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Archive for June 2015

Science Policy Around the Web – June 30, 2015

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By: Eric Cheng, Ph.D.

Photo credit link: Affordable Care Act via photopin (license)

Health policy

Affordable Care Act survives Supreme Court challenge

On June 25, 2015, the Supreme Court upheld a key provision of the Affordable Care Act that allows the federal government to provide subsidies to people in all 50 states, and not just the 16 states that administer their own online insurance exchanges. This ruling means that people in states without their own exchanges can still get federal subsidies for the purchase of health insurance allowing an estimated 6.4 million people to keep their health care coverage, according to the Kaiser Family Foundation. Without access to subsidies, millions of Americans would have been at risk of failing to meet the mandate that requires every American to buy health coverage. This ruling affirmed that the legislation that created the Affordable Care Act intended to improve the health care system and to provide support to all who needed help to buy health insurance.

Two of the court’s conservatives, Chief Justice John G. Roberts Jr. and Justice Anthony Kennedy, joined the court’s four liberal Justices in rejecting the lawsuit in a 6-3 vote to side with the Obama administration. “Congress passed the Affordable Care Act to improve health insurance markets, not to destroy them,” Roberts wrote for the majority. While Justice Antonin Scalia, who wrote in dissent, called the majority’s reasoning “quite absurd” and “interpretive jiggery-pokery.”

President Obama made a statement on the recent ruling, saying the Affordable Care Act “is here to stay.” (Robert Barnes, Washington Post)

Federal research funding

Senate panel approves $2 billion raise for NIH in 2016

A Senate appropriations subcommittee voted to approve $32 billion in funding for the National Institutes of Health (NIH) for the 2016 fiscal year, representing an increase of $2 billion from 2015. This increase in funding could be the largest increase since 2003. Senator Roy Bunt (R-Mo.), chairman of the Senate Labor, Health and Human Services, and Education and Related Agencies Appropriations Subcommittee commented that “this year’s Labor-HHS appropriations bill prioritizes programs that will provide a significant benefit to all Americans and, most importantly, provides the National Institutes of Health with a $2 billion increase to make critical life-saving medical treatments and high-quality cures available to all Americans.”

The Senate panel approval will match the $200 million request by President Obama for the development of the Precision Medicine Initiative along with a $100 million increase for new funding to combat antibiotic resistance from the previous fiscal year. Other highlights include a $350 million increase for the National Institute on Aging, the lead Institute researching Alzheimer’s disease, and an additional $70 million for the BRAIN Initiative to map the human brain. These increases to every Institute and Center are intended help NIH to continue their development of new therapies, diagnostics, and preventative measures to enhance health, lengthen life, and reduce illness and disability. (Jocelyn Kaiser, ScienceInsider)

Environment and public health

House Advances Chemical Safety Reform Bill, Teeing Up Senate Vote

The U.S. House of Representatives passed legislation to overhaul the nearly 40 year old Toxic Substances Control Act (TSCA). This bipartisan bill aims to improve chemical safety. Some updates include the determination of risk by the Environmental Protection Agency (EPA) based on scientific evidence of its health impact instead of the cost of regulation. For new chemicals, the bill would shift the burden to industry to show that the substances they manufacture are not unreasonably risky. Other provisions in the bill would increase transparency by preventing industry efforts to keep safety data secret by declaring them confidential business information. This would allow EPA to more easily share chemical information with states, doctors, and first responders. These new reforms will help address an outdated law to protect human health and the environment from significant risks while also protecting commercial and competitive interests of the United States chemical industry and the national economy. (Kate Sheppard, Huffington Post; Puneet Kollipara, ScienceInsider)

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June 30, 2015 at 9:00 am

Science Policy Around the Web – June 26, 2015

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By: Amy Kullas, Ph.D.

Public Health Policy: Organ donation

State policies have had little success on increasing organ donation

There is an extreme shortage of transplantable solid organs in the United States, which poses a considerable public health challenge. Presently, an estimated 125,000 patients require an organ transplant and of these patients, almost 80,000 remain on active waitlists for these organs nationally. Unfortunately more than 6,000 of these patients are expected to die this year while waiting for a transplant.

Since 2010, all states and the District of Columbia have implemented at least two donation-related policies. These policies varied from dedicated funds for donor recruitment activities to paid leave after donation to tax incentives. Unfortunately, many of these polices (with the exception of revenue policies) have had “no robust, significant association with either donation rates or number of transplants.” The revenue policies have people donate to a “protected state fund” which is used to fund activities to promote organ donation. These policies correlated with a modest “5.3% in the absolute number of transplants.” This brings up an interesting question and proposition: should individuals be financially compensated for donating an organ?(Chatterjee, et al, JAMA Internal Medicine)

Humans, Climate Change and Animal Extinction

The sixth mass extinction is among us. Is there anything left to do?

[The study] shows without any significant doubt that we are now entering the sixth great mass extinction event,” said Paul Ehrlich, the Bing Professor of Population Studies in biology and a senior fellow at the Stanford Woods Institute for the Environment. Ehrlich and his co-authors warn that this “extinction threatens humanity’s existence” and that soon we will pass the point-of-no-return. Ehrlich further says, “There are examples of species all over the world that are essentially the walking dead.” The overall consensus among scientists that the current extinctions rates have reached levels that have not been encountered since the dinosaurs’ downfall, approximately 65 million years ago.

As the human population continues to increase at an almost exponential rate, the laundry list of how humans are brutalizing Mother Earth is also increasing. Some of which include:

  1. Clearing the land for farming, then clearing the farmland for housing, etc.
  2. Introduction of invasive species
  3. Record high carbon emissions driving global warming and ocean acidification
  4. Toxins that leach into water supplies and can poison fragile bionetworks

But is there anything left for humanity to do? It is a complex ‘yes.’ The authors write, “Avoiding a true sixth mass extinction will require rapid, greatly intensified efforts to conserve already threatened species, and to alleviate pressures on their populations — notably habitat loss, over-exploitation for economic gain and climate change.” (Rob Jordan, Stanford Report)

Animal Rights and Biomedical Research

Captive chimps to be categorized as an endangered species

On June 12, the US Fish and Wildlife Service (USFWS) announced that it is classifying captive chimpanzees (approximately 1750) as endangered under the Endangered Species Act. The new designation will give 700+ chimps in U.S. research laboratories, in addition to those in zoos and in ‘entertainment’, the same protection as their counterparts in the wild. Bottom line, biomedical research involving chimpanzees is going to dramatically change when the classification is implemented on September 14. USFWS will start requiring a permit for doing scientific research on captive chimps, along with their sale and import.

Biomedical research involving chimpanzees had already significantly decreased since 2013 when the National Institutes of Health (NIH) pledged it would retire the majority of its captive chimps, leaving only 50 animals available for potential projects. Yet, only 66 chimps have officially been retired since June 2013. These retired chimps are transitioning to sanctuaries.

John VandeBerg, the former director of the Southwest National Primate Research Center in San Antonio, highlights how this decision will affect biomedical research, such as the development of vital vaccines. VandeBurg gives the grim forecast “There will be a lot of people who die who would not have died.” (Sara Reardon, Nature and David Grimm, Science)

New Science Policy Blog!

National Institutes of Health Office of Science Policy launches new blog

The National Institutes of Health (NIH) Office of Science Policy (OSP) has launched a new blog “Under the Poliscope: Bringing Science Policy Into Focus.” The blog will showcase some of the activities of the OSP as well as focusing on science policy matters in a general context and emerging issues of interest to the life science and biomedical communities.

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June 26, 2015 at 9:00 am

Science Policy Around the Web – June 23, 2015

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By: Rebecca A. Meseroll, Ph.D.

Water conservation

Satellite data reveals depletion of underground water supply

A decade-long study using NASA satellite data reveals that 21 of the world’s 37 largest aquifers have passed their sustainability tipping points, i.e., more water was removed from the aquifers than replenished over the course of the ten years that study was conducted. These underground water supplies account for 35 percent of human water usage around the globe, with increased use during times of drought, when above-ground water sources are diminished. Stress on the aquifers varies around the world, but was found to be highest in the areas of dense human populations, especially in dry environments. The most drastically depleted aquifer is the Arabian Aquifer, which services more than 60 million people, followed by the Indus Basin in India and Pakistan. Aquifer depletion rates are also high in locations where water-demanding activities, including various types of mining and drilling for oil and natural gas, take place, such as the Canning Basin in western Australia. Although water basins do become replenished with rainwater and snow melt, this is a process that can take thousands of years. According to Jay Famiglietti, the principal investigator of the study, the stress on the aquifers is anticipated to get worse as with global warming, as areas around the equator receive less rain and people living in those areas need to get more water from underground sources. Population growth is also expected to put strain on the aquifers. Water conservation efforts will be required to prevent further depletion of aquifers worldwide. (Todd Frankel, Washington Post)

Supplement regulation

Senator calls for scrutiny of Alzheimer’s supplements

Senator Claire McCaskill (D-Missouri), a ranking member of the Senate Special Committee on Aging, sent letters last week to fifteen major retailers asking what efforts they were making to prevent the sale of any fraudulent or potentially dangerous supplements that claim to protect against Alzheimer’s disease and dementia. The letters requested documentation of retailers’ policies relating to sale, marketing, removal, and reporting of adverse effects of dietary supplements. Federal law, as laid out by the Dietary Supplement Health and Education Act of 1994, allows supplement manufacturers to make general claims about their products’ health benefits, but prevents them from claiming a supplement can treat a specific disease. Despite this legislation, supplements claiming to protect against diseases still make it into the marketplace. In her letter to Amazon, McCaskill specifically targets a supplement called Brain Armor, touted by its manufacturer to protect against Alzheimer’s disease, dementia, and several other hallmarks of cognitive decline.   Although this particular supplement was removed from Amazon following notification by the FDA, which had been made aware of the supplement’s illegal claims during a staff meeting with McCaskill, the letter raises the question of why it had been for sale in the first place. Supplement regulation has been in the news previously this year when the New York State attorney general, Eric T. Schneiderman, sent cease and desist letters to GNC, Target, Walmart, and Walgreens, upon discovering that these retailers were selling herbal supplements that contained unlisted contaminants or did not even contain what was on the label. New legislation may be necessary to protect consumer safety if current regulations are not sufficient. (Anahad O’Connor, The New York Times)

Ebola drug development

Trials for potential Ebola drug halted

Tekmira Pharmaceuticals and the Wellcome Trust announced late last week that they ceased enrollment in a trial for the potential Ebola drug, TKM-Ebola-Guinea, because the study had reached a statistical endpoint, meaning the trial would not likely be improved by increased enrollment in the study. Although the drug was found to be an effective anti-Ebola therapeutic in rhesus monkeys, it has not proven beneficial for human patients. TKM-Ebola-Guinea is composed of several small RNA molecules, which interfere with Ebola proteins to counteract replication of the virus, inside a lipid nanoparticle. The drug tested on humans uses an older type of lipid nanoparticle compared to the one used on the monkeys, because the newer lipid nanoparticle has not undergone safety trials yet, and this difference may have an influence on the efficacy of the drug. Trial design has also presented some difficulties in analysis of the data, due to the ethical considerations of treating infected patients with a placebo instead of the drug. Everyone enrolled in the study received the drug, and outcomes were compared with those of patients at treatment centers not involved in the trial, rather than a control group receiving a placebo or a different drug. Data that have been collected from the trial thus far will still be analyzed to learn more about the tolerability of the drug and its effect on the patients that were treated with it. Clinical trials for other potential Ebola treatments and preventions, such as the antibody cocktail ZMapp and two candidate vaccines, are ongoing. (Gretchen Vogel and Kai Kupferschmidt, ScienceInsider)

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June 23, 2015 at 9:00 am

Science Policy Around the Web – June 19, 2015

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By: Sylvina Raver, Ph.D.

Drug Policy

Medical marijuana laws don’t lead to more teen toking

A recent study published in the journal The Lancet Psychiatry evaluated the association between states’ legalization of medical marijuana and marijuana use by teenagers. Marijuana is the most widely used illicit drug in the US, with approximately 19.8 million Americans reporting use within the past month. Although cannabis remains illegal under Federal law, 23 states and the District of Columbia have legalized marijuana use for medical purposes under the supervision of a medical professional. A primary concern for opponents of marijuana legalization – for either medical or recreational use – is that these laws will increase marijuana use in adolescents, among whom the perception of the risks associated with marijuana has steeply declined since the mid 2000’s. Marijuana use in adolescence coincides with a period of substantial brain maturation and can lead to negative consequences in adulthood, such as persistent cognitive impairments, an increased risk for severe psychiatric disorders like schizophrenia, and abnormal neurological activity. These serious risks underscore concerns that any effort that makes marijuana seem less dangerous to teenagers, such as medical legalization, would lead to an increased prevalence of use within this population. Indeed, Hasin and colleagues, the authors of The Lancet study, initially predicted that marijuana use among teenagers would increase in states that enacted medical marijuana laws. However, this was not the case. The authors used data from the Monitoring the Future survey, which included information from more than 1 million US teenagers between the ages of 13 and 18 over the past 24 years in 48 US states. After controlling for potentially mitigating factors, the scientists found that marijuana use among teenagers did not significantly change before and after states passed medical marijuana laws. In fact, a significant and robust post-law decrease in marijuana use was found among 8th graders, the youngest age surveyed. Adolescent marijuana use was more prevalent in states that subsequently enacted medical marijuana laws, but, contrary to the authors’ predictions, those laws did not increase marijuana use in teens. These findings argue against a widely held notion that passage of state laws that legalize medical marijuana use conveys a public message to teenagers that marijuana is relatively harmless, and therefore prompts more teenagers to use the drug, even if the new law explicitly prohibits underage use. Hasin and colleagues advocate for a shift away from the concern that medical marijuana laws will increase teenage marijuana use and favor allocating more resources to identify those factors that do influence marijuana use in this vulnerable population. (Mike Stobbe, The Associated Press)

Health Policy

Trans fats to be illegal

On Tuesday, June 16th, the FDA issued a final determination stating that partially hydrogenated oils (PTOs), the primary dietary sources for trans fats, are no longer “generally recognized as safe” for use in food products. The decision requires food manufacturers to completely remove synthetic trans fats from processed foods by June 2018. The FDA has required that trans fat content be clearly labeled on food packaging since 2006, and issued a proposal for an eventual ban on adding artificial trans fats to food in 2013. However, the food industry still includes partially hydrogenated oils in products ranging from microwave popcorn to cake frosting, and the Grocery Manufacturers Association – the food industry’s largest trade association – has been continually petitioning the government to allow “very limited” use of PTOs in the future. Food manufacturers turned to PTO’s starting in the 1950’s as an alternative to saturated fats that could improve food texture while simultaneously increasing shelf stability. PTOs have held the “generally recognized as safe” distinction since then, which means that the oils do not require FDA’s approval. The recent FDA decision is in line with the organization’s “…goal…to minimize trans fat intake as much as possible,” according to Susan Mayne, Director of the FDA’s Center for Food Safety and Applied Nutrition, and the organization estimates that a total ban on these substances could prevent up to 20,000 heart attacks and 7,000 premature deaths each year. (James Hamblin, The Atlantic; Helena Bottemiller Evich, Politico)

Climate Change

Pope Francis: Climate Change A ‘Principal Challenge’ For Humanity

Pope Francis has issued a much-anticipated 184 page encyclical letter titled “Laudato Si,” or “Praise Be: On Care for Our Common Home,” in which he argues that the threat of global climate change is a moral and ethical concern, rather than being exclusively a matter of politics, science, and economics. In the Catholic Church, encyclicals are considered to be formal teachings on Catholic doctrine, and have historically been intended for audiences of bishops and priests. However, as the pope recently stated, “This encyclical is aimed at everyone,” and in it he offers “…the world a moral vocabulary for talking about climate change, shifting global attention from the macro solutions of policy summits to the personal ethics of environmental stewardship.” Pope Francis calls on wealthy nations, the largest global producers of greenhouse gases, to shoulder the burden of limiting their consumption of non-renewable forms of energy and to assist poorer countries to develop policies and programs that support sustainable development. In his letter, the pope agrees with the overwhelming consensus of climate scientists that humans’ actions are largely responsible for the Earth’s changing climate, a stance that places him at odds with many conservative lawmakers and Republican presidential hopefuls who deny the effects of human actions on climate change. His language is clear: “ …yet a number of scientific studies indicate that most global warming in recent decades is due to the great concentration of greenhouse gases…released mainly as a result of human activity.” And while he acknowledges that efforts have been made to enact policies intended to combat climate change, Pope Francis is relatively critical of their efficacy and of humanity’s possible reliance upon new technological solutions to curb the effects of global warming. The timing of the letter may have strategic importance: The next United Nations Climate Change Conference is scheduled for November 2015, and the organizers have set the lofty objective of achieving, for the first time in over 20 years of UN negotiations, binding and universal agreements for reducing carbon emissions. (Scott Neuman, NPR; Laurie Goodstein & Justin Gillis, The New York Times; Emma Green, The Atlantic)

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June 19, 2015 at 9:00 am

Science Policy Around the Web – June 16, 2015

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By: Danielle Friend, Ph.D.

Health Policy

A Bill to Fight Obesity

A bill titled “Treat and Reduce Obesity Act of 2015” was recently sponsored by Erik Paulsen (R-MN) and would amend title XVIII (Medicare) of the Social Security Act in an attempt to treat and prevent obesity in America. The bills states that “According to the Centers for Disease Control, about 34 percent of adults aged 65 and over were obese in the period of 2009 through 2012, representing almost 15 million people”. The bill also brings up the important point that obesity also “increases the risk for chronic diseases and conditions, including high blood pressure, heart disease, certain cancers, arthritis, mental illness, lipid disorders, sleep apnea, and type 2 diabetes”. Importantly, “the direct and indirect cost of obesity is more than $450 billion annually” and that “a Medicare beneficiary with obesity costs $1,964 more than a normal-weight beneficiary”. The bill would attempt to reduce obesity rates and lower the financial costs to society by allowing the Social Security Act to cover intensive behavioral therapy for obesity. In addition, the amendment will also allow Social Security to cover pharmaceuticals used to treat obesity or for weight loss management.

Data and Biomedical Research

National Library of Medicine urged to take on broader role

Originally established in 1836 as a small collection of medical books and journal housed in the Office of the Surgeon General of the army, the National Library of Medicine (NLM) has now grown into the world’s largest biomedical library. Consisting of an extensive collection of both paper and digital sources, the NLM also runs GenBank, MedlinePlus, and This year, Francis Collins, the director of the National Institutes of Health, organized a working group to determine the new vision of the NLM. Although the working group determined that the current programs run by the NLM should stay in place, the working group also decided that the NLM should expand its role as a leader in sharing biomedical data. The working group suggests that the NLM should coordinate data science programs and run the Big Data to Knowledge (BD2K) initiative at the NIH. Other main points of the report include continuing to serving as a leader through gathering and sharing biomedical research, supporting data sharing, research reproducibility, and transparent analysis. The NLM is also encouraged to support education in biomedical informatics, data science and library science. The complete report on the future vision of NLM can be found here. (Jocelyn Kaiser, ScienceInsider)

Therapies of the Future

Researchers discover genes associated with resistance to spongiform encephalopathies

Authors of a recent paper published in Nature have discovered a human genetic variant that lends resistance to certain individuals against some types of spongiform encephalopathies. Spongiform encephalopathies are currently most visible in the public mind as “mad cow”disease and the human variant Creutzfeldt–Jakob disease (vCJD). These currently incurable encephalopathies are caused when abnormal prion proteins (PrP) spread throughout the brain, damaging tissue and ultimately leading to death. Researchers of the recent paper entitled “A naturally occurring variant of the human prion protein completely prevents prion disease” found that a variant of PrP found in certain individuals gave mice resistance to developing one kind of spongiform encephalopathy, Kuru, when exposed to diseased prions. Researchers have hopes that understanding how this genetic variant prevents the propagation of PrP may lead to future medical treatments possibly through the inhibition PrP during spongiform encephalopathies. (Glen Telling, Nature News and Views)

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June 16, 2015 at 9:00 am

Science Policy Around the Web – June 12, 2015

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By: Varun Sethi, MD, PhD

Technology and Medical Ethics

AMA Tackles Ethics of Telemedicine

A report by the Council on Ethical and Judicial Affairs (CEJA) was recently supported at a reference committee hearing but failed to get approval from the American Medical Association (AMA) House of Delegates. This report recommended that physicians providing clinical services via telemedicine must uphold the standards of professionalism as expected in in-person examinations, and be cognizant of the limitations of relevant technologies.

Some of the concerns were about parts of the recommendations dealing with informed consent. A delegate of the American College of Radiology (ACR), Todd M. Hertzberg MD, explained that there were scenarios in context of teleradiology and telepathology where in the process of informed consent was not in place and/or was less specific. A delegate from Texas, Arlo F. Weltge, MD, PhD, MPH, agreed that telemedicine is emerging as being clearly important, but expressed concern about a current case where in the Texas Medical Board was being sued for an interpretation. Weltge explained that the interpretation of the AMA code on telemedicine ethics could imply that, “anybody can set up a remote station and prescribe medications and, if you will, become an internet pill-mill.”

Other delegates supported the code and emphasized that Telemedicine is the future, and the future is here! Nonetheless, there is a strong need for ethical guidelines and these are needed as a priority. (Sarah Wickline Wallen, MedPage Today)

Biomedical Research Funding

Study claims $28 billion a year spent on irreproducible biomedical research

Economists report that the exorbitant amount of $28 billion is spent each year on irreproducible preclinical research in the United States. By reviewing literature for over two dozen studies, economists estimated that about 53% of preclinical studies have errors and are thus not reproducible. The source of these ‘errors’ varied from problems with reagents and reference materials (36%), problems with study design (28%), errors in data analysis and reporting (25%), and laboratory protocols (11%). With an estimated 56 billion dollars spent by NIH and US public and private funders, ~ 50% of this total (i.e. 28 billion dollars) was used for ‘irreproducible research’.

While the NIH has issued new criteria to strengthen the reproducibility of funded research, the authors of this report suggest that irreproducible research is not necessarily a ‘waste’. They emphasize that investment should be increased, with a relatively small part dedicated for improving the reproducibility rate of research. Other strategies include improving training researchers in the realms of study design, and stressing the use of validated reagents only etc. Microbiologist Ferric Fang is concerned about the possibility to extrapolate findings from a few studies. Calling this report ridiculous and unhelpful, Fang stressed that an irreproducible result does not imply that the original result was incorrect. (, ScienceInsider)

Research and Collaboration Policy

Funders must encourage scientists to share

Following the precedent established by the Human Genome Project, researchers and scientists agree that it is important to share large data sets (e.g. genomics, epidemiology, population level health), in order to realize the full potential and maximize benefits of that data. A recent survey reported that both the providers as well as those who use the shared data, are frustrated with the data-access process. Access protocols are very specific and tailored to different studies, augmenting the administrative burden.

An expert advisory group has published recommendations to aid researchers. They suggest that data access plans must be incorporated into the grant application process. Funders should be encouraged to standardize the process, allowing for flexibility of individual study characteristics; access procedures should be transparent, straightforward and allow for an independent appeal process to settle disputes. Participants in studies can also be protected better if data access provisions are planned at the onset, e.g. permission to share de-identified data could be incorporated in the consent form. To encourage scientists to contribute to data sharing, rewards could also be used as a motivation, the group suggested.

To protect the providers of data, it is also important to allow for justifiable restrictions for hard earned data sets. The group stressed the need for a clear explanation of the conditions imposed. Significant breaches of data/ material transfer agreements should also be treated seriously so as to act as a deterrent to such practice. In an era of international and collaborative science, scientists need to be encouraged to volunteer to share data and must also be made to feel protected. (Martin Bobrow, Nature Column: World View)

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June 12, 2015 at 9:00 am

Regulating laboratory tests for accuracy and patient safety

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By: Cheryl Jacobs Smith, PhD

On June 13, 2013, the Supreme Court ruled that Myriad Genetics did not own the sole rights to the genetic testing of genes involved in breast cancer susceptibility, BRCA1 and/or BRCA2 (1). Therefore, diagnostic companies and academic institutions could then create their own diagnostic tests surrounding BRCA1 and BRCA2. However, since the ruling, as more and more companies and academic centers are taking advantage of the “open market” on diagnostic tests, a question arises: Are all of these diagnostic tests appropriate for the clinic and safe for patient use?

The Food and Drug Administration (FDA) would certainly say no. In a document issued on October 3, 2014, the FDA outlined a draft guidance addressing gaps and areas of improvement related to the regulatory oversight of diagnostic tests (2). In the draft guidance, the FDA emphasized that the subset of in vitro diagnostic devices (IVDs) referred to as laboratory developed tests (LDTs), should have additional regulatory oversight to assure their safety and efficacy. Some recommendations include: adverse event reporting, an option to remove unsafe LDTs, critical evaluation of clinical validity and reproducibility of LDTs, and structured guidelines for informed consent.

Since 1976, the Medical Device Amendments (MDA), which amended the Federal Food, Drug, and Cosmetic Act (the FD&C Act), established a rubric for the regulation of medical devices intended for human use (3)(4). It was amended to make explicit that the act include IVDs, and thus, LDTs. The definition of an IVD applies equally whether it is manufactured by conventional manufacturers or by individual laboratories. However, since the implementation of the MDA of 1976, the FDA has enforced applicable provisions with respect to LDTs under the FD&C Act at the manufacturer’s discretion. Thus, enforcement discretion for LDTs developed as a matter of general practice rather than by law.

The Centers for Medicare and Medicaid Services (CMS) has regulated LDTs (with research LDTs exempt) since 1988 under the Clinical Laboratory Improvement Amendments (CLIA—42 U.S.C. 263a) (5). Although all clinical laboratories must be properly certified to receive Medicare or Medicaid payments, CLIA has no direct responsibilities to either Medicare or Medicaid. CLIA governs the accreditation, inspection, and certification process for laboratories. However, the statutes of CLIA do not evaluate test validity prior to marketing, nor do they assess the clinical validity of a LDT. This regulatory gray area poses a couple of questions: Can the LDTs actually identify the biological events that the manufacturers claim? And, if so, with what accuracy in the tested population? These are very important questions when the result of a particular test could determine medical treatment. Therefore, although CLIA oversight is important, it alone does not ensure that LDTs are properly designed, appropriately manufactured, and are safe and effective for patient use. With the large regulatory gaps that still exist in the LDT market, how is it that this has gone on for so long without being addressed?

Initially, when LDTs were used to measure blood type or one or two biological markers at a time, CLIA accreditation was sufficient. However, due to changes in the complexity and use of LDTs, and with the advent of complex algorithms accompanied by computational analysis, CLIA requirements alone have become inadequate. Learning this information may make consumers uneasy to have any sort of test done by a medical office in the near future. Yet, is the current state of affairs of LDTs this dire?

I have been fortunate to witness the process by which an LDT is developed. From what I observed, the process appeared highly regimented, rigorous, and repeated to ensure accuracy and specificity of both the test and the instrument running the test. The diagnostic laboratory was not only CLIA-certified but was also certified by the College of American Pathologists (CAP) Laboratory Accreditation Program that “[…] test disciplines with the most scientifically rigorous customized checklist requirements.” (6) Under CAP-certification, laboratories must maintain the accuracy of test results and ensure accurate patient diagnosis, as well as have mechanisms in place to correct cited deficiencies. In that setting, the manufactured LDTs met the CLIA and CAP-certification requirements and included data on LDT specificity, accuracy, and clinical validity to discern between various biological conditions. Consequently, for this laboratory and others with CLIA and CAP-certification, additional oversight by the FDA could prove to be redundant and burdensome slowing down LDT production and availability, in turn potentially negatively impacting patient care. However, not every laboratory that manufactures LDTs conforms to this high level of scientific and medical integrity. Therefore, how can LDTs be regulated so that consumers can be confident in the methodology and reliability of LDTs without stifling LDT production and innovation?

The FDA proposes to amend the regulations surrounding LDTs by implementing a risk-based approach toward oversight of LDTs. Similar to how other medical devices are categorized into risk classes (I-III with I being the lowest risk and III the highest risk), LDTs would be classified based on risk to the patient and/or user and receive appropriate regulations commensurate to the risk level. With the exception of some LDTs solely used for forensic purposes and those used in CLIA-certified, high-complexity histocompatibility laboratories, those laboratories with high, moderate, and low risk LDTs would be required to enforce applicable regulatory requirements that include registration and listing, adverse event reporting, pre-market review, and quality system requirements. If the FDA guidelines become regulatory law for LDTs, patients would gain a comprehensive database of reliable LDTs increasing patient flexibility with whom they choose to do the LDTs and perhaps reduce costs if saturated markets are filled with reliable LDTs rather than a mixture of quality. Additionally, LDT manufacturers would be able to scrutinize tests more comprehensively instead of relying on word-of-mouth or anecdotal evidence to improve unreliable tests.

The suggested increased regulation of LDTs by the FDA may seem burdensome but would achieve a more comprehensive manner to regulate and equalize the validity of LDTs on the market. If anything, it would be helpful to have LDT pre- and post-market evaluations standardized as these measures uncover important medical device mishaps that frequently save patients from unnecessary harm or distress (7). The Diagnostic Test Working Group (DTWG), an independent group consisting of representatives from diagnostic manufacturers and clinical laboratories, suggested adopting some, but not all, of the FDA’s recommendations. Most importantly, the group stressed that this new legislation should require LDT manufacturers to register the LDT with the FDA but that additional oversight concerning sensitivity, specificity, and validity should be applied to LDTs only in the high risk class. Furthermore, DTWG suggested that the FDA should create an additional center for LDTs (8). The later should be seriously considered as FDA is excellent in drafting regulatory frameworks that ensure patient safety, but frankly lack the money and thus, people power to execute its regulations effectively. New LDT regulation may be needed, but without increasing the FDA’s budget to allow swift and effective execution of the FDA’s recommendations, this LDT legislation could ultimately just be black ink on white paper.

Written by sciencepolicyforall

June 10, 2015 at 9:00 am