An urgent call to raise the bar in oncology.

Important breakthroughs in medical treatments have improved outcomes for patients suffering from several types of cancer. However, many oncological treatments approved by regulatory agencies are of low value and do not contribute significantly to cancer mortality reduction, but lead to unrealistic patient expectations and push even affluent societies to unsustainable health care costs. Several factors that contribute to approvals of low-value oncology treatments are addressed, including issues with clinical trials, bias in reporting, regulatory agency shortcomings and drug pricing. With the COVID-19 pandemic enforcing the elimination of low-value interventions in all fields of medicine, efforts should urgently be made by all involved in cancer care to select only high-value and sustainable interventions. Transformation of medical education, improvement in clinical trial design, quality, conduct and reporting, strict adherence to scientific norms by regulatory agencies and use of value-based scales can all contribute to raising the bar for oncology drug approvals and influence drug pricing and availability.

Pharmacy's Call for Authorities to Aggressively Enforce Laws Addressing Price Gouging.

Early reports of potential treatment for coronavirus disease (COVID-19) have raised concerns related to pharmaceutical distribution. Despite the lack of high-quality evidence, the mere hope of effectiveness of potential treatments, such as hydroxychloroquine, has led to surges in demand for these products, and many pharmacists are already informally reporting shortages through social channels. As manufacturers and wholesale distributors struggle to fulfill orders for drugs such as hydroxychloroquine, short-term price increases may seem reasonable in a free market when demand increases. However, any price increases by manufacturers, wholesale distributors, and pharmacies might be seen as exploitive gouging of consumers during a declared emergency. In addition to concerns of price gouging, increases in prescription drug utilization during the pandemic may lead to increases in spending for all payers as members may be treated for COVID-19. This article explores pharmaceutical supply chain and drug pricing nuances that may cause problems for payers and pharmacies as the country battles this global pandemic. DISCLOSURES: No funding supported the writing of this article. Mattingly reports unrelated consulting fees from the National Health Council, Bristol Myers Squibb, G&W Laboratories, Allergy and Asthma Foundation of American, and the Massachusetts Health Policy Commission. Hogue has nothing to disclose.

Higher Drug Prices from Anticompetitive Conduct: Three Case Studies.

U.S. consumers pay high drug prices. Brand-name drug companies claim that these prices are justified by pathbreaking research and development. But, sometimes the prices result from anticompetitive conduct. This article offers three case studies of how such behavior can increase price based on wakefulness drug Provigil, the allergic-reaction-treating EpiPen, and infection-treating Daraprim. The article contends that behavior that makes no sense other than by harming a competitor, that undercuts a regulatory regime, or that involves collusive conduct should not be protected. In targeting this behavior, antitrust scrutiny promises to lower drug prices.

Physician altruism and moral hazard: (no) Evidence from Finnish national prescriptions data.

We test the physicians' altruism and moral hazard hypotheses using a national panel register containing all 2003-2010 statins prescriptions in Finland. We estimate the likelihood that physicians prescribe generic versus branded versions of statins as a function of the shares of the difference between what patients have to pay out of their pocket and what is covered by the insurance, controlling for patient, physician, and drug characteristics. We find that the estimated coefficients and the average marginal effects associated with moral hazard and altruism are nearly zero, and are orders of magnitude smaller than the ones associated with other explanatory factors such as the prescriptions' year and the physician specialization. When the analysis distinctly accounts for both the patient and the insurer shares of expenditure, the estimated coefficients directly reject the altruism and moral hazard hypotheses. Instead, we find strong and robust evidence of habits persistence in prescribing branded drugs.

When Is The Price Of A Drug Unjust? The Average Lifetime Earnings Standard.

The majority of Americans believe that lowering drug prices should be the top health care priority for the federal government. Yet drug costs as a proportion of the country's medical expenditures have increased substantially in recent years. Because drugs are basic necessities, and because how much society should contribute toward providing basic necessities is a question of justice, policies regarding drug prices must fulfill principles of justice, not just economic efficiency. In this article I define a standard for when the price of a drug is unjust, using a cross-disciplinary ethical approach. Based on four principles, I propose the average lifetime earnings standard for affordability. According to this standard, a drug price is unfair if it exceeds 11 percent of the average American's disposable income. This suggests that current prices for many drugs are excessive and unjust.

A perspective on global access to insulin: a descriptive study of the market, trade flows and prices.

AIM: To describe the global insulin market. METHODS: Market intelligence data, United Nations Commodity Trade Statistics for insulin trade, the International Medical Products Price Guide for prices of human insulin and additional web searches were used as data sources. These sources were combined to gain further insight into possible links among market, trade flows and prices. Descriptive statistics and Spearman's rank order correlation were used for the analysis. RESULTS: A total of 34 insulin manufacturers were identified. Most countries and territories are reliant on a limited number of supplying countries. The overall median (interquartile range) government procurement price for a 10-ml, 100-IU/ml vial during the period 1996-2013 equivalent was US$4.3 (US$ 3.8-4.8), with median prices in Africa (US$ 4.7) and low- (US$ 6.9) and low- to middle- (US$ 4.7) income countries being higher over this period. The relationships between price and quantity of insulin (Spearman's r=0.046; P>0.1) and number of import links (Spearman's r=0.032; P>0.1) were weak. The links between price and percentage of total insulin from a country where a 'big three' manufacturer produces insulin (Spearman's r=0.294; P<0.05) and total insulin from the main import link (Spearman's r=-0.392; P<0.05) were stronger. CONCLUSIONS: This research shows the high variability of insulin prices and the reliance on a few sources, both companies and countries, for global supply. In addressing access to insulin, countries need to use existing price data to negotiate prices, and mechanisms need to be developed to foster competition and security of supply of insulin, given the limited number of truly global producers.

Putting our best foot forward: Clinical, treatment-based and ethical considerations of nusinersen therapy in Canada for spinal muscular atrophy.

Spinal muscular atrophy (SMA) is the most common genetic cause of infant mortality. SMA is a spectral disorder and is categorised based on symptom onset and severity. The median life expectancy for infants with SMA presenting before 6 months of age is less than 2 years without respiratory support. To date, there is no cure for SMA. In June 2017, nusinersen was approved in Canada as the first disease-modifying drug for SMA because of its demonstrated benefits on motor function and survival in clinical trials. However, with a price tag of almost 1 million dollars for the first year of therapy, careful clinical, treatment-based and ethical consideration of the principles of (i) best interests; (ii) universality; (iii) portability; (iv) public administration; (v) accessibility; and (vi) comprehensiveness are important guideposts to ensure transparent and equitable allocation of health-care resources for nusinersen and all other future orphan drugs.

[Beating cancer, living with it: success and ethical dilemmas]. / Combattre et vivre avec le cancer : succès et dilemmes éthiques.

Cancers are serious conditions which affect numerous families. The advances made in treatments thanks to research enable a growing number of cancers to be cured. Some cancers which are treated evolve towards a form of chronicity whereby patients have to live with the condition. These varied situations, always sensitive, mobilise and bring together patients, their families, caregivers, researchers and associations. There are many ethical dilemmas facing all those involved in this fight.

Moral Distress with Obstacles to Hepatitis C Treatment: A Council of Academic Family Medicine Educational Research Alliance (CERA) Study of Family Medicine Program Directors.

BACKGROUND AND OBJECTIVE: To determine whether family medicine program directors (PDs) experienced moral distress due to obstacles to Hepatitis C virus (HCV) treatment, and to explore whether they found those obstacles to be unethical. DESIGN: An omnibus survey by the Council of Academic Family Medicine's Educational Research Alliance was administered to 452 and completed by 273 US-based PDs. The survey gauged attitudes and opinions regarding ethical dilemmas in patient access to HCV treatment. RESULTS: Most of the respondents were male. Sixty-four percent of respondents believed that treatment should be an option for all patients regardless of cost. Forty-one percent believed that it was unethical to deny treatment based on past or current substance use, and 38% believed treatment should be offered to patients who were substance abusers. Moral distress was reported by 61% (score >3) of participants when they were unable to offer treatment to patients due to the patient's failure to meet eligibility criteria. In addition, PDs reporting moderate-to-high levels of moral distress were also likely to report the following opinions: 1) treatment should be offered regardless of cost, 2) it is unethical to deny treatment based on past behavior, 3) substance abusers should be offered treatment, 4) it is unethical for medicine to be prohibitively expensive, and 5) Medicaid policy that limits treatment will worsen racial and ethnic disparities. CONCLUSIONS: Currently, important ethical dilemmas exist in the access and delivery of HCV therapy. Although a diversity of opinions is noted, a significant proportion of PDs are concerned about patients' inability to avail equitable care and experience distress. In some cases, this moral distress is in response to, and in conflict with, current guidelines.

Restoring a reputation: invoking the UNESCO Universal Declaration on Bioethics and Human Rights to bear on pharmaceutical pricing.

In public health, the issue of pharmaceutical pricing is a perennial problem. Recent high-profile examples, such as the September 2015 debacle involving Martin Shkreli and Turing Pharmaceuticals, are indicative of larger, systemic difficulties that plague the pharmaceutical industry in regards to drug pricing and the impact it yields on their reputation in the eyes of the public. For public health ethics, the issue of pharmaceutical pricing is rather crucial. Simply, individuals within a population require pharmaceuticals for disease prevention and management. In order to be effective, these pharmaceuticals must be accessibly priced. This analysis will explore the notion of corporate social responsibility in regards to pharmaceutical pricing with an aim of restoring a positive reputation upon the pharmaceutical industry in the public eye. The analysis will utilize the 2005 United Nations Educational, Scientific, and Cultural Organization's Universal Declaration on Bioethics and Human Rights (UDBHR) to establish implications regarding the societal responsibilities of pharmaceutical companies in a global context. To accomplish this, Article 14 of the UDBHR-social responsibility and health-will be articulated in order to advocate a viewpoint of socially responsible capitalism in which pharmaceutical companies continue as profit-making ventures, yet establish moral concern for the welfare of all their stakeholders, including the healthcare consumer.

Transforming Prior Authorization to Decision Support.

PURPOSE: To evaluate a computer-based prior authorization system that was designed to include and test two new concepts for physician review: (1) the tool would minimize denials by providing real-time decision support with alternative options if the original request was noncompliant, and (2) the tool would collect sufficient information to create a patient registry. METHODS: A new prior authorization tool incorporating real-time decision support was tested with a large national payer. The tool used the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology as the content for decision making. Physicians were asked to submit the minimal amount of clinical data necessary to reach a treatment-decision node within the National Comprehensive Cancer Network Guidelines. To minimize denials, all available recommended treatments were displayed for physician consideration and immediate authorization was granted for any compliant selection. RESULTS: During a 1-year pilot in a Florida commercial health plan, 4,272 eligible cases were reviewed with only 42 denials. Chemotherapy drug costs for the prior authorization pilot were compared with a similar time period in the previous year for the state of Florida, as well as for the Southeast region and for the nation, which served as controls. The percentage change between the time periods was -9% in Florida, 10% for the national costs, and 11% for the Southeast region costs. The difference between the regional increase and the Florida decrease represented a savings of $5.3 million dollars for the state of Florida in 1 year. CONCLUSION: There is significant opportunity to reduce the costs of therapy while being compliant with nationally accepted guidelines for cancer chemotherapy.

[The willingness to pay for new drugs is based on ethical principles]. / Betalningsviljan för nya läkemedel bygger på etiska principer - NT-rådet tolkar riksdagens plattform för prioritering.

The County Council's board for new therapies (the NT Council) provides recommendations on the use of new drugs based on the ethical platform of priorities, founded by the Swedish parliament. The Council has formulated a policy that interprets the parliamentary ethical platform and operationalize its need and solidarity principle and cost effectiveness principle in four dimensions. The NT Council weighs the health economic evaluation of the drug and the four dimensions: the severity of the condition, the rarity of the condition, the effect size and the data reliability to determine the willingness to pay level and whether the platform allows a recommendation for using of the drug. The severity of the condition has a greater impact than the other dimensions. In the assessment of severity there is also a trade-off between prevention and treatment of manifest diseases and in prevention, the size of the risk of falling ill is of importance. A slightly higher willingness to pay level is reasonable for treatment of very rare conditions, but it is important that identified patients are not given priority over anonymous patient groups with equally strong needs.

Limiting the access to direct-acting antivirals against HCV: an ethical dilemma.

INTRODUCTION: Hepatitis C virus (HCV) infection affects about 200 million people worldwide and represents a leading cause of liver-related mortality. Eradication of HCV infection, achieved mainly through direct-acting antivirals (DAA), results in a decrease of mortality and an improvement of quality of life. These drugs have a maximal efficacy and an optimal tolerability. However, their high cost precludes a universal access even in wealthy countries. Areas covered: This article deals with the policies adopted for the use of the new anti-HCV drugs, especially in Europe and most of all in Italy, supposedly the developed country with the highest HCV prevalence. The literature search was performed using Pubmed and Web of Science. Moreover, national regulatory institutional websites were consulted. Expert commentary: The current policy of limitation to the access of the DAA presents a series of ethical issues that makes it non-applicable. A 'treat-all' strategy should resolve all ethical dilemmas, by virtue of the wide benefits of anti-HCV treatment not only for the advanced stage of infection, but also for the initial stages. A reduction in price of the drugs is the actual condition to achieve such a change.