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Science Policy Around the Web – February 13, 2018

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By: Saurav Seshadri, PhD

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Experimental drugs

Trump Endorses “Right to Try” for Terminally Ill Patients

Proponents of the ‘right to try’ received some encouragement from President Trump’s recent State of the Union address, in which he announced his support for such legislation at the federal level.  Right to Try laws are designed to allow terminally ill patients to obtain unapproved but possibly lifesaving drugs directly from pharmaceutical companies, without involving the FDA.  While such laws already exist in 38 states, they are currently superseded by the Food, Drug, and Cosmetic Act; a bill that would eliminate this legal conflict was passed by the Senate last August, but has yet to be approved by the House of Representatives.

In general, Right to Try laws permit terminal patients, with their informed consent, to access investigational treatments if recommended by a physician.  However, they do not mandate that the manufacturer provide the drug or that insurance cover it, and in some cases, they absolve drugmakers and physicians from liability for adverse outcomes.  In addition, the FDA already offers a path to treatment for terminal patients under its ‘expanded access’ program, in which patients are treated as clinical trial participants and their doctor’s office becomes a satellite site, with appropriate regulatory oversight.  Opponents of Right to Try legislation, including FDA Commissioner Scott Gottlieb, argue that bypassing such oversight would critically undermine the clinical trial process (for example, a patient death from a drug obtained under a Right to Try law would not factor into the FDA’s consideration of that drug for approval).  They also suggest that these laws provide false hope for desperate patients – experimental drugs need only clear the safety phase of FDA trials, meaning no data exists on their efficacy – and open patients up to risks of physical harm and medical fraud.

Despite these concerns, Right to Try laws have gained momentum on the strength of anecdotal success stories, and politicians’ unwillingness to appear heartless towards patients suffering from terminal diseases.  Yet in reality, without securing financial support for patients, these laws are likely to result in some patients going bankrupt. Without requiring that treatments be demonstrated to be beneficial and at least safe, these laws are likely to result in patients pursuing ineffective treatments, while reducing their quality of life by enduring side effects, risking complications, and forgoing hospice care.  The future of Right to Try legislation may be influenced by new Health and Homeland Security Secretary (and former Eli Lilly executive) Alex Azar, who seems likely to support Trump’s agenda, though he didn’t mention the right to try in his response to the State of the Union address.  Ideally, the final bill will prioritize the existing drug review process, ensuring safety for the majority of patients while still providing hope for the sickest.

(Ike Swetlitz, STAT news)

Chemical safety

The truth about glyphosate may be getting lost in the weeds

The World Health Organization (WHO) kicked off a massive controversy in 2015 with its report labeling glyphosate, a component of an herbicide marketed by Monsanto, as ‘probably carcinogenic to humans’.  The report has faced stiff opposition from Republican Representatives on the US House Science, Space, and Technology Committee, largely fueled by a pair of Reuters reports suggesting that key data was suppressed by the WHO to support its conclusion.  Now Dr. Christopher Wild, Director of the group that conducted the research (the IARC, International Agency for Research on Cancer) has sent a detailed response to the Committee to rebut these criticisms and defend its original finding.

The response, which was presented at a recent Committee hearing by Democratic Representative Suzanne Bonamici, specifically addresses two issues raised by Reuters.  First, that a senior scientist failed to disclose data that would have exonerated glyphosate: the data was unpublished and therefore didn’t meet IARC’s criteria for consideration.  Second, that the published version of the report had several changes from an earlier draft, all of which involved deleting or revising statements that cast doubt on glyphosate’s link to cancer.  Dr. Wild claims that most of these changes were related to a single review article, whose conclusions were reconsidered when it was found to have been ghostwritten by a Monsanto scientist, and that its drafts are works in progress and therefore confidential.  Still, the response doesn’t explain the IARC’s discrepancy with other regulatory agencies: the European Food Safety Authority (EFSA) and US Environmental Protection Agency (EPA) have both found glyphosate to be safe, and claim their review processes are more transparent than the IARC’s.

The IARC’s stance on glyphosate puts it in a delicate position with the US government, from which it receives ‘valuable support’, especially as the topic becomes more partisan.  Republican lawmakers have already threatened to pull funding to the IARC, ostensibly over its refusal to provide a witness for the hearing (Dr. Wild invited them to visit his facility in France instead).  On the other side, the EPA’s assessment has been called into question by the discovery that an EPA official may have colluded with Monsanto to ‘kill’ investigation into glyphosate, leading Democratic Representative Ted Lieu to request a probe into the issue.  In the midst of a heated debate on climate change, the glyphosate story may initially seem to be another case of Republicans denying science to fight regulations and side with big business; however, the reality may be more complicated.  A recent protest in Paris by farmers, opposed to a proposed ban on glyphosate, highlights how those most affected by such policies must balance their economic stability against potential health risks.  Ultimately, though lawmakers may earn political points by siding with these individuals, if the price is discrediting accurate science and eroding public trust in regulatory agencies, no one wins.

(Corbin Hiar, E&E News)

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February 13, 2018 at 6:01 pm

Science Policy Around the Web – January 19, 2018

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By: Allison Dennis B.S.

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Emergency Unpreparedness

IV bag shortage has hospitals scrambling to treat flu

While other hospital activities may put a predictable strain on medical supplies, the sudden onset of a particularly bad flu season has left hospitals strapped for the basic medical staple, IV bags. Intravenous (IV) therapy delivers liquids directly to the vein and is made possible by prepackaged sterile bags loaded with saline, a mix of water, salt, sugar, electrolytes, and vitamins that match what naturally exists in the body. By matching the natural composition of blood, these fluids are able to help the body rapidly return to normal after dehydration and can efficiently deliver drugs. Severe dehydration is a common side effect of flu, as one of the body’s first line of defense is to develop a fever, a process that expends a lot of water and oxygen. Additional symptoms may leave flu-sufferers uninterested or unable to drink the water they need. For patients ill enough to seek treatment at the hospital, IV therapy is often required to rapidly rehydrate their bodies and can be used simultaneously to deliver antivirals.

IV bags have been continuously in short supply in the US since 2014. Reasons for this shortage seem to stem from the complexities of safely manufacturing saline, a 10-day process that reportedly requires 29 steps, and the insatiable demand, 740 IV bags are estimated to be used each minute in the US. Production of IV drugs and saline is more tightly regulated by the FDA than other drugs because they are injected directly into the blood stream. Even the smallest contamination can result in a widespread blood infection.

In recent months, the shortage has been heightened by the coalescing of two closely monitored seasons, flu and hurricane. Half of the IV bags used in the US are manufactured in Puerto Rico, which was devastated by hurricane damage early this fall. IV bag producers are slowly returning to their pre-storm levels of production, but ongoing power outages are continuing to cause disruption. To try to alleviate this burden, the FDA has granted additional companies permission to begin manufacturing and selling the bags that are in short supply. To help hospitals struggling to meet the constant demand for IV bags, the FDA is temporarily permitting hospitals to import sterile saline from overseas.

In some cases, care providers are able to substitute pills for drugs usually administered intravenously. In others, providers may choose to administer drugs through an I.V. push, directly injecting them into the vein, a method that can be both painful and time consuming. But when it comes to treating the severe dehydration that can result when the body battle the flu, intravenous rehydration is often the only appropriate treatment.

(Linda Johnson, Associated Press)

Technology

After years of avoidance, Department of Energy joins quest to develop quantum computers

Quantum computing promises to revolutionize the way we solve complex problems through computation. While the hardware needed to make this a reality exists, software developers and thinkers are struggling to catch up. Conventional computers use bits, either 0 or 1, to create logic in a language the computer can understand. Quantum computers would expand this language to capture the ability of subatomic particles to exist in more than one state at a time. Instead of bits, these computers would use qubits, or quantum bits, allowing more information to be stored without using more energy.

But to think of quantum computing as just a more powerful conventional computer is off base. The types of problems these computers will solve will be fundamentally different. By using the properties of quantum interference, computer scientists are hoping to develop algorithms that would allow incorrect-solutions or redundant information to cancel each other out. These properties would allow quantum computers to perform incredibly complicated calculations while still delivering an interpretable result. These computers may prove an asset to modeling quantum processes themselves, a task conventional computers struggle with. On the to-do list are calculating molecular energies, modeling catalysis by enzymes, designing novel materials at the atomic level.

Overtime, programming languages evolved to allow developers to write code without constantly needing to know how computers would physically implement that code. However, learning how to use quantum hardware to perform what will be new types of computation is requiring physicists, computer scientists, and researchers to start from the beginning again. To foster collaboration, the Department of Energy has set up quantum computing testbeds, places where hardware designers and scientists can work together to simultaneously shape the computational revolution to come.

(Adrian Cho, Science)

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January 19, 2018 at 7:11 pm

FDA stem cell therapy crackdown: a stem-free clinic

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By: Belinda Hauser, Ph.D.

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

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

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

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

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

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January 17, 2018 at 11:43 am

Science Policy Around the Web – October 17, 2017

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By: Charles Wright, Ph.D.

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Source: Public Domain Pictures

Gene Therapy

FDA experts offer a unanimous endorsement for pioneering gene therapy for blindness

Gene therapy, an approach long hailed for its potential to cure intractable genetic diseases, finally has some successes in getting regulatory approval.  Recently, the U.S. Food and Drug Administration (FDA) approved the first-ever gene therapy treatment for a fatal brain disease.

Last week, another gene therapy approach—this time for a blinding disease that strikes in early-to-late childhood—also received endorsement by a 16-0 vote from the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The vote provides encouragement for patients suffering from Leber congenital amaurosis, type 2 (LCA2), which Voretigene neparvovec (Luxterna) from Spark Therapeutics aims to treat. However formal regulatory approval is still pending . Leber congenital amaurosis, although rare with an estimated prevalence of only 1:50,000 – 1:100,000, devastates vision at an early age. Newborns may show visual impairment immediately after birth, and most LCA patients lose most of their vision by the time they reach 20-30 years of age. Voretigene neparvovec aims to treat patients with mutations in the RPE65 gene, characteristic of LCA2, through gene therapy.

The recent string of successes in gene therapy comes years after the field struggled to rebuild itself after tragedy.  In 1999, the death of a participant in a trial to treat a rare metabolic disorder forced FDA to strengthen its oversight of gene therapy trials. With the first formal FDA approval for a gene therapy awarded just months ago, the recent approvals for other treatments since then suggests gene therapy may have reached a turning point.  In the future, gene therapy may be common for a variety of diseases.

If voretigene neparvovec receives FDA approval, it will be the first gene therapy treatment for a blinding disease.

(John Carroll, Science)

The Scientific Workforce

Why I’m pushing for a postdoc union

Postdoctoral fellows face a bevy of obstacles that threaten to derail even the most brilliant and committed young scientists from a research career.  Many postdocs unfortunately become intimately familiar with them in the lab; funding, publications, and tenure-track positions elude trainees and can keep postdocs from establishing themselves as independent researchers for years.  The harsh reality of the postdoc lifestyle often causes problems outside the lab as well.  Low pay, long working hours, and relationship strain all take their toll.

In response to these issues, some postdocs try to form unions to force academia to respond.  The subject of postdoc unions often pits researchers against university administrators, and the legal standing of postdoc unionization remains in flux.  Universities may claim unionization ultimately harms postdoc employment, as supervisors may write less than glowing recommendation letters for post-docs who missed work due to labor disputes. Nevertheless many postdocs feel unions may help provide a platform for receiving the benefits they need.

Brian Weitzner at the University of Washington (UW) in Seattle proposes collective bargaining rights for postdocs employed at UW campuses across the state.  In the past several months, while he and other postdocs prepare for a vote on whether to unionize, he talked with UW postdocs who share his concerns.  Fair pay, health insurance, and protection for victims of sexual harassment in the laboratory all motivate him to seek the establishment of a formal avenue with universities for resolving these and other issues.

UW postdocs are working with a local chapter of United Automobile Workers to navigate the steps needed to form a union. Their petition to vote is currently being reviewed by the Washington State Public Employment Relations Commission.

(Brian D. Weitzner, Science Careers)

Genomics

The rise and fall and rise again of 23andMe

Four years ago, the DNA-testing firm, 23andMe, teetered on the edge of oblivion.  Then, in 2015, 23andMe made the news again when it announced regulatory approval to sell a test for a rare genetic disease directly to the public.  Now, the company offers testing for 10 genetic diseases to consumers and continues to supply genomics data to collaborators to aid in drug development.

In 2007, 23andMe first gained prominence with its home-delivered kit that consumers could use to learn more about their genome.  After the company analyzed the data, costumers would receive information about their ancestry, predisposition to disease, and other miscellaneous facts such as whether they carried DNA variants that impacted their earwax consistency or urine smell after eating asparagus.  23andMe would also pool customers’ de-identified data together to analyze and sell to other pharmaceutical companies or research collaborators.

As 23andMe grew in popularity, critics became concerned that consumers were being overly impressed by advertisements indicating they could use their own genetic information to better inform personal health decisions even though many links between the DNA sequence variations reported by the test and disease remain dubious.  Alarmed by the possibility patients could make decisions about their health on information that had not been fully validated, FDA warned 23andMe it would need to provide evidence of the accuracy of the tests and ability of consumers to understand the results to continue marketing their kit as a health tool.  23andMe ignored the FDA’s warnings until the regulatory agency finally issued a cease-and-desist letter.

Since then, 23andMe decided to work with the FDA to approve the genetic tests included in its kit. The company can still only sell genetic tests directly to customers for a small number of diseases but is working with the FDA to expand its offerings to the general public.  In the meantime, the company still works with research collaborators to find new disease-relevant DNA variations, like those possibly involved in clinical depression.

Ultimately, 23andMe hopes its business model will lead to new therapies for disease.

(Erika Check Hayden, Nature)

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October 17, 2017 at 5:10 pm

Science Policy Around the Web – October 10, 2017

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By: Kseniya Golovnina, PhD.

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Gene Therapy

In a First, Gene Therapy Halts a Fatal Brain Disease

 The first historic gene therapy approval by the U.S. Food and Drug Administration in August 2017 opened a new era for the treatment of serious and life-threatening diseases. One month later Bluebird Bio announced the successful start of Lenti-D therapy in the clinical trial and gave a flutter of hope to cure cerebral adrenoleukodystrophy (CALD) also known as Lorenzo’s Oil.

Adrenoleukodystrophy (ALD), a rare disorder that affects one in 21,000 male births worldwide, is caused by mutations in the ABCD1 gene that lead to the subsequent accumulation of very long chain fatty acids in tissues, including the myelin of the central nervous system. The most severe form of ALD, known as cerebral ALD (CALD), involves the progressive destruction of the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. This leads to deafness, blindness, seizures, loss of muscle control and dementia, resulting in permanent disability or death. Symptoms of CALD progress rapidly if untreated and the only current treatment is stem cell transplantation.

Gene therapy is a technique for correcting defective genes responsible for disease development.  It utilizes viruses to deliver unmutated copies of the genes, such as the ABCD1 gene, to the cells of the patient’s body. First, blood from the patient is collected by apheresis, depleting immune response T cells and enriching progenitors of all blood cells (hematopoietic stem cells, HTS). The HTS cells are then infected with a virus carrying a functional copy of the gene, before returning the cells to the body.

Bluebird Bio is now pursuing Lenti-D therapy which uses lentiviruses to deliver a functional copy of the ABCD1 gene to patients with ALD. Results published in the New England Journal of Medicine, reported that 15 out of 17 patients (88%) were free from major functional disabilities two years after the hematopoietic stem-cell gene therapy. These results demonstrate the therapy’s efficacy over the 76% benchmark established by radiotherapy-free survival at 24 months. Bluebird’s Chief Medical Officer David Davidson expressed excitement about the patients’ progress. He announced that the first four patients treated in the expansion cohort are also doing well, as measured by their amount of the functional ABCD1 gene.

(Gina Kolata, The New York Times)

Drug pricing

FDA acts to encourage generic competition for complex drugs

What kind of feelings do you have when pharmaceutical companies announce their prices for upcoming exciting gene therapies and other innovative, life-changing bio pharmaceuticals? Positive news about development and success of first-of-their-kind drugs can be undermined by anxieties that patients will not be able to afford them. High drug prices can prevent accessibility of new therapies vital for many patients, and market analysts predict rapid inflation in healthcare spending over the next few years due to the aging US population.

The FDA is conscious of the stress expensive drugs put on both patients and the entire healthcare system. Under the leadership of current Commissioner, Dr. Scott Gottlieb, one of key goals of the the FDA is to bring more competition from generics to help drive prices down. On October 2, 2017 the agency prepared draft guidance specifically aimed at copycats of complex therapies and therefore trying to clear the way for generic drug makers to the market.

Gottlieb wrote in his blog post that the agency is looking for more efficient regulatory pathways with robust reviews and communication with pharmaceutical companies for abbreviated new drugs applications (ANDAs). He highlighted that “early and better meetings between FDA and sponsors can improve development timelines. The agency acknowledged that the complexity of the approval process may be discouraging to generic drug makers. The new guidance aims to clarify these complexities by outlining how genetic makers can prove “sameness” by showing that there is no difference in response to generic drug compared with the original. This is the second update for ANDAs process and Dr. Gottlieb assured that FDA will continue to progress in this reformation.

(Linda A. Johnson, The Associated Press)

 

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October 10, 2017 at 10:11 pm

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Science Policy Around the Web – September 29, 2017

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By: Allison Dennis, B.S.

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Hospital Budgets

Another health care deadline looms: Payments to safety net hospitals due to expire

On October 1, 2017, America’s healthcare safety net will face its first in a series of annual funding cuts to Disproportionate Share Hospitals payments scheduled by Obamacare. This safety net is held together by hospitals that shoulder the responsibility of serving the uninsured, Medicaid, and economically-vulnerable patients, often at a financial loss. The American Hospital Association reports that hospitals are only reimbursed 88 cents for every dollar they spend on Medicaid patients. Further insufficiencies arise when hospitals adhere to The Emergency Medical and Treatment Labor Act of 1986, which mandates the treatment of patients seeking emergency services regardless of their ability to pay. Highly profitable hospitals that attend communities where these patients represent a marginal burden can absorb the cost through lost profits. However, hospitals serving communities where these patients pose an undue burden are supported by subsidies from federally-funded, state-matched, Disproportionate Share Hospitals payments. The scheduled cut for this year would reduce the $21 billion allocated state-by-state in fiscal year 2017 by a total of $3.6 billion, $2 billion from federal contributions combined with $1.6 billion from state contributions.

Obamacare expanded Medicaid to cover individuals up to 138% of the Federal Poverty Level, set at $33,948 for a four-person household and $16,642 for an individual, reducing national levels of uncompensated care. Further, the law provided opportunities for individuals to purchase insurance coverage through HealthCare.gov, while implementing penalties for those who chose to forgo coverage. Lawmakers included a schedule for reducing Disproportionate Share Hospital Payments on the assumption that the proposed changes in the healthcare system would be sufficient to reduce the financial burden on safety net hospitals, lessening the need for federal and state assistance.

The rate of uninsured Americans did fall to 8.8% in 2016, compared with 16.3% in 2010. However the extent to which this change has improved revenues for safety net hospitals remains unclear. Further occluding the readiness of hospitals to cope with the budget cut are the unique challenges each may face. The agreement to extend discussions of the 2018 fiscal year budget past the October 1 deadline may give lawmakers the last minute chance to forgo the cuts for yet another year.

(Max Blau, STATnews)

Pharmaceutical Regulation

You’ve heard about precision medicine. Now get ready for precision drug ads

In 2016, $5.7 billion, about $17.5 per person, was spent by pharmaceutical companies on traditional advertising. But how many of those ads were seen by the people who need them? Their recent interest in the online giants Facebook and Pandora, suggests that pharmaceutical companies are looking to enter the new age of advertising, targeted ads. The Health Insurance Portability and Accountability Act (HIPAA) of 1996 prevents insurance companies and healthcare providers from sharing an individual’s health information, especially when it may be used for marketing purposes. However, being privy to a person’s private interactions with their electronic devices may be far more revealing about a user’s health than conversations with their doctor.

The FDA has been tasked with regulating pharmaceutical advertising on the internet. However, applying the rules surrounding pharmaceutical adverting in the age of 140 character limits and browser history mining will take some reimagining. So far, the FDA’s approach to limiting inappropriate advertising has been to call it when they see it. In 2015, the FDA famously issued a warning letter regarding an Instagram post made by Kim Kardashian, seemingly promoting a prescription-only anti-nausea pill. Nevertheless, in 2017, Pfizer successfully experimented using geographical information to target ads to online consumers without any FDA upset.

This comes as a growing field of research is investigating the observation that the words used to describe a particular disease may influence the treatment options a patient gravitates towards. Parents who were told their child had “pinkeye” instead of an “eye infection” were more likely to give their child a course of antibiotics, even when doctors stated the treatment was likely ineffective. Altering the language appearing in targeted ads on platforms like facebook may further provide means for social experimentation, adding another layer of concern for the FDA.

(Rebecca Robbins, STATnews)

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September 29, 2017 at 7:40 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