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Growing Need for More Clinical Trials in Pediatrics

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By: Erin Turbitt, PhD

Source: Flickr by Claudia Seidensticker via Creative Commons

      There have been substantial advances in biomedical research in recent decades in the US, yet children have not benefited through improvements in health and well-being to the same degree as adults. An illustrative example is that many drugs used to treat children have not been approved for use by the Food and Drug Administration (FDA). Comparatively, many more drugs have been approved for use in adult populations. As a result, some drugs are prescribed to pediatric patients outside the specifications for which they have been approved for use, referred to as ‘off-label’ prescribing. For example, some drugs approved for Alzheimer’s Disease are used to treat Autism in children. The drug donepezil used to treat dementia in Alzheimer’s patients is used to improve sleep quality in children with Autism. Another example is the use of the pain medication paracetamol in premature infants in the absence of the knowledge on the effects among this population. While decisions about off-label prescribing are usually informed by scientific evidence and professional judgement, there may be associated harms. There is growing recognition that children are not ‘little adults’ and their developing brains and bodies may react differently to those of fully developed adults. While doses for children are often calculated by scaling from adult dosing after adjusting for body weight, the stage of development of the child also affects responses to drugs. Babies have difficulties breaking down drugs due to the immaturity of the kidneys and liver, whereas toddlers are able to more effectively breakdown drugs.

The FDA requires data about drug safety and efficacy in children before issuing approvals for the use of drugs in pediatric populations. The best way to produce this evidence is through clinical drug trials. Historically, the use of children in research has been ethically fraught, with some of the early examples from vaccine trials, such as the development of the smallpox vaccine in the 1790s. Edward Jenner, who developed the smallpox vaccine, has famously been reported to have tested the vaccine on several young children including his own without consent from the children’s families. Over the next few centuries, many researchers would test new treatments including drugs and surgical procedures on institutionalized children. It was not until the early 20th century that these practices were criticized and debate began over the ethical use of children in research. Today, in general, the ethical guidance for inclusion of children in research specifies that individuals unable to exercise informed consent (including minors) are permitted to participate in research providing informed consent is gained from their parent or legal guardian. In addition to a guardian’s informed consent, assent (‘affirmative agreement’) of the child is also required where appropriate. Furthermore, research protocols involving children must be subject to rigorous evaluation by Institutional Review Boards to allow researchers to conduct their research.

Contributing to the lack of evidence of the effects of drugs in children is that fewer clinical trials are conducted in children than adults. One study reports that from 2005-2010, there were 10x fewer trials registered in the US for children compared to trials registered for adults. Recognizing the need to increase the number of pediatric clinical trials, the FDA introduced incentives to encourage the study of interventions in pediatric populations: the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA). The BPCA delays approval of competing generic drugs by six months and encourages NIH to prioritize pediatric clinical trials for drugs that require further evidence in children. The PREA requires more companies to have pediatric-focused drugs assessed in children. Combined, these initiatives have resulted in benefits such as improving the labeling of over 600 drugs to include pediatric safety information, such as approved use and dosing information. Noteworthy examples include two asthma medications, four influenza vaccines, six medications for seizure disorders and two products for treating migraines. However, downsides to these incentives have also been reported. Pediatricians have voiced concern over the increasing cost of some these drugs developed specifically for children, which have involved minimal innovation. For example, approval of liquid formulations of a drug used to treat heart problems in children has resulted in this formulation costing 700 times more than the tablet equivalent.

A further aspect that must be considered when conducting pediatric clinical trials is the large dropout rates of participants, and difficulty recruiting adequate numbers of children (especially for trials including rare disease populations) sometimes leading to discontinuation of trials. A recent report indicates that 19% of trials were discontinued early from 2008-2010 with an estimated 8,369 children enrolled in these trials that were never completed. While some trials are discontinued for safety reasons or efficacy findings that suggest changes in standard of care, many (37%) are discontinued due to poor patient accrual. There is insufficient research on the factors influencing parental decision-making for entering their child to a clinical trial and research into this area may lead to improvements in patient recruitment for these trials. This research must include or be informed by members of the community, such as parents of children deciding whether to enroll their child in a clinical trial, and disease advocacy groups. The FDA has an initiative to support the inclusion of community members in the drug development process. Through the Patient-Focused Drug Development initiative, patient perspectives are sought of the benefit-risk assessment process. For example, patients are asked to comment on what worries them the most about their condition, what they would consider to be meaningful improvement, and how they would weigh potential benefits of treatments with common side-effects. This initiative involves public meetings held from 2013-2017 focused on over 20 disease areas. While the majority of the diseases selected more commonly affect adults than children, some child-specific disease areas are included. For example, on May 4, 2017 public meeting was held on Patient-Focused Drug Development for Autism. The meeting included discussions from a panel of caregivers about the significant health effects and daily impacts of autism and current approaches to treatment.

While it is encouraging that the number of pediatric trials are increasing, ultimately leading to improved treatments and outcomes for children, there remain many challenges ahead for pediatric drug research. Future research in this area must explore parental decision-making and experiences, which can inform of the motivations and risk tolerances of parents considering entering their child to a clinical trial and potentially improve trial recruitment rates. This research can also contribute to ensuring that clinical trials are ethically conducted; adequately balancing the need for more research with the potential for harms to pediatric research participants.

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Written by sciencepolicyforall

May 24, 2017 at 5:04 pm

How Easy is it to Access Health Care in the US?

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By: Rachel F Smallwood, PhD

Source: pixabay

         Access to health care has been a concern as long as there has been health care, and it is one of the hot-button issues of health care policy debates. The recent repeal of the Affordable Care Act and passing of the American Health Care Act (AHCA) in the House of Representatives has again brought this debate front and center. The Congressional Budget Office’s analysis of the first iteration of the AHCA indicated that it would result in 24 million less people having health insurance by 2026. It would also place more of the financial burden on people making less than $50,000 per year. However, substantial changes were made to parts of the bill before it passed in the House, and there will likely be more if it is to be passed in the Senate. There is much debate and dissension on what level of access to health care should be provided by the government and whether health care is a right versus a privilege. In addition to that debate, there are other facets of the United States’ health care system that need examination and work to ensure access to health care.

There are many reasons a person may not have access to health care – not having health insurance is just one. To measure access to health care, one must first define it. Is there some quality standard that must be met for treatment to be considered health care? How do we determine whether one person’s health care is equivalent to another’s? With health care measures that range from necessary, recommended but not dire, to completely elective, even these differences can be difficult to quantify. Most institutions collecting data on health care use a working definition like that set by the Institute of Medicine in 1993: access to health care means a person is able to use health care services in a timely manner to achieve positive health outcomes. This implies that a person can enter the health care system, physically get to a place where they can receive health care, and find physicians whom they trust and who can provide the needed services.

Indeed, there are differing opinions on what constitutes “access”, and this heterogeneity is further compounded by the multiple barriers to access. For example, with the recent AHCA proposal, many representatives spoke about separating the concepts of health care coverage and health care access, while others believe that the two are not separable. There are at least four factors that limit a person’s access to healthcare. The first barrier is the availability of health services; if the necessary health care is not provided within reasonable traveling distance of a person seeking services, none of the other factors matter. The other three factors are personal barriers such as a person’s perceptions, attitudes, and beliefs about their own health and health care, organizational barriers such as referrals, waiting lists, and wait times, and financial barriers such as inability to afford insurance, copays, costs beyond deductibles, and lost wages.

The current policy in the United States is the Affordable Care Act, put into place under the Obama administration. One of the most contentious points of the law is its requirement that every person have health care coverage or pay a penalty. A 2015 survey released by the National Center for Health Statistics indicated a substantial drop in the percentage of the US population without insurance over the previous few years. There was a slight increase in the percentage of people with a usual place to go for health care (i.e. a primary care provider or clinic for regular check-ups), and a decrease in the number of people who failed to obtain needed health care due to cost, but simply requiring everyone to purchase health insurance did not induce a commensurate rise in people gaining access to health care, in accordance with the steps and measures discussed by the Agency for Healthcare Research and Quality. Additionally, there have been substantial increases in premiums, which means that those consumers still have a significant financial barrier to health care.

The numbers and policies referenced above address the country as a whole, but statistics vary widely across regions of the United States. US News ranked states on their access to health care using six metrics: child wellness and dental visits, adult wellness and dental visits, health insurance enrollment, and health care affordability. Some examples of the ranges seen between states in these measures are that 20% of adults do not have regular checkups in the highest ranked states, while around 40% do not have regular checkups in the lowest ranked states. In the highest ranked state for affordability, the fraction of people who needed to see a doctor but could not because of cost was around 7%, while in the lowest ranked state this percentage was just under 20%. While some of this is due to the differing demographics and living conditions from state to state, the discretion and freedom that states have in applying health care laws also factor in.

When comparing to other similar (high-income) nations, the United States falls short on access to health care. Although the Affordable Care Act improved access to health insurance, the US is still lagging when it comes to its residents receiving actual care. This is partially due to fewer physicians practicing general medicine in the US. In 2013, the US ranked below all other Organization for Economic Co-operation and Development countries, except for Greece, for the density of general practitioners per 1,000 people. A related measure showed that the US also had a lower percentage of physicians choosing general practitioner/primary care as their specialty than all other 35 countries. These countries are all World Bank-categorized high-income countries except for Mexico and Turkey, which are upper middle-income (and had better stats than the US). This disparity has been noted in the US and is driven by many factors including physician salaries, patient loads, and medical education emphasis (or lack thereof) on primary care. This shortage also disproportionately affects rural areas, likely contributing to some of the state-to-state variability noted above.

The United States is struggling when compared with similar nations to provide health care access to its citizens. The reasons for this struggle are multifaceted, including access to health insurance, financial barriers, and lack of primary care physicians. The political tensions and opposing principles held by individuals can also be barriers to working toward a more accessible health care system. We should be focused on developing a health care system where all can reasonably obtain health insurance, where health care costs are not prohibitively expensive, and medical education should emphasize the importance of primary care in our nation’s health and communicate the need for practitioners in under-served areas. Shedding light on these areas for improvement will allow people to work together to address our weaknesses and create a system that improves and sustains the health of our nation.

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Written by sciencepolicyforall

May 19, 2017 at 10:16 am

How GMOs Could Help with Sustainable Food Production

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By: Agnes Donko, PhD

World Population estimates from 1800 to 2100

           The world population has exceeded 7.5 billion and by 2050 it is expected to reach 9.7 billion. The challenge of feeding this ever-growing population is exacerbated by global warming, which may lead to more frequent droughts or the melting of Arctic sea and Greenland ice. The year 2016 was the warmest ever recorded, with the average temperature 1.1 °C above the pre-industrial period, and 0.06 °C above the previous record set in 2015. According to the United Nations, the world faces the largest humanitarian crisis in East-Africa since the foundation of the organization in 1945, particularly in Yemen, South Sudan, Somalia and Nigeria. In these countries, 20 million people face starvation and famine this year because of drought and regional political instability.

How could genetically modified organisms (GMO) help?

The two main GMO strategies  are the herbicide-tolerant (HT) and insect-resistant crops. HT crops were developed to help crops survive application of specific herbicides (glyphosate) that would otherwise destroy the crop along with the targeted weeds. Insect-resistant crops contain a gene from the soil bacterium Bt (Bacillus thuringiensis) that encodes for a protein that is toxic to specific insects, thus protecting the plant. Insect-resistant crops can reduce pesticide use, which decreases the ecological footprint of cultivation in two ways – first by reducing insecticide use, which in turn will reduce the environmental impact of insecticide production, and second by reducing the fuel usage and carbon dioxide (greenhouse gas) emission, by fewer spraying rounds and reduced tillage. Thus, adoption of GM technology by African nations and other populous countries like India could help with sustainable agriculture that can ameliorate the burden of changing climate and growing populations.

In developed nations, especially in the US, GM technology has been widely used since the mid-1990s, mainly in four crops: canola, maize, cotton and soybean. GM crops account for 93 percent of cotton, 94 percent of soybean and 92 percent of corn acreage in the US in 2016. Although the appearance of weed resistance to glyphosate increased herbicide usage, in 2015 the global insecticide savings from using herbicide-tolerant maize and cotton were 7.8 million kg (84% decrease) and 19.3 million kg (53% decrease), respectively, when compared with pesticide usage expected with conventional crops. Globally these savings resulted in more than 2.8 million kg of carbon dioxide, which is equivalent to taking 1.25 million cars off the road for one year.

Another way in which GM crops can help sustainable food production is by reducing food wastage in developed nations. The Food and Agriculture Organization of the United Nations (FAO) estimates that one-third of all food produced for human consumption in the world (around 1.3 billion tons) is lost or wasted each year, which includes 45% of all fruits. For example, when an apple is bruised, an enzyme called polyphenol oxidase initiates the degradation of polyphenols that turns the apple’s flesh brown. But nobody wants to buy brown apples, so the bruised apples are simply trashed. In Arctic apples, the level of the enzyme is reduced by gene silencing, thereby preventing browning. The Arctic Apple obtained USDA approval in 2015, and is expected to reach the market in 2017.

In 2015, the FDA approved the first GMO food for animal consumption, a genetically modified Atlantic salmon called AquAdvantage. Conventional salmon farming has terrible effects on the environment. However, AquAdvantage contains a growth hormone regulating transgene, which allows for accelerated growth rates, thus decreasing the farming time from 3 years to 16-18 months. This would dramatically reduce the ecological footprint of fish farming, leading to more sustainable food production. Even though FDA did not find any difference in the nutritional profile between AquAdvantage and its natural counterpart, AquAdvantage will not hit the U.S. market any time soon, because the FDA banned import and sale until the exact guidelines on how this product should be labelled are published.

This FDA action was initiated by bill S. 764 that was signed by former president Barack Obama in 2016. Bill S. 764 requires food companies to disclose GMOs without necessarily using a GMO text label on packaging. They may choose to label GM ingredients with a symbol or a QRC (quick response code) that, when scanned by a smartphone, will lead the consumer to a website with more information on the product. But this requires the consumer to have both a smartphone and access to the internet. The bill also has ‘lax standards and broad definition’. For instance, if the majority of a product contains meat, but some other less significant ingredient is produced from GM crops, then it need not be labelled. Oil extracted from GM soybean, or starch purified from GM corn are exempt from labeling, because they were only derived from GM sources, but no longer contain any genetic material in them. Contrarily, in the European Union (EU), regulations require that the phrase “genetically modified” or “produced from genetically modified [name of the organism]” must appear clearly next to the ingredient list. If the food is not packaged, the same phrase must be on the food display or next to it. The EU also unequivocally determines the level of GMO (below 0.9 %) in conventional food or feed that is exempt from labelling.

Despite its controversial guidelines for GMO labeling, bill S. 764 could end the long-fought battle of Just Label It campaign. The bill was a huge step toward the right to know, which will let individuals decide if they want to consume GM foods or not. GMOs can significantly support sustainable food production and reduce the destructive environmental impact of humanity, but only if we let it.

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Written by sciencepolicyforall

May 12, 2017 at 5:13 pm

How Science Policy Affects Pandemic Pathogen Research

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By: Samuel Porter, PhD

         In 2012, a pair of studies were published in Nature and Science weeks apart igniting one the biggest national debates about science in recent memory. These studies demonstrated that a few mutations in the highly pathogenic H5N1 strain of influenza virus (colloquially known as “bird flu”) could enable it to be transmitted through the air to mammals. At the heart of controversy was the question of whether scientists should be creating more virulent and/or pathogenic strains of deadly viruses in the lab. This controversial research is known as “gain of function” studies.

Critics claimed that the research was too dangerous that the risk of an accidental or deliberate release of these lab strains was far greater than the scientific and public health benefits. In an attempt to respond to the growing concern over their work, the community of researchers working with these pathogens voluntarily agreed to suspend this gain of function research for 60 days to discuss new policies on conducting the research safely.

But that was not enough to satisfy critics of the research, who continued to lobby the Obama administration to take official action. On October 17, 2014 the White House Office of Science and Technology Policy (OSTP), abruptly announced a pause on all U.S. Government funding of gain of function research on influenza, Middle East respiratory syndrome (MERS), and severe acute respiratory syndrome (SARS) coronavirus until the National Science Advisory Board for Biosecurity (NSABB) could make recommendations for policy regulating the research going forward. The NSABB was formed in 2005 (in the wake of the anthrax attacks in 2001), and is composed of scientists from universities around the nation, and administrators from 14 separate agencies in the federal government. The board reports to the Secretary for Health and Human Services (HHS) and is tasked primarily with recommending policies to the relevant government entities on preventing published research in the biological sciences from negatively impacting national security and public health.

The move drew harsh criticism from researchers in the field, many of whom thought that it was too broad. They claimed it would jeopardize their ability to predict, detect, and respond to potentially emerging pandemics. In the private sector, several companies said that the order would prevent them from working on new antiviral drugs and vaccines. Furthermore, many young scientists worried that an inability to do their experiments could jeopardize their careers. In an effort to bring attention to the issue, many scientists (including the two flu researchers whose research triggered the pause) formed the group Scientists for Science, which advocates against blanket bans on research. In addition, researchers were especially upset by the recommendation of the NSABB to censor the publications resulting from the experiments due to fears that this research could have a “dual use” that would threaten national security. However, not all researchers in the field support gain of function research (the opposition group is called Cambridge Working Group) and maintain that the risks of the research outweigh benefits.

The moratorium lasted until January 9th, 2017, when the OSTP released the guidelines for funding this research in the future. The new rules are essentially the same recommendations put forth by the NSABB seven months earlier. The NSABB had concluded that these studies involving “potentially pandemic pathogens” (PPP) do indeed have important benefits to public health, but warranted additional screening prior to funding approval. It directed federal agencies to create a pre-funding review mechanism using eight criteria (including whether the pathogen is likely to cause a naturally occurring pandemic, and if there are alternative methods of answering the scientific question). The results of these reviews must be reported to the White House OSTP. Importantly, the policy was implemented in the final days of the Obama administration rather than leave it to the incoming Trump administration, who, as of this date, has yet to fill nearly any top science positions, and may not have issued guidance for months, if at all.  Researchers welcomed the decision to finally lift the ban, but questioned when the projects would be allowed to resume.

What can we learn from this situation from a science policy perspective? First, we must learn not to overreact to hysteria regarding the risks of this type of research. Indeed, there are risks in performing research on potentially pandemic strains of influenza and other pathogens, as there are with other types of research. But issuing overly broad, sweeping moratoriums halting ground breaking research for years is not the answer, nor is government censorship of academic publication. While in the end, the studies were given the green light to resume, and were published without modification, there is no making up for the lost time. These studies are not machines than can simply be turned on and off on a whim without repercussions. When we delay research into learning how viruses become pandemic, we hurt our ability to detect and respond to naturally occurring outbreaks. Additionally, when American scientists are prevented from doing research that other countries are still pursuing, American leadership in the biomedical sciences is at a competitive disadvantage. (The European Academies Science Advisory Council also recently updated its recommendations for PPP research in 2015, but did not institute a moratorium.) What we learn from these studies could potentially save countless lives. Secondly, the freedom to publish without any government censorship must be valiantly defended in any and all fields, especially with a new administration with an aggressively anti-science and anti-climate stance. Lastly, the scientific community must do a better job educating the public both on the importance of these studies from a public health perspective, and on the precautions put into place to ensure that these studies are conducted safely.

In the future, there will inevitably be debates over the safety or ethics of the latest experiments in a particular field. In attempting to wade through the murky waters of a complex controversy, science policy makers should make decisions that balance public health, safety, and ethics, rather than reactionary policies like censorships and moratoriums.

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Written by sciencepolicyforall

April 21, 2017 at 8:47 am

Scientific Activism: Voting to Speed Up Discovery with Preprint Publishing

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By: Thaddeus Davenport, PhD

Source: Public Library of Science, via Wikimedia

         The election of Donald Trump to the Oval Office and the early actions of his administration have sparked a wave of protests in support of women’s rights and immigration, among other issues. Like other citizens, scientists have some cause to be concerned about the administration’s early actions that reveal a general disregard for facts and scientific evidence. In response, organizers have planned the March for Science for this Saturday, April 22nd, as an opportunity for people to gather in cities around the world to voice their support for factual information and scientific research. And while it is important to denounce the actions of the Trump administration that are harmful to science and health, it may be even more critical to acknowledge the underlying partisan divisions that created a niche for his rhetoric and to begin the difficult work of bridging the divide. For example, a Pew Research Center poll from 2015 indicates that 89% of liberal Democrats believe government investment in basic science pays off in the long-run, while only 61% of conservative Republicans feel the same way. Additionally, American adults with less knowledge of scientific topics are more likely to believe that government funding of basic science does not pay off. This suggests that improved science education and outreach will be important in building public support for scientific research. However, scientists often lead very busy lives and have little time outside of their professional activities to devote to valuable pursuits like science outreach. How, then, might scientists work towards building a better relationship with the public?

The products of science – knowledge, medicines, technology – are the clearest evidence of the value of research, and they are the best arguments for continued research funding. Efficiency in science is good not only for scientists hoping to make a name for themselves, but also for the public, who as the primary benefactors of academic research, must benefit from the products of that research. If taxpayers’ demand for scientific inquiry dissipates because of a perceived poor return on their investment, then the government, which supposedly represents these taxpayers, will limit its investment in science. Therefore, in addition to communicating science more clearly to the public, scientists and funding agencies should ensure that science is working efficiently and working for the public.

Information is the primary output of research, and it is arguably the most essential input for innovation. Not all research will lead to a new product that benefits the public, but most research will yield a publication that may be useful to other scientists. Science journals play a critical role in coordinating peer review and disseminating new research findings, and as the primary gatekeepers to this information, they are in the difficult position of balancing accessibility to the content of their journals with the viability of their business. This position deserves some sympathy in the case of journals published by scientific societies, which are typically non-profit organizations that perform valuable functions including scientific outreach, education and lobbying. However, for-profit journals are less justified in making a significant profit out of restricting access to information that was, in most cases, obtained through publicly-funded research.

Restricting access to information gathered in the course of research risks obscuring the value of research to a public that is already skeptical about investing in basic science, and it slows down and increases the cost of innovation. In light of this, there is growing pressure on publishers to provide options for open-access publishing. In 2008, the National Institutes of Health adopted a public access policy, which requires that “investigators funded by the NIH submit or have submitted for them to the National Library of Medicine’s PubMed Central an electronic version of their final, peer-reviewed manuscripts upon acceptance for publication, to be made publicly available no later than 12 months after the official date of publication: Provided, that the NIH shall implement the public access policy in a manner consistent with copyright law.” This policy was extended through an executive order from the Obama Administration in 2013 to include all federal agencies with research budgets greater than $100 million, with additional requirements to improve accessibility.

These requirements are changing scientific publishing and will improve access to information, but they remain limited relative to the demand for access, as evidenced by the existence of paper pirating websites, and the success of open access journals like PLoS and eLife.  Additionally, other funding agencies like the Bill and Melinda Gates Foundation and the Wellcome Trust have imposed even more stringent requirements for open access. Indeed, researchers will find a spectrum of open-access policies among the available journals, with the most rapid access to information allowed by so-called ‘preprint’ publishers like Given that many research manuscripts require months or years of revision and re-revision during submission to (usually multiple) journals, preprint servers accelerate the dissemination of information that is potentially valuable for innovation, by allowing researchers to post manuscripts prior to acceptance in a peer-reviewed journal. Many journals have now adopted explicit policies for handling manuscripts that have been previously submitted to bioRxiv, with many of them treating these manuscripts favorably.

Given that most journals accept manuscripts that have been previously published on bioRxiv, and some journals even look to bioRxiv for content, there is little incentive to submit to journals without also submitting to bioRxiv. If the goal is, as stated above, to improve the transparency and the efficiency of research in order to make science work for the public, then scientists should take every opportunity to make their data as accessible as possible, and as quickly as possible. Similarly, funding agencies should continue to push for increased access by validating preprint publications as acceptable evidence of productivity in progress reports and grant applications, and incentivizing grant recipients to simultaneously submit manuscripts to preprint servers and peer-reviewed journals. Scientists have many options when they publish, and by voting for good open-access practices with their manuscripts, they have the opportunity to guide the direction of the future of scientific publishing. These small, but important, actions may improve the vitality of research and increase the rate at which discoveries tangibly benefit taxpayers, and, in combination with science outreach and education, may ultimately strengthen the relationship between scientists and the public.

March for Science this Saturday, if it feels like the right thing to do, and then strive to make science work better for everyone by sharing the fruits of research.

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Written by sciencepolicyforall

April 20, 2017 at 11:44 am

Healthcare Policy – What’s in Store for Our Future Healthcare Needs?

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By: Emily Petrus, PhD

       There’s no question that the US spends too much on healthcare – in 2015 it cost 18% of its GDP, equivalent to $3.2 trillion dollars. In fact, we spend more on healthcare to cover just 34% of our population via Medicare and Medicaid than other countries who cover their entire population with universal healthcare. Most people assume that this higher spending equals better health, but unfortunately this isn’t the case.

According to a 2015 Commonwealth Fund survey, the US has the highest infant mortality and obesity rates and the lowest life expectancy of the top 13 Organisation for Economic Co-operation and Development (OECD) countries. In addition, we have the highest rates of prescription drug use, amputation due to diabetes mismanagement, and the second highest death rate from ischemic heart disease. Our relatively small percentage (14.1%) of people over age 65 also have the highest rate of at least two chronic illnesses per person. These numbers are estimated to increase as baby boomers age, so the outlook isn’t good when considering how many elderly people we can expect to suffer from chronic health issues.

However, it’s not all doom and gloom – we are in the top 3rd for surviving cancer, boast the lowest smoking rates, and have the highest access to diagnostic imaging services (such as MRI and CT scans). In this light, it makes sense that we spend more, have better access to expensive technology, and use more expensive prescription drugs. Another way to slice the data paints a different picture. The sickest 5% of the population accounts for 50% of medical spending, and accounts for 60% of spending on prescription drugs. Together these data indicate that the US could be in better shape if we had a healthier population.

How could we make the population healthier? Let’s consider that the determinants for health are 30% genetics, 70% behavior, environment and social factors, only 10% is mediated by healthcare. Other OECD countries spend significantly more on social services such as supportive housing, employment programs, retirement and disability programs. Social services are especially beneficial for people in lower income brackets, who incidentally have the poorest health in the US. The life expectancy for the poorest Americans is about 13 years less than the wealthiest. Racial disparities also contribute to gaps in healthcare outcomes for Black, Hispanic, Asian and American Indians/Alaskan Native Americans, all of whom experience worse medical care. The parameters measured included access to care, effective communication with medical staff, and a specific source of ongoing medical care, such as a primary care physician. It is estimated that these disparities amount to billions of dollars in economic loss – $35 billion in excess health care expenditures – for example, a trip to the emergency room for something that could be treated by better access to a primary care physician. Expanding Medicaid would increase medical access to poor and disadvantaged minority groups, for example, Blacks in the south. However, many states thatch have high at-risk populations decided not to expand Medicaid. Spending more on social services aimed at improving people’s health seems to be working in other OECD countries, and the National Academy of Medicine recommends the US increase spending in these areas.

Social services are unlikely to gain support from conservatives, so spending in this area is unlikely to be supported by the current administration. However, there are other areas in healthcare that can gain bipartisan support. 30% of medical expenses are considered wasteful – meaning they are for unnecessary services, fraud, and sky high pharmaceutical or administrative costs. Medicare has already saved billions of dollars by reducing overpayments to private insurers and tying medical provider payments to quality of care. Overall a goal of those involved in healthcare reform should seek to follow this example of prioritizing value over volume of care, which will translate to better outcomes at lower costs for patients and taxpayers.

So what did the Affordable Care Act (ACA, also known as Obamacare) achieve since it was passed in 2010? In the time leading up to the ACA, 82% of the American public wanted healthcare reform. Private insurance premiums were rising 10% per year, and insurance didn’t have to cover expensive benefits, so many plans came without services like mental health or maternity care. Maternity care is not just a women’s issue, healthier pregnancies result in healthier babies who become part of our population. Before the ACA, 50 million (17%) of the population was uninsured; by 2016 20 million people had gained health insurance, leaving only 10% of our population uninsured. Women and people with pre-existing conditions can’t be denied coverage or charged more by insurance companies. Lifetime spending caps were removed, meaning if you were a sick baby in the ICU you can’t be denied coverage for the remainder of your life. The most popular part of the bill allows young adults to stay on their parents’ insurance until age 26, which reduced the uninsured rate for young adults by 47%. Finally, tax credits made health insurance through exchanges more affordable for those at or below 400% of the federal poverty line.

Those are the good parts about the ACA – here’s the bad news.  High deductible plans have increased from 10% of plans offered in 2010 to 51% of plans in 2016, meaning people buying insurance can expect to pay at least the first $1,000 per year out of pocket. If it seems that premiums are jumping, they are: they rose 20% from 2011 to 2016. It’s easy to blame the ACA for rising premiums, but if we consider that premiums rose 10% per year before the ACA, 20% in 5 years doesn’t sound so bad. Some specific states are expecting huge increases, for example Oklahoma will see a 42% increase in 2016. Part of the reason premiums are rising is because healthcare gets more expensive each year – it outpaces inflation and wages. Insurance companies are also losing money because they have enrolled more sick, expensive people than they expected to when they set prices. The ACA attempted to mediate the sticker shock for insurance companies by setting up “risk corridors” to help shoulder the burden, but that part of the bill was scuttled for political reasons, and now insurance companies are passing the buck to consumers. Regarding taxes, under the ACA, those without insurance will face a penalty fee double that of the 2015 amounts.

A central campaign promise of Trump and Republicans was to repeal the ACA and provide better and more affordable coverage for all.  The American Health Care Act (AHCA) proposed several weeks ago by Republicans was a repeal and replace bill which was unpopular from the start. The AHCA was unpopular with conservatives for not going far enough to repeal the ACA, while moderates worried about the 20 million people, including their constituents, being denied or outpriced from insurance due to some elements of the bill. The AHCA removed the mandates requiring insurance companies to provide essential health benefits. This could lower premiums but insurers could also reduce services, leading to “junk plans”. Additionally, tax credits for people buying insurance would be significantly lower than current levels, making insurance too expensive for many middle-income people. Medicaid coverage was also proposed to shrink, resulting in less coverage for poor Americans. Finally, eliminating the community rating of the ACA would enable insurance companies to charge older and sicker people higher premiums, essentially pricing those who need insurance the most out of the market. The AHCA proposed to ameliorate this problem by providing larger tax credits to older individuals and setting up pools of high risk people subsidized by the government.

It is safe to assume that the Republican controlled House, Senate and the White House will try again to present bills that modify the ACA. However, it remains to be seen if they will try a bipartisan effort to fix certain parts of the bill that are flawed, or repeal and replace the ACA with something completely new.

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Written by sciencepolicyforall

April 14, 2017 at 9:22 am

The Trans-Pacific Partnership and its Impact on Pharmaceutical Affordability

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By: Shakira M. Nelson, PhD, MPH

        For many, the Trans-Pacific Partnership (TPP) was a point of great debate during the 2016 Presidential primaries and election. As a simplified explanation, the TPP is a free-trade agreement involving the United States, Canada, Australia, Japan, New Zealand, Mexico, Chile, Peru, Brunei, Malaysia, Singapore and Vietnam, intended to “level the trading playing field” through the elimination of tariffs and other laws that create trade barriers. In its final form, the TPP would impact up to one-third of world trade and 40% of the global gross domestic product. Many who debated the ramifications of the TPP did so in the context of foreign policy interests. Although aligned with foreign policy, a major part of the TPP deals with intellectual property protection, and pharmaceutical drug development. If implemented, the effects of the TPP could greatly diminish public access to affordable medicines, both domestically and internationally. Moreover, the stronghold the TPP places on intellectual property could limit the development and marketing of less expensive options.

Intellectual property can be divided into two categories: industrial property and copyright. Patents, trademarks, and industrial design fall under industrial property. Patent development is a large part of scientists’ work, seen as almost a necessity to incentivizing innovation. Many argue that, without the ability to patent inventions and significant findings, scientists would not be able to generate profits used to sustain research and development; within the pharmaceutical industry, patents are the proverbial bread-and-butter. When in place, patents create a stronghold around the release of new chemical drugs, which prevents competition by generic brands. The standard length of time of a patent for a chemical drug is 20 years, which starts from the time the drug is invented.

Many new medicines under development today fall under the category of ‘biologics’. As the name suggests, biologics are treatments made from biological sources, and are very different from chemical drugs. Created to treat a multitude of diseases, including Ebola and cancer, biological sources include vaccines, anti-toxins, proteins, and monoclonal antibodies. Given their structural complexity compared to traditional drugs, and use of recombinant DNA technology, biologics are more difficult, and costlier to make. Moreover, manufacturers have a greater burden in ensuring product consistency, quality, and purity over time. This is done through certifying that the manufacturing process remains the same over time. Because of this, it is estimated that the price to manufacture biologics cost on average more than 22 times the price of chemical drugs. Current laws state that generic biologic development, known as biosimilars, cannot be approved until 12 years after the branded product has been approved – this is known as an exclusivity period. This was enacted under the Biologics Price Competition and Innovation Act of 2009, by the Food & Drug Administration (FDA).

The challenge with current policies is establishing a period-of-time that balances the need for companies to generate profits and cash flows, which will incentive them to conduct more research and compensate them for the extensive manufacturing processes, with the need to provide greater access through launching generic drugs and biosimilars. The trouble with the proposed policies of the TPP agreement is that they seem to embolden the pharmaceutical companies by introducing changes that would prevent competition from generics and biosimilars for longer periods of time than the current basic terms. The implications of this are far-reaching, as it may lead to a significant increase in the current costs of pharmaceutical drugs and biologics, hindering the health of the patients who rely upon these treatments.

Critics of the current system of patent length and biologic exclusivity periods fear that rather than incentivizing innovation, companies are being rewarded through their ability to charge higher amounts for drugs without the fear of competition on the market. Health policy experts concur, identifying policies such as the Hatch-Waxman Act of 1984 in allowing for the creation of drug monopolies, and “going too far in compensating the pharmaceutical industry at the public’s expense”. A report released in 2009 by the Federal Trade Commission stated that biosimilar development was more difficult to achieve than traditional generic drugs. For example, development requires comparisons to the original biologic, to prove efficacy and equivalence. Biosimilars must share the same mechanism of action, with no clinically significant differences in terms of safety or potency for the approved condition of use. The steps necessary to achieve this are significant, and therefore imposing a 12-year exclusivity period on biologics may be unnecessary. US Congressmen have pushed to compromise, floating an amendment to the TPP that would lower the exclusivity period to 8 years. However, critics and patients who rely upon drug competition to lower market prices, have protested this amendment stating that costs of new drugs and biologics are too high, and 8 years is too long of a length of time to wait for affordable generics and biosimilars to come on to the market.

The impact of decreasing the length of time it takes for biosimilars to come onto the market can be seen with Neupogen, a leukemia drug that was first approved by the FDA in 1991. Delivered via injection, Neupogen costs patients $3,000 for 10 injections. With injections needed daily, this drug could carry a price tag of well over $100,000 per year. It wasn’t until recently, however, that the first biosimilar was approved on the US market. The biosimilar, Zarxio, was approved as a leukemia drug and is priced at more than $1000 less than Neupogen. This pricing has the potential to decrease the yearly costs of this drug from $100,000 with Neupogen to $55,000-$75,000. Further evidence of these financial savings was provided by the Rand Corporation, which predicted a savings of over $44 billion over 10 years with an increased approval of biosimilars, for patients who rely upon these specific cancer treatments.

Internationally, the policies of the TPP also have far reaching effects on the availability and costs of pharmaceuticals. The 12-year exclusivity period would be imposed upon the other countries involved in the TPP, where currently for some, such as Brunei, there is no current exclusivity protection. By imposing the 12-year period, global competition could become restricted. Additionally, the TPP proposes other key patent protections that play a bigger role on the international market. One protection, known as evergreening, allows drug companies to request patent extensions for new uses of old drugs. The immediate effect of this is an extension of monopolies on drug sales for minor reasons. The second protection allows pharmaceutical companies to request patent extensions if it takes “more than 5 years for an application to be granted or rejected.” Advocacy groups fear that the price of drugs would undermine the efforts of health initiatives, such as the Global Fund to Fight AIDS, Tuberculosis, and Malaria. These initiatives rely upon price competition to manage costs, with the availability of cheap generics helping drive costs down.

Although the current administration has ended the USA’s association with the Trans-Pacific Partnership, it is important to note that other countries may try to implement some of the policies, affecting the availability and affordability of drug treatments. To decrease this burden, the US could work to assist in negotiating exceptions for the poorer and smaller countries, to help them meet any challenges they may come up against. Within the US itself, it is important for policies, laws and any future trade agreements to be modified, with more of a focus on the affordability and regulation of drugs and biologics. Imposing price controls may offer a modest benefit, but may not be a long-term solution. A focus on lowering the patent length for new drugs and biologics can be an immediate step. Although the push back from pharmaceutical lobbyists will be substantial, alleviating the financial burden on families afflicted with cancer and diseases should be the focus.

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