It is crazy to deny terminal patients access to possibly life-saving drugs for fear of offending the Statistics gods!
Whether for-profit or not, insurance companies and the pharmaceutical industries are weighing in on experimental therapies; we have to be sure that reasonable interventions are given a reasonable chance, and that reasonable costs can be factored in.
Quoting my wife who is a two-time cancer survivor and advocate:
"When people are diagnosed with cancer, their treatment should be determined by their condition, peer-reviewed guidelines, their doctors AND their own informed wishes. People are devastated when told they have cancer. Rarely are they resigned to die without a fight; they will grasp for treatment options as that represents hope. They will find another doctor or get a second opinion if that hope is taken away."
And, I add, their eyes will glaze over when they are presented with adverse side effects lists (like those new drug TV commercials regurgitate in CYA fashion) or doctor-speak 'risk:benefit ratios.'
"If there is an appropriate drug to try prolong life, if not to treat in an attempt to cure the cancer (and they are not at end-stage at the time), the insurance company and their CEO have to recognize their obligation to the patient and their family."
Note the real conflict of interest going on here: business people (even insurance or health plan administrative doctors) cannot practice medicine without a doctor:patient relationship; as such, they have no right to dictate whether a known therapy or proper research trial should be disallowed.
"Nevertheless, the unfortunate experience many of us have encountered is that it seems all they care about is their money, not the patient, in most cases. If the patient is receiving treatment and their clinician has determined it is no longer working for them and there are no alternatives, then appropriate medications should be given and totally paid for (i.e., no co-pays or deductibles) to keep them as comfortable as possible. No insurance company should make medical decisions for patients."
Sent by Harvey Frey, MD, PhD, Esq. of HARP.org; Nov 2, 2013 11:50 PM
Subject: [Dialog] How Dying Patients Get Access to Experimental Drugs
In a provocative and excruciatingly compelling NY Times piece, Dr. Sanghavi states:
"Before drugs in the United States can be prescribed by doctors and bought by patients, they must undergo three stages of rigorous clinical trials and approval by the Food and Drug Administration. This safety requirement dates back to 1962, when the sedative thalidomide was revealed to have caused thousands of birth defects. This policy has helped cause drug-development costs to skyrocket to hundreds of millions of dollars and drug approval to take up to 10 years, but it has also protected the public from untold risks and side effects." Furthermore, "Circumventing the deliberate pace of clinical trials can be dangerous."
Sanghavi, DM. "The Pills of Last Resort; How Dying Patients Get Access to Experimental Drugs." NY Times, pub. Oct. 31, 2013
Statistics is not so primitive, so rigid a science and politically locked in that it is impossible to conceive how to use these self-presented patients to learn more about the drug. Also, as a condition for getting the drug, they must agree to being studied. The doctors must find age and stage matched patients who have not asked for the drug, and use them as controls. They need to study them carefully for side-effects, just as they would in a Phase I study, and adjust doses as they might in a Phase II study.
Example of Clinical Progress Expediting the Treating of Difficult Cancers
Note the approval of Ibrance [generically, Palbociclib, a drug intended to be used in the context of FDA-approved breast-cancer treatments] in combination with letrozole was expedited when clinical research data demonstrated the treatment combination "stopped tumor growth for more than 20 months, on average, in estrogen receptor-positive, human epidermal receptor 2-negative postmenopausal women who have metastatic breast cancer and are naive to having had treatment for the advanced disease."
"Pfizer’s Advanced Breast Cancer Drug Ibrance Approved for Sale" is about Armental, posted by The Wall Street Journal (WSJ) Feb. 4, 2015
Example Where Clinical Progress is Lacking—From Fish Oil Comes Prostate Cancer?—I think Not!
A recent observational study suggests fish oil may be linked to prostate cancer, however, that study does not prove a causal relationship (i.e., there is no evidence it actually causes prostate cancer). Nevertheless, it did show, as have other studies before this one, there is an association between having higher omega-3 blood levels and finding an increased incidence of prostate cancer.
The author of a KevinMD blog on this subject, Dr. David L. Katz says it is noteworthy that:“those who went on to develop prostate cancer also had higher PSA levels at baseline, were more educated, and were more likely to have first degree relatives with prostate cancer. If any of these caused these men to increase their intake of omega-3s as a protective measure, the study could be evidence of causality opposite [emphasis added - jgk] the claimed direction: Perhaps risk for prostate cancer, real or perceived, increased intake of omega-3s, rather than the other way around.” Or, it could mean higher blood levels of omega-3s (thought of as being protective) are achieved by eating more contaminated (as with carcinogens) fish.
On the other hand, higher intake of omega-3s does seem to have value: “One meta-analysis links higher omega-3 consumption to lower risk of diabetes. Another, just out and representing almost a million women, shows an inverse association between omega-3 intake and breast cancer risk: more fish oil, less breast cancer. If omega-3 fat truly did raise prostate cancer risk but lower breast cancer risk, I guess we men dining with our wives would need to get used to saying: I certainly WON’T have what she’s having!”
The body, mind, sprit connection: does diet, physical activity, or weight control have beneficial effects on cancer survival?
"In the U.S., as of 2006, approximately 11,400,000 adults and children had cancer, and that number is expected to increase to nearly 17,000,000 by 2020." Why? and what role does diet play?
Regarding Breast Cancer:
In an attempt to answer the above question as far as diet is concerned, and specifically about soy with its estrogen-like qualities, Shu et al  completed the Shanghai Breast Cancer Survival Study; it was population-based and involved 5042 breast cancer patients. They found that "soy food consumption was significantly associated with decreased risk of death and breast cancer recurrence, with adjusted 4-year mortality rates of 10.3% and 7.4% and 4-year recurrence rates of 11.2% and 8.0%, respectively, for women in the lowest and highest quartiles of soy protein intake. This well-designed study has a number of strengths, including detailed data on treatment, clinical characteristics, recurrence, vital status, and lifestyle habits, including diet."
Ballard-Barbash R, Neuhouser ML. "Challenges in Design and Interpretation of Observational Research on Health Behaviors and Cancer Survival." JAMA. Editorial. 2009;302(22):2483-2484.
1. Horner MJ, Ries LAG, Krapcho M; et al. SEER Cancer Statistics Review, 1975-2006. Bethesda, MD: National Cancer Institute; 2009. https://seer.cancer.gov/csr/1975_2006/.
2. Shu XO, Zheng Y, Cai H; et al. Soy food intake and breast cancer survival. JAMA. 2009;302(22):2437-2443
The human side of being assaulted by cancer (or any devastating diagnosis or condition)
See: "Indignity of Cancer Therapy (and What to Do About It)." Medscape Oncology, 2015-04-14 [subscription service]
Hot Topic in Cancer Research: Genetic Cancer Predisposition
The "work by Zhang and colleagues provides the most comprehensive blueprint to date of genetic childhood-cancer predisposition. Although they have probably described only the proverbial tip of the iceberg in terms of the complexity of cancer susceptibility, their work provides firm ground on which to reconsider how to approach the pediatric patient with cancer. At a minimum, this work highlights the fact that family history alone is an unreliable guide to the likelihood of a cancer-predisposition syndrome in any patient with a newly diagnosed cancer."
Their and others' "data provocatively suggest that germline sequencing should be routinely incorporated into clinical care, given that many pediatric patients with cancer have a genetic predisposition [... and as] more survivors of childhood cancer .... have children of their own [we] need to invest now to enrich our ability to provide world-class and evidence-based cancer-predisposition counseling to families afflicted by childhood cancer. Indeed, the cancer-research community must redouble its efforts to understand the clinical consequences of germline mutations in cancer-susceptibility genes, including the lifetime penetrance of the mutations, their epistatic interactions with other susceptibility genetic alterations and environmental influences, and perhaps most important, the best approaches for counseling patients and developing efficacious prevention strategies through the continuum of infancy to old age."
John M. Maris, M.D. "Defining Why Cancer Develops in Children." N Engl J Med 12/10/2015; 373:2373-2375 DOI: 10.1056/NEJMe1513921
Referring to J. Zhang and Others. "Germline Mutations in Predisposition Genes in Pediatric Cancer." article pub. by the N Engl J Med, pub. online: November 18, 2015