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Clinical Trials Policy Revision: For Better or Worse

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By: Jenn L. Nguyen, Ph.D., M.P.H.

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source: pixabay

As the largest public funder of biomedical research in the United States, the NIH wants to ensure that conducted trials are relevant to health priorities of the US, trials are conducted efficiently and are not duplicates of previously conducted trials, and trials contribute to scientific knowledge. In an editorial in 2016, NIH leaders noted a need for quality and efficiency improvements to clinical trials. NIH has introduced several initiatives, to enhance clinical trials stewardship by addressing accountability, transparency, efficiency, and dissemination. However, along with the widely acknowledged improvements some recent changes may hinder the pursuit of scientific knowledge.

To address accountability, all investigators and staff conducting and overseeing clinical trials must take the Good Clinical Practice (GCP) training. The training is mandatory for individuals involved with the design, conduct, oversight, or management of clinical trials. While the training may not be sufficient by itself, it does provide a standard of knowledge, a base of knowledge, standards, and guidelines for all clinical trials.

The second change requires that all grant applications for clinical trials be submitted under clinical trials specific funding opportunity announcements (FOA). Investigators interested in conducting a clinical trial can no longer submit under parent funding announcements, which made identifying clinical trials more difficult in the past. The FOAs will list specific review criteria for reviewers to consider clinical trials-related information, such as focus on the rationale, design, and operational and analysis plans. This new policy will increase NIH accountability and efficiency, as it will ensure that required information is submitted with each clinical trial application, allow staff to better track clinical trial proposals and study, and allow staff to uniformly apply appropriate review criteria.

A substantial change, however, is the limited eligibility of trainees to conduct interventional social science research, Institutional training (T) awards, which provide money to institutions for workforce training, do not allow money to be given to trainees involved in clinical trials (the exception is for D43s and K12s), Fellowship (F) awards, which support individual trainees,  do not support trainees involved in independently conducted clinical trials, but trainees can propose a research experience with a sponsor/co-sponsor.  For Career Development (K) awards, applicants may apply to either FOAs that specify “clinical trials required” or FOAs that are for “no independent clinical trials.” Scientists are concerned this may limit postdocs and students to get support for their fellowships and adequate career training.

To further address efficiency and accountability, applications must be submitted using a clinical trials protocol template that consolidates information from multiple forms, has structured data fields, and will collect information at the study level. This requirement will ensure that all investigators will submit the same information. In addition, the forms will contain fields forcing investigators to be clear and concise about their analytical and dissemination plans.

Addressing efficiency, NIH now requires use of a single Institutional Review Board (IRB) to review multisite studies. Prior, each institute involved with the study required duplicate or multiple IRB reviews, which involved the redundant assembly of experts to assure that the same proposed study was in line with the rights and protections of human and animal research subjects. Multiple reviews resulted in delays and at times, conflicting reviews. Guidance to establish a single IRB on record has been published.

Finally, there are significant changes for registration and reporting of clinical trials to address accountability, transparency, and dissemination. Investigators are now required to register their clinical trial(s) in the ClinicalTrials.gov database within 21 days of enrollment of the first participant. NIH makes the argument that this effort may help reduce the number of trials that fail, as it will require scientists to disclose their results even if the studies do not support their hypotheses urthermore, all investigators must adhere to the NIH policy on Dissemination of NIH-clinical trials. There have been longstanding concerns that investigators are not reporting all results (especially negative or non-significant results, not reporting results in a timely manner, and even sometimes, deviating from their own research protocol.

Along with these initiatives, The National Institutes of Health (NIH) broadened what was considered a clinical trial: “a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes.” Adaption of this updated definition did not take effect until earlier this year and has alarmed some scientists. Clinical trials have been traditionally understood as experiments or observations for/in clinical settings to answer three questions: 1) Does the proposed treatment/intervention work? 2) Is the proposed treatment or intervention more effective than other treatments? 3) Are there side effects?

Scientists critical of the new definition first and foremost recognize and appreciate the motivation for NIH to increase transparency and replicability, specifically efforts for pre-registration, data sharing, and protocol sharing of trials. Yet, many scientists who conduct basic and behavioral research disagree agree that their work and studies should now be considered clinical trials. These scientists, and even scientific associations, remarked that the new clinical trials definition is too broad and traditional criteria to evaluate a trial might be inappropriately applied to their proposal. There is also concern that these changes will increase the administrative and bureaucratic burden for many scientists, specifically for exploratory scientists. To address and alleviate concerns, NIH released a set of case studies to help scientists identify and understand what is considered a clinical trial and must adhere to all the changes in the policy. While this effort provided clarification, many scientists are calling for NIH to hold further conversation with the extramural community.

While scientists recognize the need and laud NIH’s effort to address clinical trials stewardship, many of the same scientists are worried that these benchmarks set the wrong standards for success and rigor. Scientists are also worried about the additional administrative burden these changes will bring. As NIH enforces the policies, they have promised to monitor trouble issues and work with the community to find a solution without compromise.

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Written by sciencepolicyforall

February 20, 2018 at 3:52 pm

The impact of the growing student loan burden on graduate education

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By: Rebecca McPherson, Ph.D.

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Every individual has his or her reason for pursuing a graduate degree. For many, a graduate degree is a path to a specific career; for others, it is simply a personal goal; and at times, it is a requirement for job advancement. Although the benefits of a higher degree are as numerous as the types of degrees and disciplines offered, there is one unifying theme across higher education: rising and often unavoidable student loan debt. Forbes reported that in 2017, the student loan debt in the U.S. had reached an astonishing $1.3 trillion. Of the 44.2 million U.S. borrowers with some type of student loan debt, over 2 million owe at or more than 100,000 dollars. At the top of this mountain of debt are U.S. graduate students, a quarter of whom will borrow close to $100,000 to complete their education,  while a tenth will end up borrowing upwards of $150,000.

Educational debt can extend beyond the typical student loan programs. While tuition poses a major cost to the student, many may not be able to make enough money through stipends or part-time work to meet their cost of living. The Report on the Economic Well-Being of U.S. Households in 2016-May 2017 showed an increase in the rate of borrowing for educational related expenses. Although student loans remain the primary source of borrower debt, credit cards, home-equity loans, and private loans also make up a portion of the total educational debt burden.

With a climate of ever-increasing educational costs and associated debt, many current, past, and future students are left asking, what is being done to curb the impact of mounting educational debt? The Institute for College Access & Success (TICAS) is responsible for enacting and overseeing the National Agenda for the Student Debt Policy in the U.S. Currently, TICAS focuses on risk-reduction strategies for student borrowers, including developing income-driven repayment plans, increasing access to Pell grants, and streamlining student borrower application processes.

The growing concern surrounding the ever-increasing student debt load is also being tackled at the state level. In recent years, several individual states have introduced policies aimed at protecting the student borrower, especially from predatory private student loan lenders. The Private Student Loan Transparency and Improvement Act was passed in Oklahoma in 2013 and requires private and alternate student loan providers to be transparent in the lending and repayment conditions. The state of Connecticut followed by being the first state to implement a borrower’s bill of rights in 2015. Several states offer student loan forgiveness and repayment incentives to entice student loan debt holders to work in specific regions.

What does the rising burden of student debt mean, and what are the ultimate costs to the borrowers? Ultimately, the price of student debt means that many young adults will have to put off major life decisions. Among graduate student borrowers, 43 percent state difficulty in regularly meeting student debt repayment obligations, 61 percent  say they are unable to save for retirement due to debt load, and 45 percent lack the means to save for an emergency fund. The costs of tuition has substantially increased since the early 1990’s and continues to grow; this initial rise in costs was caused by a decline in state support to colleges and universities.   Although there are no exact numbers showing the impact to low income students, the pursuit of a higher degree may, for many students, be out of reach.

Although several policies at both the state and national levels exist, there are gaps in how to deal with and resolve the mounting student debt crisis, and questions remain as to the impact on future training.  TICAS has indicated several areas of the lending and repayment process in need of continued improvement, such as enhancing educational tax benefits, preventing predatory lenders, and promoting student borrower awareness.

Students pursuing advanced degrees – beyond a bachelor’s degree – shoulder the bulk of the student debt burden. At times, wage outcomes fall below borrower expectations in certain fields of study. This can have a serious economic impact for borrowers who are unable to meet loan repayment requirements. Borrowers who have completed a higher level of education at a not-for-profit institute, and consequently have a higher burden of debt (i.e., $100,000 or more) are less likely to fall behind or default on payments.  Counter to this, student borrowers who have attended a for-profit institute or did not finish their degrees are more likely to fall behind on repayments or default on their student loans.  Additionally, those seeking advanced degrees will find that they have a greater ability to borrow from the federal loan program, which often has high caps and few or no credit checks required before borrowing. This leads some to put the blame for the student debt crisis on the federal government, arguing that unchecked borrowing causes educational institutions to continually raise tuition and fees for graduate education.

As of February 2018, no new federal laws regarding the student loan debt burden have been passed. However, several initiatives have been proposed by the current administration in an effort to tackle the expanding costs of the student debt. One such recommendation is the expansion of the income-based repayment plan (IBR), which would consolidate all loans into a single repayment of 12.5 percent of income with loan forgiveness after 15 years. Other recommendations include lowering federal loan interest rates; eliminating the Public Service Loan Forgiveness (PSLF) program, which would instead give focus to the proposed IBR; and a pushing for tuition reduction by imposing educational institutes to cut administrative costs.  On the upcoming 10 year anniversary of the start of PSLF, many argue that cutting the program would dissuade many young professionals from entering into the lower paying public health service.

Ultimately, the impact of the student loan debt burden may negatively affect graduate level training. There are calls to protect borrowers from predatory lenders and proposed legislation to tackle the debt crisis. The willingness to take on large debt and possibly delay major life decisions lies with the individual. For now, the student debt debate continues.

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Written by sciencepolicyforall

February 6, 2018 at 12:13 pm

How do healthcare and health outcomes in the US compare to those of other developed countries?

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By: Vanessa Gordon-Dseagu, PhD

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For much of 2017, the Trump administration engaged in numerous attempts to repeal the Affordable Care Act – legislation that was considered by many to be the first step towards the introduction of universal healthcare within the US. As the debate continues over the role of the government in the provision of healthcare, comparisons with systems abroad may reveal important shortcomings within those found in the US. There are a number of methods to assess and compare the performance of a healthcare system; this essay focuses upon the key areas set out by the Peterson-Kaiser Health System Tracker – spending and health outcomes (those amenable to health-service provision) as well as the uninsured/access. By looking at the provision of healthcare internationally, we may learn key lessons in how to improve the US system.

Cost

In 2016 the US spent around 17% of its GDP on healthcare (around $3 trillion). This is almost double the amount spent by other industrialized nations, while the amount of healthcare used per capita is similar across countries. The key reason for this appears to be cost. Patients in the US pay far more for their healthcare to be provided than their international counterparts, with one study finding hospital prices in the US to be 60% more than those in Europe. One reason for this appears to be the high cost of insurance administration within the US system caused by, oftentimes, fractured and duplicative provision. Hospitals must employ large departments to properly bill insurance companies and verify insurance coverage, costs that are passed on to the patient. In comparison, nationally or regionally organized systems, free at the point of need, do away with the need for insurance administration. The Affordable Care Act (ACA) sought to address some of the administrative waste and duplication found within US healthcare provision by employing economies of scale and streamlining the insurance procedure.

The cost of prescription drugs in the US is also an issue for concern for patients and policy-makers alike. The US spends more per capita on prescription drugs than other countries within the Organization for Economic Cooperation and Development (OECD). A 2016 JAMA study found that per capita spending on prescription drugs was $858 a year within the US, compared with $400 for 19 other industrialized countries. One factor likely influencing this is the more rapid uptake of new and, thus, more expensive prescription drugs within the US compared with other OECD countries. While an equally important factor is the price at which the drugs are sold in the US compared to other countries – drug prices in the US are substantially increased compared with other markets. The experience internationally is that policies, such as centralized pricing and universal healthcare, as well as other price controlling strategies, are effective at reducing drug costs. While the majority of developed countries enact some form of price regulation, within the US, the federal government is legally prohibited from negotiating drug prices for its widely administered Medicare program. Further to this, for each new drug the US Food and Drug Administration approves, it upholds a market exclusivity period, ranging from six months to twelve years, in which it will not approve a generic form of the same drug, limiting access to cheaper generic drugs. This, combined with US patent law protections, means that, on average, a brand-name drug manufacturer can expect their product to be on the market between 12-16 years before  the introduction of generic competitors. In comparison, across Europe 13 countries have policies which make generic drug substitution mandatory, 13 have voluntary protocols, with only five forbidding the practice.

The uninsured and access to healthcare

The World Health Organization considers universal health coverage to be a system within which 1) individuals can use the health services they need; 2) the care is of a sufficient quality to be effective; 3) individuals are not exposed to financial hardship caused by accessing the services. Of the 35 countries within the OECD, 32 have healthcare legislation that is in keeping with the WHO definition of universal healthcare. The US is now the only high-income country not to offer universal health insurance coverage.

Although the introduction of the ACA reduced the number of uninsured in the US from 44 million in 2013 to a low of 28 million in 2016, by the end of 2017 the number of uninsured had again risen by 1.3%, an estimated 3.2 million individuals. The causes of this increase may relate to the uncertainty surrounding the potential repeal of the ACA under the current administration, the repeal of the individual mandate within the Trump tax bill, the exit of several insurers from the ACA market and increasing health insurance costs.

The benefits of healthcare coverage upon health outcomes are well documented and relate to access to preventive, ambulatory, primary and secondary healthcare. Further to this, access to healthcare is particularly important for those with complex or ongoing chronic conditions. A review of the evidence related to health insurance coverage and health concluded:

“There remain many unanswered questions about U.S. health insurance policy, including how to best structure coverage to maximize health and value and how much public spending we want to devote to subsidizing coverage for people who cannot afford it. But whether enrollees benefit from that coverage is not one of the unanswered questions. Insurance coverage increases access to care and improves a wide range of health outcomes. Arguing that health insurance coverage doesn’t improve health is simply inconsistent with the evidence.”

To overcome some of these issues, members of the House of Representatives (with a parallel bill introduced in the Senate in 2017) have been developing the Expanded & Improved Medicare for All Act, colloquially known as ‘Medicare for all’. The bill seeks to take the role of government in healthcare one step further than the ACA and introduce:

“A system in which a single public or quasi-public agency organizes health care financing, but the delivery of care remains largely in private hands. Under a single-payer system, all residents of the U.S. would be covered for all medically necessary services, including doctor, hospital, preventive, long-term care, mental health, reproductive health care, dental, vision, prescription drug and medical supply costs”.

Health outcomes

In relation to access, the US system has been found to fall short, particularly in the areas of primary care, prevention and management of chronic conditions. In its comparison of healthcare systems, the Commonwealth Fund concluded that the US ranked last on access. The insufficiencies of these areas of care contribute to the lower life expectancy found within the US compared with the majority of OECD countries – in their 2016 report, the US was found to rank 26 out of the 35 OECD countries. The same investigation found that the US also fell below the OECD average in several more nuanced indicators of health (for example maternal and infant mortality). In the same study undertaken by the Commonwealth Fund, the US ranked last out of 11 comparable countries for healthcare outcomes. The report also found that the US had the highest rate of mortality considered amenable to the provision of care. Compared with the other 10 countries under investigation, the US performed better in areas such as in-hospital mortality following a cardiovascular disease related event and five-year survival for some cancers. The lack of easily available preventative services may also contribute to the high, and increasing, rates of non-communicable diseases such as diabetes, as well as other conditions such as overweight and obesity, for which the US has the highest rates within the OECD.

Despite the substantially increased costs of healthcare in the US compared with international counterparts, the population is not experiencing a corresponding improvement in health outcomes. International comparisons suggest that tighter regulation of costs, particularly those related to insurance administration and prescription drugs, may go some way to addressing this issue. The introduction of universal coverage is also likely to allay some of the deficiencies inherent to healthcare provision within the US. How the issue of the provision of healthcare is broached by this, and future, administrations will have a substantial impact upon the health of the nation.

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Written by sciencepolicyforall

February 1, 2018 at 11:42 am

FDA stem cell therapy crackdown: a stem-free clinic

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By: Belinda Hauser, Ph.D.

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The building blocks of life are stem cells, they don’t kill or cure anything, but they promote regeneration. Stem cells are classically defined as an undifferentiated cell capable of giving rise to more stem cells or differentiating into any cell type. Stem cells have given scientists insight into understanding how cells function and dysfunction in development. Moreover, research in stem cell development has lead to promising treatment possibilities; it is believed that stem cells have the potential to repair or replace damage caused by age, injury or disease. However, stem cell therapies have been controversial, arising from the practice of isolating and culturing stem cell derived from human embryos, and later, introducing pluripotent stem cells from previously differentiated cell types. This controversy is entrenched in both political and ethical debates, broadly affecting the regulation of cord blood harvesting, human cloning and clinical trials.

Today, common stem cell therapy uses include blood transplants or bone marrow transplants. The Food and Drug Administration (FDA) has only approved hematopoietic progenitor cells, derived from umbilical cord blood, for use in the United States. Harvesting of cord blood is considered safe for the mother and baby since the blood is collected after birth. Stem cells collected from the blood of the cut cord are used to treat a variety of diseases including blood cancers such as leukemia, and lymphomas, and blood diseases of the immune system. Given the scarcity of approved options, patients desperately seeking therapy may turn to treatments that are illegal and potentially harmful. The FDA has gone to great lengths to evaluate the potential risk associated with new and current products through both animal and human studies in order to ensure safety in the use of biological products. Thereby, to determine the effectiveness and safety of new investigative products, well-controlled human studies must be designed and executed. This attention is applied to all clinical trials and is well documented. For example, the federal government requires all clinical trials to be cited and it is standard protocol for the National Institutes of Health (NIH) to list all clinical trials being conducted via Clinicaltrials.gov. This promotes awareness and gives consumers an opportunity to be well informed of all trials being conducted.

Preceding the FDA’s goal to develop and license stem cell therapies for patients and prevent consumer exploitation is their concern for consumer safety and education. In March 2017, the FDA provided materials to clarify the benefits and risks of stem cell therapies. They warned that when injected, unproven stem cell treatments present the risk of mobility of implanted cells, i.e. metastasis, risk of excessive proliferation, i.e. tumor growth, contamination, stem cell failure, or reaction of the injection site. Therefore, new investigative products must go through a rigorous protocol to determine their effectiveness and safety in well-controlled human studies.

In August 2017, the FDA cracked down on unscrupulous stem cell clinics, announcing increased enforcement of regulations and oversight of stem cells clinics across the country. For example, the FDA seized five vials of (live) smallpox virus vaccine from the California stem cell treatment centers in Rancho Mirage and Beverly Hills, California.  A Florida clinic, now called U.S. Stem Cell Clinic of Sunrise, Florida, caught the attention of the FDA after stem cell treatments it delivered to women with macular degeneration, an eye disease, caused permanent damage. Staff member used stem cells from fat isolated from each patient’s stomach and then injected cells into their eyes. A common practice of clinical trials is to pay human subject-volunteers to participate in studies. However, to receive this unproven treatment patients were required to pay $5,000 to receive the stem cell injections. Permitting patients to pay for participation is a topic of ethical debate for even the most scrupulously designed trials. The FDA issued a notice warning U.S. Stem Cell Clinic for marketing products without FDA approval and condemning their exploitation of consumers. An inspection performed  by FDA investigators found evidence of significant deviations from good manufacturing practices in manufacturing of at least 256 lots of stem cell products produced by the clinic. In an attempt to impede the investigation, the U.S. Stem Cell Clinic attempted to refused access of the FDA investigators to the employees of the clinic.  Ultimately, the clinic was cited for failure to establish appropriate written procedures to prevent contamination, risking infection of human subjects. It is required that U.S. Stem Cell Clinic comply and correct the failures stated in the warning letter. If the clinic fails to address the outlined issues, actions will be taken by the FDA, these include seizure, injunction and or prosecution.  Moreover, U.S. Stem Cell Clinic  administered the product both intravenously and directly into the spinal cord of patients hoping to treat a number of serious diseases (Parkinson’s disease, amyotrophic lateral sclerosis (ALS) heart disease, pulmonary fibrosis, and chronic obstructive pulmonary disease (COPD), all without FDA review or approval. In fact the FDA has not approved any biological products manufactured by U.S. Stem Cell Clinic for any use.

Overall, the challenge of regulation and compliance continues to loom over all stem cell clinics in the U.S.; however, the FDA is dedicated to enforcing continuous regulation, while educating and protecting U.S. consumers. The building blocks of life are stem cells, manipulated properly, they have the ability to treat disease without posing unacceptable risk. Safely figuring out how will take time.

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Written by sciencepolicyforall

January 17, 2018 at 11:43 am

Pharmaceutical Detailing: in the US the Details are Tied the Prescriber’s Name

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By: Allison Dennis B.S.

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While U.S. privacy laws protect patients from direct pharmaceutical marketing and shield their personal information from data mining, physicians are routinely identified based on their prescribing habits and targeted by pharmaceutical companies through personalized marketing campaigns. By their very nature, these campaigns aim to influence the behavior of prescribers. In other countries, including those protected by the European Union’s Data Protection Act, the personal identification of prescribers through medical data is strictly forbidden. However, in the U.S. these personalized campaigns are made possible by a robust pipeline of data sharing.

The pipeline begins with pharmacies, who routinely sell data derived from the vast volume of prescriptions they handle. While the prescribers’ names are usually redacted, IMS Health, a key health information organization in the pipeline, can easily use the American Medical Association (AMA)-licensed Physician Masterfile to reassociate physician ID numbers with the redacted names. The physician ID numbers are issued by the U.S. Drug Enforcement Administration (DEA) and are sold to AMA through a subscription service. IMS Health uses the prescription data to develop analytic tools for sale to pharmaceutical companies desperate to gain a marketing edge with individual prescribers. The tools consolidate the activity of nurse practitioners, dentists, chiropractors, and any professionals who can legally file a prescription. Marketers can use these tools to determine how much each named physician is prescribing, how that compares to other named physicians, what their specialty is, etc.

The data contained in the AMA’s Physician Masterfile is applicable for informing research and conducting surveys of practicing physicians, yet the need to identify physicians by name is usually not needed for public health research and enables prescriber manipulation.  The prescriber reports compiled by IMS Health enable pharmaceutical companies to take a data-driven approach to direct-to-physician advertising, a practice known as detailing. During a 17-month period between 2013 and 2015, pharmaceutical companies reported spending $3.5 billion in payments to physicians covering promotional speaking, consulting, meals, travel, and royalties. While many of the expenditures may be tied to legitimate collaborations between pharmaceutical companies and medical professionals, the U.S. Department of Health and Human Services warns that free samples, sham consulting agreements, subsidized trips, and industry-sponsored continuing education opportunities are all tools used by vendors to buy medically irrelevant loyalty. Indeed, physicians themselves seem conflicted over the significance of these relationships. When residents were asked if contact with pharmaceutical representatives influenced their prescribing practices, 61% believed they were unaffected. However, the same residents felt that only 16% of their peers were similarly immune to contact with pharmaceutical representatives.

Studies examining the role of detailing  have found it associated with higher prescribing frequency, higher costs, and lower prescribing quality, all with no contrasting favorable associations. Recent concerns over conflicts  of  interest arising from increased exposure of physicians to detailers led several academic medical centers to restrict sales visits and gift giving and implement enforcement mechanisms. Compared to hospitals with no detailing limitations, hospitals with limitations underwent an 8.7% relative decrease in the market share of detailed drugs and a 5.6% relative increase in the market share of non-detailed drugs. Overuse of brand-name drugs, which are most commonly associated with detailing, cost the US approximately $73 billion between 2010 and 2012, one-third of which was shouldered by patients. Advocates of the practice lament the lack of formal academic opportunities for physicians to learn about new drugs, believing the educational materials provided by pharmaceutical representatives fulfills a need.

The most tragic example of the potential harms of detailing targeting individual prescribers comes from the early days of the prescription opioid crisis. Purdue Pharma, the maker of OxyContin, used prescriber databases to identify the most frequent and least discriminate prescribers of opioids. Sales representatives, enticed by a bonus system that tracked their success according to upswings captured in the prescriber database, showered their target prescribers with gifts while systematically underrepresenting the risk of addiction and abuse from OxyContin. Recruitment into Purdue’s national speaker bureau and subsequent paid opportunities were further used to entice lukewarm and influential prescribers.

The last decade has seen several attempts to address the influence of detailing at the institutional, professional, and executive levels. Individual hospitals have begun limiting the access of physicians to vendors. The American Medical Student Association began issuing a conflict-of-interest scorecard, allowing all U.S. medical schools to track and assess their own detail-related policies, including those related to the limiting of gifts from the industry, industry-sponsored promotional speaking relationships, permitted accesses of pharmaceutical sales representatives, and overall enforcement and sanction of these policies. In 2016, 174 institutions participated. The AMA, which licenses the list of physician names used by health information organizations companies, has offered physicians the chance to block pharmaceutical representatives and their immediate supervisors from accessing their prescribing data. However, the Physician Data Restriction Program does not limit the ability of other employees at a pharmaceutical company to access prescribing data of doctors who have opted out. Physicians must renew their request to opt out every three years and are automatically added to the Masterfile upon entering medical school. Five years after the program’s introduction in 2006, just 4% of practicing physicians listed on the file had opted out.

In 2007, the state of Vermont outlawed the practice of selling prescription data for pharmaceutical marketing without prescriber consent. The law was quickly challenged by IMS Health, the Pharmaceutical Research and Manufacturers of America, and other data aggregators and eventually struck down by the U.S. Supreme Court. Vermont legislators held that detailing compromises clinical decision making and professionalism and increases health care costs and argued that the law was needed to protect vulnerable and unaware physicians. However, the Court held that speech in the aid of pharmaceutical marketing is protected under the First Amendment and could not be discriminately limited by Vermont law.

Congress made the first federal attempt to address the issue by enacting the Physician Payment Sunshine Act in 2010, which required companies participating in Medicare, Medicaid, and the State Children’s Health Insurance Program markets to track and collect their financial relationships with physicians and teaching hospitals. The transparency gained from the disclosures have allowed many researchers to systematically evaluate connections between conflicts of interests and prescribing behavior.

As policy makers and private watchdogs scramble to address the issues of detailing, the availability of physician names and prescription habits continues to facilitate the implementation of novel tactics. Limits on face time have pushed detailers to tap into the time physicians are spending online. When the names of prescribers are known, following and connecting with prescribers through social media accounts is straightforward. Companies like Peerin have emerged, which analyze prescriber Twitter conversations to learn whose conversations are most likely to be influential and which prescribers are connected. LinkedIn, Facebook, and Twitter all offer the ability to target a list of people by name or e-mail address for advertising. While all online drug ads are limited by the U.S. Food and Drug Administration, pharmaceutical companies are experimenting with the use of unbranded awareness campaigns to circumvent direct-to-consumer regulations.

While personalized prescriber marketing campaigns may be turning a new corner in the internet age, a simple opportunity exists at the federal level to de-personalize the practice of physician detailing. It is unclear the extent that the DEA stands to gain from selling physician ID subscriptions. However, in context of the downstream costs of the overuse of name-brand drugs this may be an appropriate loss. The U.S. Government’s central role in the reassociation of prescribers’ prescriptions could be directly addressed through systematic implementation of revised policy in order to preempt downstream prescriber manipulation.

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Written by sciencepolicyforall

November 9, 2017 at 10:41 pm

Science For All – Effective Science Communication and Public Engagement

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By: Agila Somasundaram, PhD

Image: By Scout [CC0], via Wikimedia Commons

         In 1859, Charles Darwin published the Origin of Species, laying the foundation for the theory of evolution through natural selection. Yet more than 150 years after that discovery and despite a large volume of scientific evidence supporting it, only 33% of the American population believes that humans evolved solely through natural processes. 25% of US adults believe that a supreme being guided evolution, and 34% reject evolution completely, saying that humans and all other forms of life have co-existed forever. Similarly, only 50% of American adults believe that global climate change is mostly due to human activity, with 20% saying that there is no evidence for global warming at all. A significant fraction of the public believes that there is large disagreement among scientists on evolution and climate change (the reality being there is overwhelming scientific evidence and consensus), and questions scientists’ motivations. Public skepticism about scientific evidence and scientists extends to other areas such as vaccination and genetically-modified foods.

Public mistrust in the scientific enterprise has tremendous consequences, not only for federal science funding and the advancement of science, but also for the implementation of effective policies to improve public and global health and combat issues such as global warming. In her keynote address at the 2015 annual meeting of the American Society for Cell Biology, Dr. Jane Lubchenko described the Science-Society ParadoxScientists need society, and society needs science. How then can we build public support for science, and improve public trust in scientists and scientific evidence?

Scientists need to be more actively involved in science outreach and public engagement efforts. Communicating science in its entirety, not just as sensational news, requires public understanding of science, and familiarity with the scientific process – its incremental nature, breakthrough discoveries (that don’t necessarily mean a cure), failures, and limitations alike. Who better to explain that to the public than scientists – skilled professionals who are at the center of the action? In a recent poll, more than 80% of Americans agree that scientists need to interact more with the public and policymakers. But two major hurdles need to be overcome.

Firstly, communicating science to the public is not easy. Current scientific training develops researchers to communicate science in written and oral formats largely to peers. As scientists become more specialized in their fields, technical terms and concepts (jargon) that they use frequently may be incomprehensible to non-experts (even to scientists outside their field). The scientific community would benefit tremendously from formal training in public engagement. Such training should be incorporated into early stages of professional development, including undergraduate and graduate schools. Both students and experienced scientists should be encouraged to make use of workshops and science communication opportunities offered by organizations such as AAAS, the Alan Alda Center for Communicating Science, and iBiology, to name a few. Secondly, federal funding agencies and philanthropic organizations should provide resources, and academic institutions should create avenues and incentives, for scientists to engage with the public. Both students and scientists should be allowed time away from their regular responsibilities to participate in public outreach efforts. Instead of penalizing scientists for popularizing science, scientists’ outreach efforts should be taken into consideration during promotion, grants and tenure decisions, and exceptional communicators rewarded. Trained scientist-communicators will be able to work better with their institutions’ public relations staff and science journalists to disseminate their research findings more accurately to a wider audience, and educate the public about the behind-the-scenes world of science that is rarely ever seen outside. Engaging with the public could also benefit researchers directly by increasing their scientific impact, and influence research directions to better impact society.

While increasing science outreach programs and STEM education may seem like obvious solutions, the science of science communication tells us that it is not so simple. The goals of science communication are diverse – they range from generating or sharing scientific excitement, increasing knowledge in a particular topic, understanding public’s concerns, to actually influencing people’s attitudes towards broader science policy issues. Diverse communication goals target a diverse audience, and require an assortment of communicators and communication strategies. Research has shown that simply increasing the public’s scientific knowledge does not help accomplish these various communication goals. This is because people don’t solely rely on scientific information to make decisions; they are influenced by their personal needs, experiences, values, and cultural identity, including their political, ideological or religious affiliations. People also tend to adopt shortcuts when trying to comprehend complex scientific information, and believe more in what aligns with their pre-existing notions or with the beliefs of their social groups, and what they hear repeatedly from influential figures, even if incorrect. Effective science communication requires identifying, understanding and overcoming these and other challenges.

The National Academies of Sciences, Engineering, and Medicine convened two meetings of scientists and science communicators, one in 2012 to gauge the state of the art of research on science communication, and another in 2013 to identify gaps in our understanding of science communication. The resulting research agenda outlines important questions requiring further research. For example, what are the best strategies to engage with the public, and how to adapt those methods for multiple groups, without directly challenging their beliefs or values? What are effective ways to communicate science to policymakers? How do we help citizens navigate through misinformation in rapidly changing internet and social media? How to assess the effectiveness of different science communication strategies? And lastly, how do we build the science communication research enterprise? Researchers studying communication in different disciplines, including the social sciences, need to come together and partner with science communicators to translate that research into practice. The third colloquium in this series will be held later this year.

Quoting Dr. Dan Kahan of Yale University, “A central aim of the science of science communication is to protect the value of what is arguably our society’s greatest asset…Modern science.” As evidence-based science communication approaches are being developed further, it is critical that scientists make scientific dialogue a priority, and make use of existing resources to effectively engage with the public – meet people where they are – and bring people a step closer to science – why each person should care – so that ‘post-truth’ doesn’t go from being merely the word of the year to a scary new way of life.

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Written by sciencepolicyforall

July 22, 2017 at 11:27 pm

The Economic Impact of Biosimilars on Healthcare

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By: Devika Kapuria, MD

          Biologic drugs, also defined as large molecules, are an ever-increasing source of healthcare costs in the US. In contrast to small, chemically manufactured molecules, classic active substances that make up 90 percent of the drugs on the market today, biologics are therapeutic proteins that undergo production through biotechnological processes, some of which may require over 1000 steps. The average daily cost of a biologic in the US is $45 when compared with a chemical drug that costs only $2. Though expensive, their advent has significantly changed disease management and improved outcomes for patients with chronic diseases such as inflammatory bowel disease, rheumatoid arthritis and various forms of cancer. Between 2015-2016, biologics accounted for 20% of the global health market, and they are predicted to increase to almost 30% by 2020. Worldwide revenue from biologic drugs quadrupled from US $47 billion in 2002 to over US $200 billion in 2013.

The United States’ Food and Drug Administration (FDA) has defined a biosimilar as a biologic product that is highly similar to the reference product, notwithstanding minor differences in clinically-inactive components, and for which there are no clinically meaningful differences between the biologic product and the innovator product in terms of safety, purity and efficacy. For example, CT-P13 (Inflectra) is a biosimilar to infliximab (chimeric monoclonal antibody against TNF-α) that has recently obtained approval from the FDA for use of treatment of inflammatory bowel disease. CT-P13 has similar but slightly different pharmacokinetics and efficacy compared to infliximab. With many biologics going off patent, the biosimilar industry has expanded greatly. In the last two years alone, the FDA approved 4 biosimilar medications: Zarxio (filgrastim-sndz), Inflectra (infliximab-dyyb), Erelzi (etanercept-szzs) and Amjevita (adalimumab-atto).

Unlike generic versions of chemical drugs (small molecules that are significantly cheaper than their branded counterparts), the price difference between a biosimilar and the original biologic is not huge. This is due to several reasons. First, the development time and cost for biosimilars is much more than for generic medications. It takes 8-10 years and several hundred million dollars for the development of a biosimilar compared to around 5 years and $1-$5 million for the generic version of a small molecule drug. With only single entrants per category in the US, biosimilars are priced 15-20% lower than their brand name rivals, which, though cheaper, still amount to hundreds of thousands of dollars. By the end of 2016, the estimated global sales from biosimilars amounted to US $2.6 billion, and nearly $4 billion by 2019. Estimates for the cost savings of biosimilars for the US market are variable; the Congressional Budget Office estimated that the BPCI (Biologics Price Competition and Innovation) Act of 2009 would reduce expenditures on biologics by $25 billion by 2018. Another analysis from the Rand Corporation estimated that biosimilars would result in a $44.2 billion reduction in biologic spending between 2014 and 2024.

In the United States, a regulatory approval pathway for biosimilars was not established till the Patient Protection and Affordable Care Act of 2010. However, biosimilars have been used in Europe for over a decade, and this has led to the development of strategies for quicker adaptation, including changes in manufacturing, scaling up production and adapting to local healthcare policies. These changes have led to a competitive performance of biosimilars in the European market, with first generation biosimilars taking up between 50-80% of the market across 5 European countries, with an expected cost savings of $15 to$44 billion by 2020. One example that demonstrates a significant discount involves the marketing of Remsima, a biosimilar of Remicade (infliximab). In Norway, an aggressive approach towards marketing of Remsima was adopted with a 69% discount in comparison to the reference product. After two years, Remsima has garnered 92.9% of the market share in the country.

The shift to biosimilars may be challenging for both physicians and patients. While safety concerns related to biosimilars have been alleviated with post marketing studies from Europe, there still remains a significant lack of awareness about biosimilars amongst healthcare providers, especially about prescribing and administering them. Patient acceptance remains an important aspect as well, with several patients loyal to the reference brand who may not have the same level of confidence in the biosimilar. Also, like with generics, patients may believe that biosimilars are, in some way, inferior to the reference product. Increased reporting of post marketing studies and pharmacovigilance can play a role in alleviating some of these concerns.

In 2015, the FDA approved the first biosimilar in the US, after which, it has published a series of guidelines for biosimilar approval, under the BPCA act, including demonstrating biosimilarity and interchangeability with the reference product. This includes a total of 3 final guideline documents and 5 draft guidance documents. Starting in September 2017, the World Health Organization will accept applications for prequalification into their Essential Medication list for biosimilar versions of rituximab and trastuzumab, for the treatment of cancer. This program ensures that medications purchased by international agencies like the UNICEF meet standards for quality, safety and efficacy. Hopefully, this will increase competition in the biosimilar market to reduce price and increase access to medications in low-income countries.

Both human and economic factors need to be considered in this rapidly growing field. Increasing awareness among prescribers and patients about the safety and efficacy of biosimilars as well as improving regulatory aspects are essential for the widespread adaptation of biosimilars.

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Written by sciencepolicyforall

July 19, 2017 at 10:42 am