In the current landscape of high costs of cancer treatment, many FDA and EMA approved cancer drugs entered the market based on surrogate end-points, without evidence of benefit on overall survival or quality of life, as reported by clinical studies analyzing cancer drug approval between 2008-2013 : A cost-benefit Analysis of Cancer Drugs.
Even though the efficacy of new cancer drugs is only improving marginally, the costs are sky-rocketing like never before. There is a growing call to action for the regulatory bodies to raise the scientific threshold for approving new cancer drugs which are currently able to show some arbitrary proof of efficacy and enter the market without any real evidence that the drug actually will benefit the patient by improving survival and quality of life metrics. There are newer regulatory pathways created by FDA, such as breakthrough designation, fast-track or accelerated pathways, to get drugs for life-threatening or rare diseases to the market more rapidly based on surrogate endpoints; but there are subsequent studies mandated to show proof of efficacy. A 2009 government accountability office (GAO) report criticized the US FDA for failing to enforce postmarketing study commitments for drugs approved on surrogate endpoints (https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(09)61932-2/fulltext). Thus today, based on analysis of approved drugs in the last decade, there is a growing concern about putting overpriced, toxic drugs in the physician’s toolkit that do not improve overall survival.
Survival Benefit of Approved Cancer Drugs
There are two such studies, one looking at drugs approved between 2008-2012 by US FDA (Kim and Prasad, JAMA Intern Med., 2015) and between 2009-2013 by EMA (European Medical agency) (Davis C et al, BMJ., 2017), both highlighting the above issue. The FDA analysis reported that 36 of 54 (67%) of cancer drug approvals were based on surrogate endpoints such as tumor size reduction or days a patient remains disease free (progression free survival). After 4.4 years of follow-up for these FDA approved cancer drugs, only 5 of the 36 (14%) approved showed improved overall survival, while 31 (86%) of these had either failed or lacked any data on survival impact. For the EMA analysis of cancer drugs approved between 2009-2013, there were 48 drugs approved to go to market for 68 cancer indications and only 35 (51%) of these had shown improvement in survival or quality of life. The survival benefit and clinical meaningfulness of these drugs was judged using an ESMO-MCBS (European Society for Medical Oncology Magnitude of Clinical Benefit Scale) scale, which is a standardized approach used to assess the magnitude of clinical value and validity of cancer drugs. What is even more troubling is that despite the questionable efficacy of many of these approved cancer drugs on the market, their costs continue to remain outrageously high.
A specific example of this is the drug Regorafenib which is prescribed to treat late stages of colorectal cancer, a cancer of the colon or rectum that is the third most common cancer in the United States (American Cancer Society). Regorafenib was given a grade of 1 by the ESMO-MCBS tool which means that it has almost nonexistent clinical benefits or benefits to one’s quality of life (Davis C et al, BMJ., 2017). In addition, this drug is very cost ineffective with exorbitant costs and minuscule clinical benefit (Cho SK et al, Clin Colorectal Cancer., 2018). And yet, it was launched in the market as a ‘breakthrough’ medicine for late stage colorectal cancer.
Essentially, this blog is intended to make patients and their loved ones aware of the ground realities of cancer drugs and to urge them to do a cost-benefit analysis, consider all of their treatment options and make a judicious choice rather than blindly following the current market recommended newer and more expensive options.
Cancer patients often have to deal with different chemotherapy side effects which affect their quality of life and look out for alternative therapies for cancer. Taking the right nutrition and supplements based on scientific considerations (avoiding guesswork and random selection) is the best natural remedy for cancer and treatment related side-effects.