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Archive for May 2019

Science Policy Around the Web – May 31st, 2019

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By: Silvia Preite, Ph.D.

Image by rawpixel from Pixabay 

Common drink and food sweetener – High-Fructose Corn Syrup – accelerates colon cancer growth in mouse models: what about in humans?

Increased consumption of sugar-sweetened drinks has been associated with higher risk of obesity and intestinal cancers. However, whether sugar directly contributes to tumor development, independently from obesity, is less clear. A common sweetner of sodas, fruit-flavored drinks and processed foods is high-fructose corn syrup (HFCS). A recent study published in Science revealed that consumption of HFCS accelerated colon cancers in predisposed mice bearing a mutation in a tumor-suppressor gene commonly found in human colorectal colon cancers. Strikingly, the human diet equivalent amount of HFCS required to see such effects in mice corresponds to 12 ounces of a sweetened drink – one can of soda per day!

Mice fed with HFCS did not become obese or developed metabolic syndrome, however, developed larger and more advanced tumors, compared to water-treated animals. Mechanistically, HFCS leads to increased levels of fructose and glucose in the intestinal lumen and serum, that can be transported and utilized inside the tumor to generate energy and support its growth. The identification of these events opens new possibilities for the development of therapeutic strategies aimed at controlling tumor growth; in particular, targeting of fructose metabolism may selectively slow tumor progression without affecting survival of normal cells. 

Further studies are needed to assess if similar tumorigenic mechanisms take place in humans. Moreover, whether prolonged and extensive consumption of HFCS has a greater detrimental effect on human health compared to other types of sugar remains to be determined. Regardless, this study could contribute to increase public awareness about the potential deleterious effects on physical health and tumor development due to sweetened drinks and processed food whose comsumption is globally rising. 

(Source: Goncalves et al., Science, 2019)

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May 31, 2019 at 3:08 pm

Gene editing- Regulatory and ethical challenges

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By: Chringma Sherpa, Ph.D.

Image by Colin Behrens from Pixabay 

When power is discovered, man always turns to it. The science of heredity will soon provide power on a stupendous scale; and in some country, at some point, perhaps, not distant, that power will be applied to control the composition of a nation. Whether the institution of such control will ultimately be good or bad for that nation, or for humanity at large, is a separate question.

William Bateson, English biologist who coined the term “genetics.”

On November 25, 2018, in an allegedly leaked YouTube video, He Jiankui, a scientist at the Southern University of Science and Technology in Shenzhen, China, revealed the birth of the first gene-edited babies using a technology called CRISPR. There has been a general consensus in the scientific community that heritable changes should not be made to prevent the off-target and unwanted genetic changes artificially produced in an individual during gene editing to be passed on to his/her offspring(s). He became the first scientist to publicly violate this consensus resulting in an international scandal and criminal/ethics investigations into both He and his collaborators.

In the wake of He’s CRISPR-babies scandal, scientists worldwide are debating on the ethical and regulatory measures that would discourage another wayward and rogue scientist like He from attempting such an irresponsible feat.  At the 2nd international summit on human gene editing that convened two days after He’s video became public, He presented his work. The summit was well attended by ethicist and journalist besides scientists. At the summit, David Baltimore of the California Institute of Technology, who chaired the organizing committees for both the 1st and 2nd international summits on human gene editing read one of the conclusions from the 1st summit held at Washington DC in 2015 – “It would be irresponsible to proceed with any clinical use of germline editing unless and until (i) the relevant safety and efficacy issues have been resolved, based on appropriate understanding and balancing of risks, potential benefits, and alternatives, and (ii) there is broad societal consensus about the appropriateness of the proposed application”. Baltimore called He’s work outright irresponsible on the basis of the statement from the 1st summit. At the summit, many other ethical and safety-related questions were raised which He failed to answer or did not answer convincingly. 

He’s scandal has driven various organizations to draft new guidelines and sanctions aimed at preventing unethical and unapproved use of genome editing.  China has imposed new laws requiring human gene editing projects to be approved by China’s health ministry first to avoid fines and blacklists. Both the 2nd human gene editing summit and the WHO panel that convened in March 2019, have proposed a central registry of human gene-editing research and called for an international forum/ committee to devise guidelines for human gene editing based on common norms and differences of opinions between countries.  To allow time for the creation and effective implementation of new regulations, the WHO also called for a global moratorium on heritable editing of human eggs, sperm, or embryos for the next five years. Supporting the WHO panel’s recommendations, Francis Collins, director of the National Institute of Health, said that “NIH strongly agrees that an international moratorium should be put into effect immediately”. However, not all scientists are in favor of a moratorium, as they believe it might stifle the growth of a technology that might be safe and beneficial in the near future. Jennifer Doudna of the University of California, Berkley, one of the co-inventors of CRISPR gene editing, says that she prefers strict regulation that precludes the use of germline editing until scientific, ethical, and societal issues are resolved over a moratorium. David Baltimore agrees with Doudna stating that the word moratorium was intentionally not used in both the human gene editing summits as a moratorium would be hard to reverse.  Science historian Ben Hurlbut of Arizona State University, who had numerous discussions with He before Lulu and Nana were created, thinks a blanket moratorium on clinical germline editing would have prevented He from proceeding. Both the two human gene editing summits and a 2015 essay by Baltimore, Doudna, and 16 co-authors had already outlined numerous guidelines for clinical germline editing. According to Hurlbut, He weighed these criteria and believing that his procedure met all the guidelines proceeded. A categorical prohibition of germline editing would not have allowed him to use his subjective judgment and act out of self-interest. 

The modern debate over CRISPR editing is not the first time the scientific community has come together to discuss game-changing biological technologies, and it is heavily informed by two prior events. In 1970, Paul Berg and his postdoctoral researcher David Jackson used the recombinant DNA technology to create the first chimeric DNA. This invention created an uproar among the scientists and the general public who feared that this technology would lead to the creation of uncontrollable and destructive superbugs, the exaggerated versions of which can be seen in some science fiction movies. Yielding to the opinions and sentiments of the fellow scientists, Berg held himself from cloning such recombinant DNAs and in 1974, he pleaded for a voluntary moratorium on certain kinds of recombinant DNA research until their safety issues have been resolved.  He also moved quickly to organize the Asilomar conference (Asilomar II) in 1975 that bore semblance to the 2nd human gene editing conference in that it invited not only the scientists but lawyers, ethicists, writers, and journalists to weigh in on the risk-benefit analysis of the Recombinant DNA technology. On the recommendation of Asilomar conference, Donald Fredrickson, then director of the National Institutes of Health (NIH), initiated the formation Recombinant DNA Advisory Committee (RAC) to act as a gatekeeper of all research that involved recombinant DNA technology. The scope of the committee, which was composed of stakeholders, including basic scientists, physicians, ethicists, theologians, and patients’ advocates was later expanded to encompass the review and approval of human gene therapy research. Due to the redundancies of regulatory oversights between the US Food and Drug Administration (FDA) and RAC, RAC was reinstated as only an advisory body providing advice on the safety and ethical issues associated with emerging biotechnologies in 2019.

While this is a successful example of scientific self-regulation, the second event resulted in a major setback in the field of gene therapy. On September 13, 1999, Mark Batshaw and James Wilson of University of Pennsylvania supervised the administration of adenovirus to an 18-year-old Jesse Gelsinger in a gene therapy clinical trial. Gelsinger died of liver and kidney failure and brain damage three days later. Like the birth of CRISPR babies, Gelsinger’s death was an instance where new technology was used prematurely without a thorough assessment of its safety profile. It is suspected that both the clinical applications headed by He and Wilson might also have been motivated by fame and financial gain; He and Wilson both had financial stakes in private biotechnology companies that would benefit from these human trials. In the aftermath of Gelsinger’s death, Wilson was banned from carrying out FDA regulated clinical trials for the next five year, nearly all gene therapy trials were frozen, and many biotechnology companies carrying out these trails went bankrupt. This was a dark period in the history of gene therapy, and it would take almost another decade of introspection, reconsideration, and more basic experimentation for gene-therapy to re-emerge as a viable therapeutic strategy.

Figure 1: The regulatory status of human germline gene modification in various countries. Thirty-nine countries were surveyed and categorized as “Ban based on legislation” (25, pink), “Ban based on guidelines” (4, faint pink), “Ambiguous” (9, gray), and “Restrictive” (1, light gray). Non-colored countries were excluded in this survey. Adapted from Araki, M. and Ishii, T (2014): “International regulatory landscape and integration of corrective genome editing into in vitro fertilization” Reproductive Biology and Endocrinology, 2014 12:108

Scientists at both the Asilomar and human gene editing conferences passionately debated the safety of the relevant technologies but deliberated on the discussion of the big ethical issue associated with these technologies – the ultimate creation of designer babies. That gene editing sits on the slippery slope to eugenics was recognized since the days of Charles Darwin and Gregor Mendel when the study of genes and heredity was still in its infancy and the discovery of DNA as the genetic material was half a century away. One of the earliest proponents of genetic manipulation for human benefits was Francis Galton, Charles Darwin’s cousin. Galton proposed an unnatural and accelerated selection of beneficial traits by marriage between people of desirable traits. The danger that someday some rogue scientists might use germline gene editing technology in favor of eugenics lurks in the mind of those who understand the potential of the currently available gene editing technologies. However, more fearful is the idea that the wave of positive eugenics would soon give way to negative eugenics – elimination of undesirable traits as it did around World War II as exemplified by the famous case of Carrie Buck, a woman who was designated “mentally incompetent” and involuntarily sterilized. 

Various countries have their own regulation and legislation on germline editing to prevent any backlash from this powerful technology. Figure 1 presents a summary of the regulatory landscape of germline gene modification surveyed in thirty-nine countries by Araki Motoko and Tetsuya Ishii.  In the US, Congress has shown strong support against germline gene editing. In 1996, it passed a rider as part of the annual appropriations bill that prohibits the use of federal funds for any research involving human embryo. In another appropriations bill passed in 2015, Congress banned the FDA from considering applications involving the therapeutic modification of the human germline. 

Human gene editing holds great promises in treating many life-threatening and previously intractable diseases. Only when this discipline of science is held to high ethical standards and regulated sensibly at international, national, and a personal level, shall we reap the benefits of this powerful technology.

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May 29, 2019 at 9:25 am

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How are we welcoming our next generation-The first 1000 golden days?

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By: Deepika Shrestha , Ph.D.

Source: Wikimedia

The most important period for a child’s development, especially for the brain and immune system, is the first 1000 days of life. The Developmental Origins of Health and Disease (DoHAD) hypothesis suggests that the roots of many complex diseases and behavioral risks originate very early – between the time window of pre-conception to early postnatal periods. Indeed, genetic, epigenetic and environmental evidence indicate that most adulthood health and disease risk have been coded during fetal development in the intrauterine environment, lending support for the DoHAD hypothesis

The increased prevalence of an obesogenic environment and rate of chronic diseases in mothers increases the risk of childhood obesity and future cardiometabolic risk in the offspring. Moreover, increased risks can be transgenerational within families. This is also the case for Autism, Attention Deficit Hyperactive Disorder (ADHD), and other mental and psychological disorders. Given that over 50% of all mothers are overweight or obese, there is a growing cause of concern about the quality of fetal growth and development. Further, recent statistics revealed a sobering picture of increased incidence of various maternity-related illnesses such as postpartum depression (CDC Reports: as high as 1 in 5 women) and maternal mortality (CDC MMR 2019 report: 700 deaths per year- out of which relatively 3 in 5 deaths preventable, 31.3% deaths occurred during pregnancy, 16.9% on the day of delivery, and 51.8% over 1 year of post-partum days) in the US. These numbers show that many women in the US are among the most vulnerable and need sufficient support from family, society and governmental policy. There have been successful campaigns to institute policies raising awareness of issues concerning fetus growth, development and maternal/infant nutrition in developing countries, such as Golden 1000 Days. The United States needs similar programs special focusing on maternal care, especially on the nutritional, psychological, mental and financial needs of pregnant mothers and women in reproductive age groups. 

Another program has also been put in place to improve nutrition in mothers and children. The Women, Infant and Child (WIC) program provides supplemental nutritional support to roughly 8 million low-income mothers and young children under 5 years of age. With approximately 6 billion USD of funding in 2016, the current WIC program is the result of an update in 2009 after rigorous review by the Institute of Medicine (IoM) to reflect the latest nutritional science as well as public health concerns. However, recent evidence indicates that food and nutrients supplements through the WIC program might not match the nutritional need of the participants as it fails to account for women’s prepregnancy obesity status, gestational weight gain, and gestational diabetes.  For instance, concentrated fruit juice may increase the risk of gestational diabetes risk and is not a healthy food option. This important policy needs fair re-evaluation based on the updated scientific evidence for nutritional needs.

Another point of concern for expecting mothers is the lack of psychological care. Mothers-to-be undergo extensive physiological and psychological changes during pregnancy. Therefore, this 1000-day window is a sensitive time period —a time where pregnant women require support and potential intervention. Recent data highlight increasing trends of maternity related illnesses, be it postpartum depression or maternal mortality. More importantly, in the US these issues disproportionately affect women of color or low socioeconomic status.  Alarmingly, 42% of mothers  are sole or primary earners and may lack adequate financial support from their spouse and family. The Pregnancy Discrimination Act and Family and Medical Leave (FMLA) act was put in place to protect pregnant women in the job place for 12 weeks after childbirth. However, the United States is one of the few countries in the world with almost no access to paid parental leave—only 14% of civilian workers have access to any amount of paid parental leave in 2016, a slight increase from 11% in 2010

Access to paid parental leave currently serves as elite benefits and is dependent upon company policy. The most generous policies afford 16 weeks for birth mother, 8 weeks for birth father, 8–16 weeks for adoptive parents (16 for primary, 8 for secondary) according to PL+US’s report. About 23% of mothers go to work within 10 days of giving birth and are disproportionately from low-income families.In addition to maternal paid leave, recent mothers often require considerable sick leave for the first year, and providing a flexible policy could be a steppingstone towards helping the psychological as well as physiological health of a child. Furthermore, it is no secret that inspected and reputable day care facilities takes a major chunk of the family income, and are unaffordable to many families.

In addition, there is also a need for Newborn Rights. Irrespective of socioeconomic status, each baby has inborn rights and deserve equal family bonding time and breast-feeding needs. Children born to poor maternal care during pregnancy and lactating period are at increased risk of having neurological problems, poor school achievement, early school dropout, low-skilled employment, and providing poor care to their own children, thus contributing to the intergenerational transmission of poverty and malnutrition. On the contrary, children who get good nutrition and care in their first 1000 days are ten times more likely to overcome life threatening childhood disease, have higher educational retention in schools and are likely to earn more than 21% in wages as adults and also to have healthier families on their own. Therefore, there is an unmet need for a stronger policy that invests in children and their families from the very beginning and helps each child to be a healthy and contributing member of the society in their adulthood. It takes a major process and significant effort to raise a child into a healthy adult who is mentally, spiritually and physically fit to keep going in a productive society.

Paid maternity leave and insurance coverage needs support from Government/Congress to mothers or family unit regardless of the beneficiary’s work status. Investing in this policy may cost taxpayer a small percent of GDP (Gross Domestic Products) but will have a huge return in the long run when Health is valued as development index. There is ample evidence to show that countries that fail to invest in the well-being of women and children in the first 1,000 days lose billions of dollars to lower economic productivity, health issues, societal inequality and higher health costs. This is a main point of concern given that the US is lagging far behind other developed nations in this human development index (HDI). Currently, there is a huge disparity of investment in the first 1000 days based on socioeconomic status, and there is a clear and unmet need for structural and policy intervention. Issues related to the maternity period such as nutritional aspect, mental health, and paid maternity (or paternity) leave should not be considered only a women’s issue. Therefore, more than ever, there is a heightened need for research resources to understand maternity health issues and also concrete plans to address these issues.

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May 24, 2019 at 2:21 pm

Breast cancer screening: How do we maximize benefit while minimizing harm?

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By: Catherine Lerro, Ph.D., M.P.H.

Image by Bruno Glätsch from Pixabay 

Breast cancer is the most commonly diagnosed tumor in US women, and the second leading cause of cancer death in women with an estimated 268,600 diagnoses and 41,760 deaths predicted for 2019. Despite these seemingly sobering numbers, mortality due to breast cancer has declined over the past several decades. Today, women diagnosed with early stage disease are about 99% as likely to be alive five years after their diagnosis as cancer-free women. These declines in cancer death have been largely attributed to both improvements in treatment and successful implementation of mammography (breast x-ray) screening programs, considered a hallmark of preventative cancer care. Some researchers estimate that upward of 380,000 breast cancer deaths have been averted since 1989 due to mammography and improved breast cancer treatment. In fact, the Affordable Care Act has provisions to ensure that women with private health insurance, public health insurance (e.g. Medicare, Medicaid), or health insurance purchased through a state exchange are covered for breast cancer screening. 

The idea behind mammographic screening is that breast cancers diagnosed at an early stage are more likely to respond well to treatment, preventing cancer-related death. While not all cancers have population-wide screening programs, breast cancer is a good candidate for screening. First, breast cancer is common enough to warrant subjecting women to a mammogram at regular intervals during a defined period of known risk. If a disease is very rare, it is likely not a good candidate for population-wide screening because the costs would outweigh the potential benefit. Second, there must be a good test available that is both sensitive and specific. In other words, the test should detect as many true cases as possible, while minimizing the number of patients with false-positives that require more invasive testing such as a biopsy. Finally, there must be some benefit to detecting disease early. For breast cancer, women with early stage disease may be more easily treated and have better prognosis compared to women with distant-stage disease.

Currently, mammograms are recommended for much of the adult female population in the US over the age of 50. Many different organizations release breast cancer screening guidelines on a regular basis including (but not limited to) the US Preventive Task Force, the American Cancer Society, the American College of Obstetricians and Gynecologists, and the American College of Radiology. While the recommendations share some similarities, there are important differences and no one guideline is universally accepted. For example, for women ages 50-74, the US Preventative Task Force recommend biennial mammograms, while the American College of Radiology recommends yearly mammograms. These differences may arise from the data used to develop the guidelines and how the data are valued. For example, the US Preventive Task Force counts mortality reduction as the soul benefit of mammography and considers potential risks such as false-positive tests. The American College of Radiology considers other mammography benefits outside of morality reduction such as less aggressive treatment for early stage cancers. The American College of Radiology also have recently amended their guidelines to consider race, with the option to screen African American women, who are at greater risk of more aggressive breast cancers, starting at younger ages at the discretion of both the patient and physician. 

Understanding how and if breast cancer screening guidelines are integrated into clinical practice is a murkier area still. In recent years, most major guidelines recommend less routine screening and have endorsed a more individualized approach that involves discussion of the benefits and harms of screening and incorporates patient preferences and beliefs, especially for younger women. However, studies have found that despite these changes in recommendations, breast cancer screening in practice in the US has changed very little. This may be driven by US health system traits, such as fee-for-service payment systems and concerns about litigation. Furthermore, both clinicians and patients may overestimate the benefits and underestimate the harms of mammography, particularly for younger women.

The benefits of diagnosing breast cancer early cannot be overstated, as response to treatment and survival depends greatly on stage at diagnosis. However, the potential harms of screening are often overlooked. Of course, there are economic costs incurred for any wide-scale screening program. Just as importantly, we should seriously consider the physical and emotional costs of overdiagnosis and overtreatment. A 2018 report in the Journal of the American Medical Association found that for every 10,000 women screened for breast cancer, more than half under the age of 60 will experience a false positive test result. Almost 10% of women will undergo at least one unnecessary biopsy. Additionally, the authors demonstrated that through screening more women were potentially overdiagnosed (cancers diagnosed and treated that would have never become clinically evident) than deaths were averted. There may be psychological consequences to false positive test results, including both short-term and long-term anxiety. Unnecessary biopsy and overdiagnosis could potentially have long-term physical health consequences that would otherwise be avoided. 

How do we improve mammography screening in the US, maximizing the benefits while minimizing the risks? What is clear is that there is no simple solution. In a health system that largely favors more testing at potential cost to patients, institutional changes in how health insurance reimburses clinicians for care should consider looking beyond fee-for-service models. The newest breast cancer screening guidelines also favor individualized approaches, prioritizing screening among high-risk women and educating patients about the potential benefits and harms of screening with full consideration of their own medical history and preferences. Clinicians may consider tools that utilize detailed patient information to assess an individual patient’s risk of breast cancer, as well as tools soliciting patient preferences that support shared decision-making. Finally, it is important that all women requiring regular mammograms have access to breast cancer screening and high-quality treatment regardless of age, race, geographic location, or socioeconomic status, in order to minimize disparities in stage at diagnosis and breast cancer survival. 

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May 15, 2019 at 11:30 am

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Science Policy Around the Web – May 14th, 2019

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By: Mary Weston, Ph.D.

Source: Pixabay

Rural areas drive increases in global obesity

While past studies have found that the increase in global obesity is largely driven by urban regions, a newly published paper argues that this rise is actually being led by those in rural areas. 

Global increases in BMI (body mass index) have been observed for decades, but no one had evaluated differences in urban and rural regions on a large-scale across many countries. The new Nature study evaluated BMI values in 200 countries from 1985-2017, finding that rural areas are responsible for more than 55% of the global rise in the average BMI and more than 80% of the rise in some low- and middle-income countries (LMICs). 

Previous theories argued that urbanization caused rising BMI largely because of the availability of cheap, ultra-processed foods, a lifestyle that provides more transportation options, and greater access to non-physical leisure activities (smartphones, cable television). In contrast, rural areas were thought more likely to consume more local produce, have less access to ultra-processed and packaged food, and participate in high energy expending activities. However, rural areas, even in LMICs, have now begun to resemble urban areas because of access to ultra-processed foods and cheap mechanized devices that reduce transport and farming energy expenditure.

Obesity results in higher health care costs, lower life expectancy, and reduced quality of life. Thus, prevention strategies are vital but currently, most preventative measures are targeted towards urban areas. Given this new data, funding priorities and strategies need to adjust to address this growing issue. 

(Barry M. Popkin, Nature)

After outcry, USDA will no longer require scientists to label research as ‘preliminary’

After protests, the US Department of Agriculture (USDA) has stopped requiring their staff scientists to label all published peer-reviewed research as “preliminary.” Released last week, the revised USDA guidelines now require the following language when disclaimers are necessary: “The findings and conclusions in this [publication/presentation/blog/report] are those of the author(s) and should not be construed to represent any official USDA or U.S. Government determination or policy.” Not all publications will be obliged to contain this statement. 

Previous USDA guidelines, implemented last July, required publications to carry the label: “The findings and conclusions in this preliminary publication have not been formally disseminated by the [USDA] and should not be construed to represent any agency determination or policy.”  This disclaimer caused concern over claims that it was confusing and possibly misleading. Scientific publications are peer-reviewed (evaluated by professionals in the field for quality and accuracy) and considered completed work, not preliminary. Some among the scientific community feared the disclaimer might reduce the impact of the published research conclusions or be used to diminish findings that conflict with views of the current administration. 

While reaction towards the disclaimer change has been generally positive, some non-USDA researchers are still concerned that the latest guidelines have the potential to jeopardize scientific integrity. The new guidelines say that the USDA can request “corrections” or “changes” to research papers if they pertain to a “prominent issue,” a significant scientific advancement, or could influence trade/policy decisions. Rebecca Boehm, an economist at the Union of Concerned Scientists, stated that “removing ‘preliminary’ from the disclaimer is a step in the right direction, but there still may be unnecessary obstacles preventing agency researchers from publishing their work in peer-reviewed journals.” 

(Ben Guarino, Washington Post)

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May 14, 2019 at 4:56 pm

Science Policy Around the Web – May 10th, 2019

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By: Caroline Duncombe

Image by Steve Buissinne from Pixabay 

Drug prices will soon appear in many TV Ads

When watching an advertisement for a prescription drug on the television, a new addition will be added to that long list of side-effects: the product’s cost. On Wednesday, the health and human services secretary, Alex Azar, announced that the Trump administration will require pharmaceutical companies to state the priceof prescriptions in television advertisements in “legible” text, similar to the current standard of side effects. This policy will only apply to drugs whose whole-sale-price exceeds $35 per month.

            Such a change in policy has received substantial pushback from the drug industry. They claim that including the list price would confuse consumers who would potentially pay a different amount post-insurance (that is if you have insurance).  In addition, drug companies claim that such a requirement infringed on the companies’ First Amendment Rights. One of the driving forces behind the new policy change are the patient advocacy groups, who have pointed out that televised drug ads direct consumers to the higher priced medications.  The hope of such a policy is to potentially incentivize drug companies to lower their prices. Mr. Azar in a statement points out “If you’re ashamed of your drug prices, change your drug prices.” 

(Glenn Thrush and Katie Thomas, NYT)


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May 10, 2019 at 3:07 pm

Living in America with a chronic disease: Drug prices here and why they are so high.

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By: Mohor Sengupta Ph.D.

Image by Liz Masoner from Pixabay 

The USA has the highest average prices on drugs compared to all other developed nations across the globe. The average expenditure on drugs per person is around $1200 per year in the U.S., while it is roughly $750 in Canada, according to a 2014 survey. Let us look at a specific example. Nexium is a drug that helps reduce stomach acidity. It is manufactured by AstraZeneca in Sweden and sold to customers in the U.S., Canada, U.K., Australia, New Zealand, India and Turkey. The 40 mg pill costs $3.37 in Canada, $2.21 in the U.K., Australia and New Zealand, less than 37 cents in India and Turkey and $7.78 in the U.S. Specialty medicines, like those used for cancer can cost $10,000 a month in the U.S

Fred Smith, whom I interviewed recently, is a 26-year-old freelance musician and trumpet instructor. Shortly after his 26thbirthday, his health insurance coverage under his mother’s provider plan ended. He went on to buy his medical insurance from the private provider Blue Cross Blue Shield only to realize that he had to pay nine times the cost for each of two medicines, Vyvanse and Viibryd, and 18 times the cost for a third medicine, Adderall, compared to the amount paid while on his mother’s insurance. 

So why do Americans pay more for their medicines? 

  • Drug manufacturers in the U.S. can set the price of their products. 

While this is not the norm elsewhere in the world, federal law in the U.S. does not allow FDA or public insurance providers to negotiate drug prices with manufacturers. Medicare Part D is a 2003 legislation that prevents the nation’s largest single-payer health system from negotiating drug prices. Medicaid, which is the public healthcare program for people with limited income and resources, must cover all FDA-approved drugs, irrespective of the cost. However, drug makers must provide rebates to the government for drugs billed to Medicaid. In general, the biggest cost of medicines is borne by Medicare and private insurers. Private insurance providers do not usually negotiate prices with drug manufacturers. This is because middlemen or third-party pharmacy benefits managers that administer prescription drugs, such as CVS Health, receive payments from drug companies to shift market share in favor of these insurers. These deals also leave consumers with a limited choice. 

Drug makers in the U.S. not only set their own prices but they are also authorized to raise prices. Martin Shkreli became the “most hated man in America” overnight when he raised the price of a generic anti-parasitic drug Daraprim from $13.5 a pill to $750 a pill, a 5000% increase. Mr. Shkreli explained to critics that the hike was warranted because Daraprim is a highly specialized medicine and likened it to an Aston Martin previously sold at the price of a bicycle. He added that the profits from the price increase would go into improving the 62-year-old recipe of the drug. 

Deflazacort, a steroid used to treat Duchenne muscular dystrophy, is a generic compound that has been available worldwide for decades and costs $1000-$2000 per year. Yet, Illinois-based Marathon Pharmaceuticals acquired FDA approval to sell deflazacort under the brand-name Emflaza at $89,000 per year. 

Speaking of generic drugs, here is the next big reason for unaffordable brand-name medicines. 

  • Government-protected monopolies for certain drugs prevent cheaper generics from entering the market. 

The U.S. has a patent system that allows brand-name drug makers to retain exclusive selling rights for 20 years or more. Makers of drugs for rare diseases can also enjoy indefinite monopoly of sale. Moreover, these rare drug makers can extend their solo market dominance by making minor and non-therapeutic modifications to the patented product, like changing the dye component in the coating. They also often pay generic manufacturers to delay their products from entering the market. 

Additionally, FDA approval of generics following expiration of brand-name drug patents can be a long process; it can take up to 3-4 years for generic drug manufacturers to get FDA approval. It is estimated that prices of generic medicines fall to 55% of the brand-name medicine price once two generics enter the market and 33% of the brand-name cost when five generics become available. 

However, why would a brand-name manufacturer applying for a patent cite an unaffordable price to begin with?

  • Unjustified cost of research and development are cited by drug makers. 

It is generally agreed among critics that drug makers put an unjust price on their product citing the research that went into producing it. Because most of the R&D is funded by the National Institutes of Health via federal grants or by venture capital, the cost of research cited by the drug makers is above exaggeration. In reality, companies spend no more that 10-20 percent of their revenue on the research. 

Sofosbuvir was made by Michael Sofia, a scientist with a Princeton-based pharmaceutical company called Pharmasset. He even received the 2016 Lasker-DeBakey Clinical Medical Research Award for inventing it. Sofosbuvir is recommended for management of hepatitis C. After Gilead Sciences acquired Pharmasset for $11 billion in 2011, it applied to FDA for a new drug combining Sofosbuvir and Ribavirin, first made in 1972 by scientists at International Chemical and Nuclear Corporation (currently Canada-based Bausch Health Companies). Gilead priced their product at $84,000 for a single course of treatment in the U.S. The pricing caused a huge controversy when patients on Medicaid were denied the drug until becoming seriously ill. Moreover, generic licensing agreements to produce Sofosbuvir in 91 developing countries, which bear the burden of more than half the world population with hepatitis C, came under fire when Gilead asked for prices unaffordable by consumers in these countries. 

This brings us to the final cause of high drug prices. 

Doctors are often unaware that their prescriptions could be cheaper for their patients if they purchased two generic medicines instead of the brand-name prescription drug that is just a combination of the two. Vimovo, manufactured by Horizon Pharma, is a drug used to treat symptoms of osteoarthritis, rheumatoid arthritis, and ankylosing spondylitis. It is a combination of two generic medicines, naproxen (brand-name Aleve) and esomeprazole (brand-name Nexium). Naproxen is the anti-inflammatory component (NSAID) and esomeprazole is the aforementioned stomach-acidity reducer. It is added to the combination to reduce side effects of the NSAID. Whereas a month’s supply of Aleve and Nexium cost one patient $40, his insurance company was billed $3252 for the same supply of Vimovo. Moreover, not everyone who uses NSAIDs experiences stomach problems and do not need the additional esomeprazole component. 

Several Americans do not fill their prescriptions because they cannot afford to. Data show that 36 million Americans between the ages of 18 to 65 did not fill their prescriptions in 2016. Many resort to buying medicines online from foreign sellers or get them imported. Both routes are illegal and therefore we do not know the exact percentage of the population participating in these practices. 

I interviewed Tammy Connor, who regularly gets her medications from abroad. Tammy takes Synthroid, a brand-name drug, which is used to manage symptoms of hypothyroidism. She has been procuring it from Canada at 1/3rd its U.S. price for many years. In the middle of 2018, the U.S. began blocking drug purchases from Canada, preventing her from continuing this cost-saving practice. Eventually, she got a referral to a U.K.-based drug company called Medix Pharmacy, where she pays 1/3rd the amount that she would have to pay if she purchased Synthroid from the U.S. “Ironically, Medix gets its Synthroid supply from Canada”, Tammy said.

Big Pharma” is a major lobbying group in the U.S. This is a group of a few gigantic pharmaceutical companies which have together kept their profit margins rising amidst public outcry of drug unaffordability. Big Pharma also includes corporations that push overpriced drugs to customers. With their deep pockets, they can spend astronomical amounts on advertising and lobbying. 

Unaffordable prices of life-saving medicines cause many people to skip taking necessary medications, thanks to the Big Pharma. Now, more than ever, is the time that something was done about this. 

Recommended links: 

  1. http://money.com/money/4462919/prescription-drug-prices-too-high/
  2. https://jamanetwork.com/journals/jama/article-abstract/2545691
  3. https://www.cnbc.com/2017/05/10/americas-10-most-expensive-prescription-drugs.html
  4. https://www.renalandurologynews.com/home/news/almost-1-in-10-americans-cant-afford-medications-says-cdc/

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Written by sciencepolicyforall

May 9, 2019 at 4:23 pm