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Posts Tagged ‘FDA

Science Policy Around the Web – July 21, 2017

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By: Rachel F Smallwood, PhD

Source: pixabay

Cancer

Engineered Cell Therapy for Cancer Gets Thumbs Up from FDA Advisers

A panel of advisers has recommended that the FDA approve chimeric antigen receptor T-cell (CAR-T) therapy for treatment of acute B-cell lymphoblastomic leukemia. The committee unanimously agreed that the risk to benefit ratio was favorable enough to proceed with approval of the drug (tisagenlecleucel), manufactured by Novartis. CAR-T therapy utilizes a patient’s own immune cells to find and attack cancer cells. In a recent trial in humans, 82.5% of patients went into remission following treatment with the drug; there have also been promising results from its use in glioblastoma treatment. The treatment would specifically be for pediatric and young adult patients who did not respond well to initial treatments or who relapsed from being in remission.

Despite have strong positive effects, there are potential risks posed by CAR-T therapy. In the study mentioned above, almost half of the patients experienced an inflammatory reaction called cytokine release syndrome. Although all of those cases were treatable, the condition can be life-threatening. Novartis also reported neurological problems. Other CAR-T trials have had several deaths due to brain swelling, but those were in adult populations and were some differences in the therapies.

The FDA often does take the recommendations of its advisers, but there is much to consider in this decision. It would essentially be approving a living drug that is individualized to each patient; the patients’ own blood cells are sent to a manufacturing center, where they are genetically engineered to target leukemia cells. The cell population is then allowed to proliferate, and the entire process takes around twenty-two days. This process presents a quality assurance and control problem to the FDA. However, the target population typically has a poor prognosis and very few options, so the panel considers the potential for increased survival and quality of life to be worth the risks. (Heidi Ledford, Nature News)

Stem-Cell Therapy

Unapproved Stem-Cell Treatments Touted on Federal Database Clinicaltrials.Gov

ClinicalTrials.gov is an online database, curated by the National Library of Medicine and the National Institutes of Health, that logs clinical studies occurring around the country and allows them to be searched by patients, family members, healthcare providers, and researchers. The information on the site is provided by the researchers or sponsors of the individual studies themselves. It allows patients and healthy people to become aware of opportunities to participate in medical research. These studies involve a wide range of treatments, including drugs, devices, behavioral therapies, and procedures.

A recent study found that the database is being abused by clinics advertising for stem cell trials. These trials target individuals looking for treatment for a variety of conditions, and all of them charge for participation. There are very few FDA-approved stem cell therapies, and most clinics that utilize stem cell therapies assert that they do not need FDA approval since they are practicing medicine and do not substantially alter the stem cells (although that is disputed).  Since the researchers themselves indicate in the database whether they need FDA approval, there is little oversight to ensure these studies are correctly representing the risks and benefits of their treatment.

Although a disclaimer was added this spring that informs visitors that the presence of a trial in the database does not indicate government endorsement of it, many people do not realize that they could potentially be participating in a for-profit procedure that does not have the proper oversight to ensure patient safety. In one such case, three women were blinded who paid to receive stem cell therapy for macular degeneration. Most legitimate research studies will not require payment for participation, although travel and lodging costs associated with participation may be incurred.

While many patients may receive treatment at one of these clinics without an adverse event or even with a positive result, critics of these types of clinics are calling for regulation of entries into the ClinicalTrials.gov system. They assert that a federal resource for medical research should not be used to advertise for for-profit clinics that are utilizing therapies that have not been studied or reviewed for safety and efficacy. (Laurie McGinley, Washington Post)

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July 21, 2017 at 10:08 am

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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July 19, 2017 at 10:42 am

Science Policy Around the Web – July 7, 2017

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By: Leopold Kong, PhD

Food Policy

Food and Microbiota in the FDA Regulatory Framework

More and more probiotic food products, or microbiota-directed foods, claiming to “improve” the body’s microbiota have been hitting the shelves, with sales valuing over US$700 million in the US alone and US$36.6 billion globally this past year. However, there is little framework regulating their ingredients or guaranteeing the scientific accuracy of their health claims that has resulted in costly legal action. For example, in September 2009, Dannon settled a US$35 million consumer class action suit challenging the claimed health benefits in their ads. A similar class action suit against Procter & Gamble’s Align probiotic has been certified and set for Oct. 16, 2017. A paper recently published in the journal Science calls for greater clarity in policy regulating probiotic products. Importantly, the authors urge that probiotics should be clearly classified as a dietary supplement, a medical food, or a drug. If classified as a dietary supplement, probiotics can make claims on nutrient content and effect on health, but not on treatment, prevention or diagnosis of disease. If classified as a medical food, probiotics must contain ingredients that aid in the management of a disease or condition, with “distinctive nutritional requirements”, that is scientifically recognized. Finally, if classified as a drug, probiotics will require clinical trials to prove its medical claims. An alternative, and perhaps cheaper, way forward is to regulate probiotics as a kind of over-the counter medical food, requiring testing only for their active ingredients that can be used in a variety of products. (Green et al., Science)

Antibiotic Resistance

Untreatable Gonorrhoea on the Rise Worldwide

Over 78 million people are infected with gonorrhea each year, a sexually transmitted disease that has traditionally been treated effectively with anti-microbials. However, recently published data from 77 countries show that antibiotic-resistant gonorrhea is getting more pervasive and harder to cure. “The bacteria that cause gonorrhea are particularly smart. Every time we use a new class of antibiotics to treat the infection, the bacteria evolve to resist them,” said Dr. Teodora Wi, Medical Officer, Human Reproduction, at the WHO. The data found widespread resistance to ciprofaxacin, azithromycin, and even to the last-resort treatments, oral cefixime and injectable ceftriaxone. New drugs are under development, including a phase III trial of a new antibiotic, zoliflodacin, launched by the non-governmental organization Drugs for Neglected Diseases Initiative and Entasis Therapeutics, a biotech company in Waltham, Massachusetts. Better prevention through education on safer sexual behavior and more affordable diagnostics will also be needed moving forward. (Amy Maxmen, Nature News)

Maternal Health

U.S. has the Worst Rate of Maternal Deaths in the Developed World

A recent six-month long investigation by NPR and ProPublica has found that more women in the US are dying of pregnancy related complications than any other developed country. Surprisingly, this rate is increasing only in the US, which stood at ~ 26.4 deaths per 100,000 births in 2015, translating to nearly 65,000 deaths annually.  This is three times worse than for women in Canada, and six times worse than for women in Scandinavian countries. Reasons include older new mothers with more complex medical histories, unplanned pregnancies, which are the case half the time in the US, greater prevalence of C-sections, and the fragmented health system. This is in contrast with progress in preventing infant mortality, which has reached historic levels in the US. Better medical training for maternal emergency and more federal funding for research in this area may improve the situation for American mothers. (Nina Martin and Renee Montagne, NPR)

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Science Policy Around the Web – June 23, 2017

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By: Saurav Seshadri, PhD

Drug Policy

Trump’s New Policy to Tackle Sky-High Drug Prices Makes Sense — Sort Of

Tackling high prescription drug prices was a repeated promise of the Trump campaign. The Trump administration has now taken its first step towards fulfilling this pledge, outlined in a blog post by Food and Drug Administration (FDA) commissioner Scott Gottlieb. The agency will pursue a Drug Competition Action Plan, whose goal will be to eliminate obstacles to the development of cheap generic drugs – particularly those caused by loopholes in existing FDA policies, which are exploited by pharmaceutical companies to extend their patent exclusivity period and maximize profits. An example of such ‘gaming’ the system, cited in the post, is the practice of limiting access to branded products for comparative testing by generic developers. Ultimately, the FDA will work closely with the Federal Trade Commission (FTC) to address such issues, since directly regulating business practices is outside its mandate.

On its face, the FDA’s effort is a step in the right direction. Availability of generics reduces the cost of medications by over half within the first year, and according to a recent Congressional report, manufacturers state that ‘competition…is the primary driver of generic drug prices’. However, it ignores evidence that the real driver of increased drug spending is new, branded medicines, not overpriced generics. In fact, early indications are that Trump’s policies will favor the pharmaceutical companies that produce such medicines, by reducing regulations and apparently abandoning his promise to enable the government to negotiate drug pricing through Medicare. Overall, these actions signal a commitment to promoting free market mechanisms in the pharmaceutical industry; time will tell whether this approach will actually lead to more affordable drugs. (Julia Belluz, Vox)

Cancer

In a Major Shift, Cancer Drugs go ‘Tissue-Agnostic’

With the landmark approval of Keytruda in May, the Food and Drug Administration (FDA) appears to have ushered in a new era of cancer drug development.  So far, cancer treatment and drug evaluation have largely used the tumor’s tissue of origin as a starting point. Keytruda (an immune system enabling drug developed by Merck and approved for melanoma in 2014) marked the first departure from this approach, receiving priority approval to treat any solid tumor containing a mutation in the mismatch repair pathway, regardless of context. Recently released data suggests that another tissue-agnostic cancer therapy is on the way: larotrectinib (a cell growth inhibitor developed by Loxo Oncology) showed high efficacy for any tumor with a certain biomarker (TRK fusion). Several other such drugs, whose indications will be based on tumor genetics rather than location, are in the clinical pipeline.

Although these advances have generated significant excitement in the cancer community, some caveats exist. First, identifying the patients that could benefit from tissue-agnostic treatments will require individual initiative and depend on the cost of screening, particularly when considering markers that are rare for a certain tumor type. A potential solution is suggested by the NCI-MATCH trial, part of the NIH’s Precision Medicine Initiative (PMI) – in it, patients can enroll in one of several parallel clinical trials if a corresponding drug-targeted mutation is found in their tumor’s genome. If these trials prove effective, patients could eventually be regularly matched with a personalized, tissue-agnostic, biologically valid treatment, based on a standardized screen.  Second, researchers caution that tissue-agnostic studies should have a strong scientific rationale and/or breakthrough-level efficacy. Otherwise, such efforts ‘could actually slow drug development if there are differential effects across tumor types by diverting resources from enrolling patients in a predominant population or in the tumor type most likely to respond’.

Despite these concerns, the tissue-agnostic paradigm offers great promise for cancer patients. NIH-funded resources such as The Cancer Genome Atlas could be invaluable to this field moving forward. (Ken Garber, Science)

Scientific Publishing

US Court Grants Elsevier Millions in Damages from Sci-Hub

A New York district court has awarded academic publishing giant Elsevier $15 million in damages from Alexandra Elbakyan, founder of the website Sci-Hub, for copyright infringement. Elbakyan, a 27-year-old neuroscientist turned programmer, started Sci-Hub in 2011 with the goal of ‘remov[ing] all barriers in the way of science’. The site allows users to download research papers that would normally be blocked by a paywall, by obtaining credentials from subscribing institutions and using them to access publisher-run databases like ScienceDirect. Over 60 million papers are posted on Sci-Hub, and users downloaded 28 million articles in 2016.

Elbakyan’s case is reminiscent of Aaron Swartz, another high-profile champion of open access to scientific research. Faced with federal charges related to his hacking of journal archive JSTOR, Swartz tragically committed suicide in 2013. Both Elbakyan and Swartz found publishers’ ability to profit from restricting access to scientific literature, effectively withholding knowledge from anyone outside of a privileged inner circle, as well as the legal protection provided to this system, to be deeply unethical. Their willingness to act upon these convictions has earned each a sizable following in the scientific community.

For their part, publishers claim that fees go towards overhead, and point to significant efforts to expand free and open access programs. While judges have so far been sympathetic, Elsevier’s legal battle has been largely one-sided. Elbakyan has been ignoring rulings requiring her to shut down Sci-Hub since 2015, opting to simply change domains instead, and since she is currently based in Russia and has no American assets, she is unlikely to pay any damages. (Quirin Schiermeier, Nature News)

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June 23, 2017 at 11:00 am

Science Policy Around the Web – June 10, 2017

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By: Allison Dennis, BS

Source: pixabay

Animal Testing

Lack of Clarity Puts Chemical Safety and Animal Welfare at Odds

In the lineup of American stereotypes, the health-nut who cares about the chemicals in his shampoo is often the same person who cares if that shampoo was tested on animals or not. However, a bill signed June 22, 2016, known as the Frank R. Lautenberg Chemical Safety for the 21st Century Act, may be placing those two views at odds. The bill requires the U.S. Environmental Protection Agency (EPA) to implement a risk-based process to evaluate the safety of chemical substances currently being used in the marketplace and approve the use of new chemicals before their introduction. The bill was passed with bipartisan support and offered EPA the new-found power to fully regulate the use of well-known carcinogens like asbestos.

Yet the pathway forward for the EPA is daunting. More than 62,000 substances find their way into and onto our bodies through the products we use and our environment. While many of these substances have become associated with disease over time, how can the EPA certify the risks associated with different exposures to varying amounts of each substance on such an extensive list? The Act itself suggested that once the EPA has evaluated the existing information on the 62,000 substances currently in use, it spend the next twelve months triaging chemicals according to their potential risk. Next, the highest priority chemicals will be evaluated on a three-year deadline to develop knowledge of their toxicity and guidelines for their regulation. Ultimately, by clearly cataloging the risk of common chemicals the Frank R. Lautenberg Chemical Safety for the 21st Century Act promises to greatly reduce the amount of animal testing needed in the long-term.

In the meantime, however, the companies that use to-be-regulated substances in their products may be inclined to undertake independent toxicity testing, collecting enough data to guarantee that their favorite substances meet the low-risk criteria and avoid a drawn-out evaluation. Defining toxicity requires careful experimentation, which can sometimes be carried out in human cells outside of the body, but often require evaluation in animals. Animal rights groups like the Human Society find concern with the lack of transparency in the pre-prioritization process. They fear the eagerness of companies to provide data without any clear guidelines about how that data will be evaluated or what substances will require extensive evaluation could result in extensive and unnecessary animal testing. Further they suggested that the EPA require any new pre-approval data obtained by companies to be collected using non-animal methods. (Maggie Koerth-Baker, FiveThirtyEight)

CRISPR

Small Study may Reveal Big Concerns over CRISPR-Based Therapy

A one-page letter published in Nature Methods last week reports unexpectedly high levels of unintended changes to the genomes of mice that underwent a CRISPR-based therapy. Since it’s renaissance as a therapeutic tool in 2012, CRISPR has occupied the imaginations of scientists, doctors, patients, investors, and ethicists. CRISPR technology provides a relatively straight-forward and reproducible means to gene editing on the cellular level, but its applications to create heritable mutations in the human germ line is on hold until more is understood about the long-term effects such treatments would have.

The original study sought to explore potential long-term effects of germline manipulation by CRISPR in a mouse model. Guide RNA along with the Cas9 enzyme were injected into mouse zygotes, which introduced a correction in a mutation in the rd1 gene of otherwise blind mice. Initiating this change before the first cell division enabled this corrected mutation to be inherited by all cells arising in the developing mouse, consequently restoring the ability for the eyes to develop normally. In a follow-up experiment described in their one page letter, the researchers looked for mutations in the genomic DNA of two CRISPR-treated adult mice compared with a control mouse, revealing over 2,000 unintended mutations following CRISPR treatments. None of these mutations appeared to affect the mice, suggesting that deep genomic sequencing may be required to reveal unanticipated changes in an outwardly healthy mouse. Further, the nature of these unintended mutations offered few clues explaining how they might have occurred.

This result stands in contrast with other reports quantifying the extent of these unintended changes, which found CRISPR to be highly specific. While the CRISPR-Cas9 system has been observed to sometimes alter off-target regions of the genome, this activity can usually be curbed through the careful design and evaluation of the guide RNA. The limitations of this small study have been discussed extensively since its publication. However, the findings have sparked the need for further investigation into the long-term-whole-animal effects of germline-editing by CRISPR. As human germline-editing creeps closer to reality, the FDA will be tasked with developing an entirely new means of evaluating the safety of such technologies (Megan Molteni, Wired)

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June 10, 2017 at 11:33 am

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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May 24, 2017 at 5:04 pm

Science Policy Around the Web – May 2, 2017

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By: Allison Dennis, BS

Public Health

You Can’t See What You Don’t Test For

The CDC relies on blood testing by pediatric health care providers to monitor lead exposure in children nationwide. However, many doctors may not be prompted to seek a test. According to a recent study, only half of estimated cases of elevated blood lead levels were reported to the CDC. From 1999 to 2010 an estimated 1.2 million cases of elevated lead blood level counts were predicted by state-by-state surveys conducted by the CDC. In contrast, only 607,000 cases were reported to the CDC by the 39 states that report elevated blood lead levels.

There is no safe threshold for lead exposure, however the CDC moved to revise the acceptable limit to 5ug/dL from 10ug/dL in 2012. Currently all doctors who treat children receiving Medicaid benefits are required to submit lead level tests for children at age 12 and 24 months or for those who have not received a test by the age of 7 years to meet formal eligibility. However, the number of tests performed annually falls short of those expected if doctors were following the rules. Each state maintains its own guidelines for when doctors should request testing and report to the CDC. And 12 states do not submit data to the CDC.

The greatest discrepancies between numbers predicted from survey data compared with state reported values were observed in Western states. When diagnosing lead exposure doctors may overlook environmental risk factors when treating patients in communities where lead has not been an issue historically. While public housing in Northeast cities like Baltimore have been recognized as a systemic source of lead exposure, the risk may not be so obvious in communities in California where buildings are assumed to be newer than 1978 when lead paint was banned nationally. (Susan Scutti, CNN)

Drug Policy

Access To Lethal Injection Drugs Challenges States To Get Creative

The FDA ruled on Thursday, April 20th that the Texas Department of Criminal Justice and the Arizona Department of Corrections have 90 days to destroy or return a batch of execution drugs imported from India. The Texas bound shipment seized by the FDA in July 2015 consisted of a thousand or more vials of sodium thiopental, which is used as the anesthetic in three-drug lethal injection protocols. While the FDA had abstained from enforcing a law preventing the import of sodium thiopental, which has no approved use in the United States, a US District Court permanently ordered the FDA to impose the ban.

Implementation of the import ban on sodium thiopental follows a long line of restricted access to lethal injection drugs. Political pressure on drug manufacturers has dramatically reduced the availability of products appropriate for use in lethal injection protocols. The last US manufacturer of sodium thiopental, Hospira, discontinued its production in 2011. In 2012, the European Commission blocked the export of drug for lethal injection to the United States. In May 2016, Pfizer announced it would end the use of its products in lethal injections, making it the final FDA approved manufacturer of potential drugs to do so. States have been responding by adapting protocols to meet drug availability or seeking third-party suppliers.

The modern use of lethal injections for corporate punishment were proposed in 1977 by the Oklahoma state medical examiner as being more humane and was first executed in Texas in 1982. While the practice is associated with drugs and medical professionals, it has never been subject to clinical trials or peer review. (Susan Scutti, CNN)

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May 2, 2017 at 10:08 am