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Posts Tagged ‘clinical trials

Science Policy Around the Web – November 20, 2018

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By: Andrew Wright, B.S.

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Source: Wikimedia

 

Habitat Loss Threatens All Our Futures, World Leaders Warned

Recent reports have suggested that humanity has only 12 years to avoid catastrophic environmental collapse due to 1.5C of industrial warming. While solutions to the threat of runaway climate change have been given a new sense of urgency by these findings, there exists a commensurate and oft less visited issue, that of rapid declines in global biological diversity. Driven primarily by agricultural land conversion of terrestrial and marine ecosystems (via forest clearing and river damming, respectively), vertebrate species have declined by 60% on average since 1970 according to the World Wildlife Fund’s most recent Living Planet Report . While this decline appears strongest in South and Central America and in freshwater habitats, the report joins a compendium of literature suggesting holistic declines in biodiversity among birds, insects, fish, and terrestrial vertebrates as part of an ongoing anthropogenic mass extinction event.

To address some of the issue, the UN Convention on Biological Diversity (CBD) is currently meeting in Sharm El Sheikh, Egypt to discuss progress on the Aichi biodiversity targets for 2020.  These targets came out of The Convention on Biological Diversity, a multilateral treaty signed in 1992 focused on preserving biodiversity, sustainable use of biological resources, and equitable sharing of resources. The Aichi biodiversity targets specified that people would be aware of risks to biodiversity, and that biodiversity values would be adopted by public, private, and governmental entities by 2020. Given the rapidity, intensity, and ubiquity of the decline in species, most, if not all, of these targets will likely be missed. As such, the delegates from the 196 signatory nations will also work on creating new biodiversity targets to be finalized at the next CBD meeting in China.

Since a comprehensive solution seems necessary given the increasingly global nature of trade, authors of the new targets hope to garner a greater deal of international attention, and intend to make the case that government commitment to reversing or pausing biodiversity loss should receive equivalent weight as action on climate change.

(Jonathan Watts, The Guardian)

The Ethical Quandary of Human Infection Studies

The United States has greatly improved its ability to soundly regulate the ethics of clinical studies since the infamous malfeasance of the Tuskegee syphilis study. Most significantly, the National Research Act of 1974 established the Institutional Review Board to address how to adequately regulate the use of human subjects by adhering to the principles of respect for persons, beneficence, and justice.

The National Research Act provided a substantial step forward and provided a clear requirement for universal informed consent. However, the expansion of clinical studies to new international regions of extreme poverty, due in part to the influx of private money from large charitable organizations, has come with novel ethical considerations. In these newly explored populations where income, education, and literacy levels may be lower, emphasis is now being place on how to recruit volunteers without implicitly taking advantage of their circumstances.

One area of concern is compensation levels. While compensation in a malaria infection study in Kenya was tied to the local minimum wage, the number of volunteers recruited far surpassed expectations. This may have been due to the fact that payment during this study was guaranteed and consistent, in contrast to local work.

Aware of the concern, two of the largest private medical research funding organizations, the Bill and Melinda Gates foundation and the Wellcome Trust have recently instituted ethical guidelines putatively reinforcing the principle of beneficence, placing special emphasis on maximizing benefits over risk. It is an open question whether these protections will be sufficient, but at the very least it is important that rules to be put in place proactively rather than as a reaction.

 

(Linda Nordling, Undark/Scientific American)

 

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November 20, 2018 at 11:58 am

Science Policy Around the Web – September 19, 2018

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By: Saurav Seshadri, Ph.D

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Clinical Trials

Universities are worse than drug companies at reporting clinical trial results

Reporting the methods and results of clinical trials, in a timely and accurate manner, is critical to the process of drug development.  Such reporting is described by the World Health Organization as a ‘scientific, ethical, and moral responsibility’.  However, a recent study conducted by Oxford University’s Evidence-Based Medicine (EBM) DataLab has found that less than half of clinical trials conducted in the EU since 2004 actually fulfilled this responsibility.  In fact, an abysmal 11% of trials with non-commercial sponsors had reported their results, with many institutions reporting results for 0% of up to 200 registered trials.  Industry sponsors did a better job, with almost 70% of trial results reported, and higher rates of reporting among companies with more trials.  Of equal concern, inaccurate reporting appeared to be rampant in the EU Clinical Trials Register database.  For example, 30% of trials that were listed as ‘completed’ or ‘terminated’ did not have an end date, and therefore couldn’t be evaluated for timeliness (results are due after 1 year).

Clinical trial data reporting is governed by a similar system in the US: sponsors are required by the FDA to post results on ClinicalTrials.gov within a year of completion.  But the problem of lax reporting is well known, and the current results are in line with previous reviews.  In February, the EBM DataLab launched a tool to monitor trial reporting, aiming to increase public accountability by ranking sponsors, tabulating potential fines, and regularly notifying the FDA of non-compliance.  Legally, investigators can be charged $11,569 per day for late reporting, and according to the tracker, unclaimed fines now add up to more than 650 million dollars.  The Oxford team recently launched a similar tool for the EU.

Many factors are likely to contribute to the problem of non-compliance in academia.  These include an absence of a ‘culture of reporting’, unfamiliarity with data curation or registration procedures, pressure to save data for a high-profile publication, and unwillingness to accept negative results.  In the end, effecting a change in academic reporting habits will require a combination of public scrutiny and institutional support.  The EBM DataLab has at least the first part well in hand.

(Tania Rabesandratana, Science)

Health insurance

Merger of Cigna and Express Scripts Gets Approval From Justice Dept.

In a recent announcement, the Department of Justice (DOJ) appears to have cleared the way for a more consolidated healthcare landscape.  After over a year of review, the DOJ has granted approval for the $52 billion merger between Cigna, a major health insurance company, and Express Scripts, a leading pharmacy benefits manager (PBM).  According to the companies, this union between the entities who pay for and provide prescription drugs will allow them to share patient information and eventually negotiate better drug pricing.  While the details are unclear, the DOJ apparently agrees on the principle; a separate merger between Aetna and CVS Health, valued at $69 billion, is now expected to be greenlit as well.

The decision represents a path forward for insurers, whose previous attempts to merge with each other were blocked by federal antitrust officials.  Overall, the shift towards vertical integration is largely a response to Amazon, which caused many health companies’ stocks to plummet when it revealed its intention to enter the healthcare industry earlier this year.  Amazon’s partnership with investment titans Berkshire Hathaway and JPMorgan Chase & Co, as well as its purchase of online pharmacy Pillpack.com, make it a credible threat even to well-established companies in the notoriously complicated healthcare industry.  Until recently, the Cigna-Express Scripts deal itself was vehemently opposed by activist investor Carl Icahn, who urged shareholders to vote against it on the grounds that competition from Amazon would soon render the PBM model inviable.

The mergers may also be an attempt by established companies to show that they are trying to adapt their business models to address growing public dissatisfaction with drug pricing and access.  However, there is no guarantee that any discounts that these more powerful, merged organizations are able to obtain will be passed on to customers.  Critics ranging from the pharmaceutical lobby to the Trump administration have suggested that PBMs are designed to maximize profit rather than help patients.  On the other hand, the merger may increase competition among insurers (and thereby benefit patients) by enabling Cigna to enter new markets.  With Amazon closing in, healthcare companies’ survival may ultimately depend on their ability to balance putting themselves or their customers first.

(Reed Abelson, The New York Times)

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September 19, 2018 at 11:35 am

Science Policy Around the Web – July 27, 2018

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By: Emily Petrus, Ph.D.

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Innovation

Artificial Intelligence Has a Bias Problem, and It’s Our Fault

While computer and data scientists are working to create systems which can reason and perform complex analysis, groups of ethicists, lawyers and human rights advocates express increasing concerns about the impact artificial intelligence will have on life. It is becoming apparent that human bias regarding race, gender and socioeconomic position also influence algorithms and data sets used to train machine learning software.

Most artificial intelligence (AI) systems are trained on data sets culled from the internet. This results in skewed data which over-represents images and language from the United States. For example, a white woman in a white dress results in algorithms labeling a picture as “bride” or “wedding”, while an image of a North Indian bride is labeled as “performance art”. If that seems like a harmless hiccup, think about algorithms designed to detect skin cancer from images. A recently published study did a decent job detecting dark moles on light skin, but only 5% of the data set depicted dark skinned people, and the algorithm wasn’t even tested on that data set. This bias could skew accurate diagnoses for already underserved minority populations in the United States. Finally, AI will have a huge impact on financial markets beyond the replacement of humans to do jobs, particularly in manufacturing. Decisions on loan eligibility and job candidate hiring decisions are being filtered through AI technology, which is guided by data which may be biased.

It is apparent that computer scientists must make concerted efforts to un-bias data training sets and increase transparency when they develop new AI systems. Unfortunately, these common-sense suggestions are just that: suggestions. Before Obama left office in Fall 2016, a roadmap was created by the administration to guide research and development of AI systems. There’s no teeth in policy dictating fairness and inclusivity in AI development, but private and academic institutions are making gains in this arena. The Human-Centered AI project at Stanford University and Fairness, Accountability, Transparency, and Ethics (FATE) in AI research group at Microsoft are two examples of these types of efforts. Both groups seek to increase inclusivity in AI algorithms and reduce bias – human and computer generated. AI can also be trained to detect biases in both training data and the models by conducting an AI audit. An effort of developers in academia and private industry will be necessary to produce and prove their AI is unbiased, and it is unlikely that federal regulations would have the power or dexterity to administer any concrete regulations regarding this technology. Like most other scientific advances which bring significant monetary gains, the pace is breakneck but corners should not be cut. Legislation is unlikely to be able to keep up with the technology, but incentives to keep the playing field fair should come from within the AI community itself.

(Ben Dickson, PC Mag)

Scientific oversight

NIH delays controversial clinical trials policy for some studies

How does the brain process images of faces? How do we respond to frustrating situations? What does the mind of a sociopath look like in an MRI? These are all basic science questions in brain research which may discover treatment options for future studies. But for the moment, no drugs or interventions are being tested in many basic research labs funded by the National Institutes of Health (NIH). This means they’re not clinical interventions, or by definition, clinical trials, right? Maybe…

Basic researchers studying the healthy human brain sigh a breath of relief as the NIH decided to delay new rules applying to the classification of human trials. At issue is the re-classification of research which can be considered a clinical trial. The intent of the new guidelines was to increase reproducibility and transparency in government funded human research, for example requiring more rigorous statistical practices.  In practice, investigators will be required to upload their studies to clinicaltrials.gov, take mandatory trainings, and produce significantly more paperwork to continue receiving funding for their basic research. In addition, researchers were concerned that this would create more confusion in the public, as their research would be inaccurately represented as a clinical trial.

After the announcement last year, professional societies and academics sent letters of complaint to NIH, prompting congress to delay the implementation of the requirements to September 2019. This delay also gives leniency to basic researchers who apply to funding opportunity announcements seeking studies labeled as clinical trials, meaning they would not be immediately disqualified from being scored. Although many researchers hoped the NIH would drop all requirements for basic research, the delay is welcome for now. “This delay is progress because it gives them more time to get it right, and in the interim people aren’t going to be in trouble if they get it wrong,” said Jeremy Wolfe, a cognitive psychologist at Harvard Medical School.

(Jocelyn Kaiser, Science)

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July 27, 2018 at 4:51 pm

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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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February 20, 2018 at 3:52 pm

FDA stem cell therapy crackdown: a stem-free clinic

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By: Belinda Hauser, Ph.D.

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source: pixabay

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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January 17, 2018 at 11:43 am

Science Policy Around the Web – September 12, 2017

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By: Saurav Seshadri, PhD

Vaccination Medicine Vaccine Ampoules

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Infectious Diseases

Is a Zika vaccine worth the effort?

A collaboration between pharmaceutical giant Sanofi and the US Army to develop a vaccine for the Zika virus has come to an end.  About a year ago, Sanofi received $43.2 million in funding from the Biomedical Advanced Research and Development Authority (BARDA, a division of DHHS) to move a Zika vaccine candidate, generated by the Walter Reed Army Institute of Research, into Phase II development.  BARDA has now decided to ‘de-scope’ the project, leading Sanofi to abandon its efforts to develop or license the candidate.

The number of cases of Zika has declined sharply since its peak in early 2016.  While this ‘evolving epidemiology’ has hampered Zika-related clinical research and drug development, it may actually be a welcome relief for Sanofi. The French company has endured months of political pressure to agree to pricing assurances for any vaccine produced from the collaboration, with lawmakers, including Senator Bernie Sanders, arguing that it would be a ‘bad deal‘ for a private company to profit from research funded in part by American taxpayers.  In particular, the exclusivity of Sanofi’s license,  uncommon for such agreements, has been singled out as ‘monopolistic’.  Sanofi has been defending itself vigorously against this characterization, pointing out that it took on significant risk itself for a vaccine that was far from approval, and that it has already discussed reimbursing the US government for its investment through milestone and royalty payments.  Ultimately, ending the collaboration puts this PR-damaging debate to rest, while also providing Sanofi a face-saving opportunity to avoid committing to a drug with limited prospective demand and profitability (as recently transpired with the dengue fever vaccine Dengvaxia, which only reached 55 of its projected 200 million euros in sales in 2016).

In its statement, Sanofi says that it continues ‘to believe that public-private partnerships are the right model to address…public health challenges’ posed by infectious diseases.  Indeed, several pharmaceutical companies responded to the WHO’s declaration of Zika as a public health emergency in 2016; of these, Takeda and Moderna appear to still have ongoing large-scale collaborations with BARDA to develop Zika vaccines.  While the drop in Zika prevalence is clearly a good thing, it’s unclear how it will affect the economic and scientific feasibility of such collaborations in the future.  One solution is to promote vaccine development before an outbreak occurs: groups such as the Coalition for Epidemic Preparedness Innovations (CEPI) hope to facilitate this approach, but the need to allocate limited resources makes its practicality questionable.  However, the alternative is usefully illustrated by the Ebola epidemic of 2014.  Despite concerted global efforts that led to successful vaccine development by Merck, current outbreaks are small enough that the deployment of vaccines may not even be warranted.  Barring an overhaul of regulatory processes and/or financial priorities, it seems likely that when the next epidemic emerges, we’ll be playing catch-up again.

(Eric Sagonowsky, FiercePharma)

Neuroscience

Is every human brain study a clinical trial?

Basic research into the mechanisms underlying cognition and their impairment in a range of brain disorders is the primary focus of hundreds of neuroscience laboratories.  While such studies feed into drug discovery for diseases such as autism, schizophrenia, and bipolar disorder, since they do not directly involve testing any treatments, they are not commonly considered to be clinical trials.  This perception became technically incorrect in 2014, following an NIH announcement broadly redefining ‘clinical trial’ to include any study in which ‘one or more human subjects’ receive an intervention and ‘health-related biomedical or behavioral outcomes’ are observed.  Last year, the NIH revised its data reporting policies for such trials.  These more stringent policies are now being implemented, and will affect grant applications submitted in 2018.

Several members of the scientific community have begun to voice their concern about the changes.  The Association for Psychological Science (APS) and Federation of Associations in Behavioral & Brain Sciences (FABBS) have both sent critical letters to the NIH. A petition by neuroscience researchers pushing back against the policy has garnered over 3,400 signatures.  Opponents argue that the requirements imposed by the ‘clinical trial’ label are overly burdensome and would impede basic research.  These requirements include timely study registration and public disclosure of results through ClinicalTrials.gov. Further, they demand that all staff receive Good Clinical Practices training. Investigators dread the bureaucracy that will be involved in complying with these mandates.  Perhaps most concerning for scientists is the constraint that new proposals must respond to a Funding Opportunity Announcement, which have specific stipulations about study objectives, design, oversight, and evaluation.  While these rules are intended to promote scientific rigor and transparency, the more immediate effects may be to stifle exploration and creativity and to deter basic researchers who may not know how to tailor their applications to reflect clinical values.

For its part, the NIH is steadfast that the broad redefinition is ‘intentional’ and that current standards of data reporting are ‘unacceptable’.  Policymakers argue that they are simply asking scientists to inform the public about the existence and outcome of their research.  While this sounds unimpeachable in theory, scientists are already reporting practical challenges: for example, asking potential study participants to sign a clinical trial consent form can scare them away.  While the NIH is making efforts to provide guidance to the community, it is running out of time to stamp out confusion before next January, let alone achieve enthusiastic compliance.  Neuroscientists are likely to face setbacks in funding and progress as a result.

(Sara Reardon, Nature News)

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September 12, 2017 at 5:52 pm

Science Policy Around the Web – July 21, 2017

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By: Rachel F Smallwood, PhD

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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