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Posts Tagged ‘NCI

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 – April 14, 2015

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By: Elisavet Serti, Ph.D.

Ebola and Public Health

After Ebola devastation, a measles outbreak threatens West Africa

A second health crisis, after the most widespread Ebola epidemic in history, is threatening thousands of people in West Africa. The Ebola crisis has devastated public health systems and, as a result, has slowed down measles immunizations, a viral lethal disease that could affect the region even worse than Ebola. Even before Ebola hit, only an estimated 62% to 79% of children were vaccinated against measles in Liberia, Guinea, and Sierra in 2012 and 2013, according to the Demographic and Health Surveys — and that was with only one dose of the vaccine. Because the virus is so exquisitely transmissible, 95% of the population must be protected with two doses of vaccine to stop measles. “The secondary effects of Ebola are likely to be as bad as or worse than the direct effects,” says epidemiologist Justin Lessler of Johns Hopkins Bloomberg School of Public Health in Baltimore, Maryland, who with his collaborators warned about a potential post-Ebola measles outbreak.

The symptoms of measles generally appear about seven to 14 days after a person is infected and include fever, runny nose, cough, red eyes, and sore throat, followed by a rash that spreads over the body. The measles virus is highly contagious and spreads through the air through coughing and sneezing. Common measles complications include ear infections and diarrhea but some people may suffer from severe complications, such as pneumonia and encephalitis that could be lethal. In the United States, as many as one out of every 20 children with measles gets pneumonia, the most common cause of death from measles in young children. Encephalitis is less common (1 out every 1000 children), which can leave the child deaf, or with intellectual disability. Measles is characterized as one of the most contagious viruses on Earth — five to 10 times more infectious than Ebola — and is among the first diseases to erupt in the wake of a disaster. In humanitarian crises in poor countries, it can kill up to 20% of those infected; usually those weakened by malnutrition and vitamin A deficiency.

Dr. Lessler and his collaborators estimated in a new study that a regional post-Ebola measles epidemic would strike roughly twice as many people as a pre-Ebola epidemic; an estimated 227,000 of infections compared with 127,000, and cause 2000 to 16,000 additional deaths. At the high end, the death toll could exceed the nearly 10,000 people killed by Ebola to date. However, nearly all of those deaths could be avoided by effective mass vaccination campaigns. That is why Liberia, with help from Centers for Disease Control and other international partners, is trying to launch a measles campaign as early as May that will target all children between 9 months and 5 years of age. Sierra Leone and Guinea are still affected by the Ebola disease so planning for measles vaccination is not considered a priority for the time being. (Leslie Roberts, Science)

Federal Biomedical Research

National Cancer Institute Director steps down

Harold E. Varmus, the Nobel Prize winner cancer biologist who has led the the National Cancer Institute (NCI) at the National Institutes of Health for nearly five years, stepped down from his post on March 31st. Dr. Varmus was co-recipient of the 1989 Nobel Prize for Physiology or Medicine for research into the cellular origins of cancer while conducting research at the University of California, San Francisco, where he joined the faculty in 1971. He was director of the entire NIH under President Bill Clinton from 1993 to 1999. Dr. Collins, the current NIH director, praised Dr. Varmus as being gifted with “unparalleled expertise.”

In his farewell letter to NCI staff, Dr. Varmus stressed the advances in oncology that NCI researchers led during his time as the NCI director in spite of the congressionally imposed budget cuts and budget sequestration that his institute and the entire NIH have had to endure in recent years. He wrote that his years at NCI “have not been easy ones for managing this large enterprise” and that “we have endured losses in real as well as adjusted dollars, survived the threats and reality of government shutdowns and have not yet recovered all the funds that sequestration has taken away.” He noted a range of scientific breakthroughs that have stemmed in part from NCI investments in recent years, from more widespread use of the HPV vaccine to greater acceptance of imaging tests in heavy smokers to pinpoint lung cancer, based on an NCI trial.

Dr. Varmus plans to return to New York and will head a laboratory in the Meyer Cancer Center at Weill-Cornell Medical College and also work with the New York Genome Center. Dr. Douglas Lowy, currently deputy director of the NCI and a researcher whose work led to development of the HPV vaccine to prevent cervical cancer, will become acting director April 1. (Thomas M Burton, Wall Street Journal)

Emergency Medicine Response

Having a companion is associated with faster treatment of stroke victims

The human element is much more significant in emergency medicine than initially believed by researchers. Dr. Gal Ifergane, a neurologist at Soroka University Medical Centre in southern Israel recently published in Medicine a striking story about the positive impact that relatives and friends have in the care and treatment of stroke victims when they accompany them to the ER. The most common stroke involves a clot blocking blood vessels in the brain, which causes almost immediate brain cell death because of the absence of oxygen transport. Thrombolytic therapy is very effective and uses drugs to dissolve the clot and restore the flow of blood. If started within a couple of hours of a stroke occurring, it can limit brain damage and reduce long-term disability. Time is a matter of life and death for these patients. Another key step is using a computed tomography (CT) scanner to ensure that there has been no bleeding in the brain, in which case thrombolytic drugs would cause excessive bleeding and would probably be lethal for the patient. In order to reduce the time to CT scan and correct treatment, paramedics have been trained to recognize strokes and warn hospitals in advance so that the medical team prepares accordingly.

Evidence for the significance of the human element in this case of emergency medicine came from 15 months of observation at Soroka Medical Center, where the ER staff recorded the number of companions escorting each stroke sufferer (over 700 in all) and monitored their progress. The results of this study showed that stroke victims arriving with someone were more than twice as likely to be correctly diagnosed by the triage nurse, and had their CT scans performed earlier. Patients eligible for thrombolytic medication also received treatment much faster if accompanied; although the patient sample was too small for the researchers to be sure it was because they had relatives or friends bringing them into the ER. The differences between the two groups cannot go unnoticed; patients with one companion had CT scans an average of 15 minutes sooner than those unaccompanied. A second companion reduced this time wait 20 minutes more; however, three or more companions did not confer any additional benefit. Dr. Ifergane believes that this is probably a combination of focusing the attention of clinical staff on their loved ones, and providing basic care when they initially arrive at the hospital. (The Economist)

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April 14, 2015 at 11:36 am

Science Policy Around the Web – March 20, 2015

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By: Amanda Whiting, Ph.D

Biotechnology and Bioethics

Scientists Seek Ban on Method of Editing the Human Genome

With great power comes great responsibility. The inventors of a simple and effective technique for editing and making heritable changes to human DNA have called for a worldwide ban on exactly that until the scientific and ethical consequences can be fully studied and evaluated. While the technique, known as CRISPR-Cas9, can and is used in laboratory research experiments, the authors of a recent paper are concerned about the ability of physicians and scientists to push ahead into human DNA. While clinical use of this technology is highly regulated in the United States and Europe, the paper’s authors are concerned about research in countries with fewer regulations and urge that “scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans” until the full implications “are discussed among scientific and governmental organizations.”

The ethical considerations of such a technological ability are huge – the power to edit, repair, alter or enhance any part of the human genome in a way that can be passed on to offspring would have major implications for future generations of humans. But where do you draw the line? It’s one thing to want to correct a genetic flaw leading to a known and crippling illness, or to potentially free a family from a legacy of disease. It’s another to edit a “flaw” based on one opinion or standard to be more “beautiful” or “intelligent.” There are also concerns over potential mistakes – when the DNA is accidently changed in a way not intended – as well as with the actual consequences of making a “correct” change when the entire spectrum of effects is not known or well-understood at this time. Should any editing take place at all in humans? Is the power to direct our own genome too much?

Science in the 21st century seems to follow a trend of first developing a technique, trying to understand the consequences of using that technique, and then finally developing the necessary policy. With this ban, perhaps policy will have time to catch up to our scientific ability. (Nicholas Wade, New York Times)

Federal Research Funding

Cancer institute plans new award for staff scientists

The National Cancer Institute (NCI) is planning to try out a new “experiment” in funding science by targeting a new award at staff scientists, rather than graduate students, post-doctoral researchers or principle investigators (PI). In this way, NCI hopes to address some of the current flaws in biomedical funding, which encourage labs to over rely on (cheaper) trainees to do research (rather than longer term employees), creating an over-abundance of highly trained post-docs to very few actual PI positions at the end of the day.

The K05 “research specialist award” would be aimed at researchers with a masters, Ph.D., M.D., or other advanced degree and the applicant would need to be sponsored by a PI and the institution at which they would work. These 5 year, renewable rewards would cover 100% of the cost of the scientist’s salary (but not any supplies), and would be portable if the scientist chose to move to another lab. While mostly positively received, there are uncertainties such as creating even more competition for a shrinking pool of federal funding, and the worry that NCI would be swamped with applications. NCI plans to start with approximately 50 to 60 awards, totaling $5 million, over the next 18 months. Requests for applications can be expected later this year. (Jocelyn Kaiser, ScienceInsider)

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Written by sciencepolicyforall

March 20, 2015 at 1:06 pm