collectively we are hopeful for continued evolution of treatment, care, and soon, a cure.
By Joshua Mansour, MD
Dr. Joshua Mansour is a board-certified hematologist/oncologist working and in the field of hematopoietic stem cell transplantation and cellular immunotherapy in Stanford, California.