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Science Policy Around the Web December 10th, 2019

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By Kellsye Fabian MS, PhD

Image by allinonemovie from Pixabay 

Two New Drugs Help Relieve Sickle-Cell Disease. But Who Will Pay?

The FDA recently approved two new treatments for sickle-cell disease — Adakveo and Oxbryta. Sickle-cell disease is caused by a gene mutation that results in abnormal hemoglobin and sickle-shaped red blood cells. These cells clump together, restricting the flow in blood vessels and limiting oxygen delivery to the body’s tissues. This leads to severe pain and organ damage. Adakveo, made by Novartis, can prevent occurrence of bouts of severe pain caused by misshapen blood cells getting stuck in blood vessels. Oxbryta, made by Global Blood Therapeutics, can prevent disease-induced anemia that can result in permanent damage to the brain and other organs. For a disease that has been historically overlooked, the availability of these new cutting-edge medicines may represent new hope for people with sickle-cell disease. However, the new drugs might not be accessible to all ~100,000 Americans with the disease. Each treatment must be taken for life and is priced at $100,000 a year, which is double the median family income in the US.

Novartis and Global Blood Therapeutics have been in negotiation with insurance providers about covering the new drugs. Both companies are optimistic that most insurers will pay for the new treatments. The companies argue that without these drugs, the management of sickle-cell disease is expensive. Treating sickle-cell disease complications such as pain, stroke, and organ damage costs about $10,000 a year for children and about $30,000 for adults. Not included in that amount is the economic burden on adults who cannot work due to debilitating disabilities associated with sickle-cell disease and on family members who often wind up as caregivers.

Despite this, some experts and patient advocacy groups question the drug makers’ justification for the treatments’ hefty price tags. Actual development costs and taxpayer support must be considered when setting the price for these treatments. 

More affordable options are available for sickle-cell disease patients. Hydroxyurea, which was approved in 1998 and can reduce the frequency of pain crises and stroke, costs about $1,000 a year. While some patients on public insurance programs have $0 co-pays for Hydroxyurea, only 30% of sickle-cell patients take it. Therefore, insurers may possibly require sickle-cell disease patients to be treated with hydroxyurea before moving on to the more expensive Adakveo or Oxbryta.

Medicaid covers about 50% of sickle-cell disease patients while Medicare covers another 15%. It remains unclear how these programs can afford to pay for all who might need the new drugs. 

(Gina Kolota, New York Times

FDA warns Liveyon about selling unapproved stem-cell products that pose a risk to consumers

The FDA has issued a warning to Liveyon Labs and Liveyon LLC of Yorba Linda, California for making and selling unapproved umbilical cord blood products. The agency also issued a warning for significant deviations from safety practices that create serious risks for patients that receive the stem cell therapy.

In 2018, Liveyon LLC distributed contaminated non-FDA-approved umbilical cord blood products processed by the San Diego-based company, Genetech, Inc. The products were linked to the hospitalization of twelve patients who received the injections or infusions. Liveyon conducted a voluntary recall and began making its own products called Pure and Pure Pro through Liveyon Labs. These products are marketed mainly as a treatment for patients with back, knee, and other joint problems. 

An FDA inspection conducted in May revealed that Liveyon Labs and Liveyon LLC were manufacturing and distributing products that are considered drugs although they did not have the approval to do so. An approved biologics license application is needed to lawfully market a drug and an approved investigational new drug application (IND) is required for a drug to be used in humans during the development stage. No such licenses or INDs exist for Pure and Pure Pro. The inspection also documented that the companies did not meet safety standards, including failing to screen donors’ relevant medical records for communicable disease, inadequate aseptic practices to prevent contamination, and deficient environmental monitoring, such as failing to establish a system for cleaning and disinfection the processing room and equipment. According to the FDA, Liveyon took some corrective actions after the inspection. However, Liveyon has yet to provide “proof of updated policies and procedures” and it did not address its lack of required approvals. 

The FDA requested a response from the companies within 15 working days that details how the issues will be corrected. Failure to correct the problems could lead to seizure, injunction or prosecution. Liveyon said it would cooperate with the FDA. 

(Laurie McGinley, The Washington Post

Written by sciencepolicyforall

December 10, 2019 at 10:57 am

Science Policy Around the Web December 6th, 2019

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By Hannah King, PhD

Image by Steve Buissinne from Pixabay 

200,000 Uninsured Americans to Get Free H.I.V.-Prevention Drugs

World AIDS Day, held annually on December 1st, has led to a flurry of AIDS-related announcements this week.

The Trump administration has released an announcement outlining how it plans to distribute HIV prevention drugs donated by the pharmaceutical company Gilead, which manufactures the drugs. These drugs will be available to 200,000 uninsured Americans who have a prescription and recent evidence of their HIV-negative status.

While other programs exist in the US to provide these drugs free of charge (including those run by cities in high-incidence areas and a program from Gilead itself) this announcement marks the first time the government has provided such HIV-prevention medicine free to individuals not enrolled in a federal health program. With 37,500 new HIV infections per year in the US, and 1.2 million Americans estimated as being at high risk for HIV-acquisition, widespread distribution of this preventative treatment is an important public health initiative. 

Access to HIV prevention and treatment medication is also a worldwide issue. In further encouraging news, an Indian drug manufacturer has announced that it will make a pediatric formulation of HIV medication, that is strawberry flavored and the size of sugar granules! The current medication is either formulated as hard tablets, or requires refrigeration, reducing either the tolerability to children or accessibility of the product. As 160,000 children are born with HIV each year, but only approximately half receive treatment, this new formulation will hopefully reduce the AIDS-related morbidity and mortality in this vulnerable population.   

(Donald G. McNeil Jr., New York Times

China’s CRISPR babies: Read exclusive excerpts from the unseen original research

Further details of the research purporting to use CRISPR to create gene-edited babies have emerged, reinforcing the “serious, unresolved safety concerns” associated with this human research. 

MIT Technology Review has released excerpts from an unpublished manuscript outlining a study by the Chinese researcher He Jiankui which describes the creation of the first gene-edited human babies. The mutation introduced into the embryos is a deletion in the gene expressing a protein called CCR5. A deletion in this CCR5 protein occurs naturally in some individuals and renders them resistant to infection with HIV – the stated rationale for this experiment. However, the manuscript shows the deletion that was introduced into the genomes of these babies is similar, but not identical to the naturally occurring deletion, and no attempt is made by the authors to validate its ability to confer HIV resistance.

Furthermore, despite the manuscript describing the experiments a “success”, the data shows the CCR5 deletion is absent in one chromosome on one of the babies, meaning she carries one copy of the functional CCR5 gene, and is still susceptible to HIV infection. Data in the manuscript, showing DNA sequencing of cells from the babies following their birth also indicates that not all cells in these babies share the same genetic code, suggesting that not all cells carry the CCR5 deletion, or that other “off-target” effects with unknown health implications may be present.

This newly available data, in addition to the many ethical concerns previously raised, further demonstrate this experiment is not the “success” nor the path forward to “control the HIV epidemic” that He Jiankui claims it to be.

(Antonio Regalado, MIT Technology Review)

Written by sciencepolicyforall

December 6, 2019 at 1:51 pm

Science Policy Around the Web November 29th, 2019

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By Maria Disotuar, PhD

Source: Pixneo

To Drive Down Insulin Prices, W.H.O. Will Certify Generic Versions

Without insulin, a person with type 1 diabetes cannot survive, and the cost and accessibility to insulin continues to be a problem for individuals suffering from this incurable autoimmune disease. Diabetes mellitus is a chronic metabolic disease characterized by high blood glucose levels. There are two types of diabetes, Type 1 diabetes results from the loss of pancreatic β-cell function, resulting in an inability to produce insulin, a peptide-based hormone. On the other hand, Type 2 diabetes patients are resistant to insulin. Those suffering from Type 1 diabetes require daily insulin therapy to stay alive, and patients with type 2 diabetes require insulin therapy to maintain a healthy lifestyle. Currently, more than 400 million people worldwide have diabetes and this number is expected to increase in the coming years. The main problem being that there are no generic forms of insulin and the price for current insulin analogs has gone from approximately $20 per vial to $250 per vial depending on the type of insulin. This price increase over the past 20 years has made insulin unaffordable for many individuals particularly for younger generations of Americans struggling to pay student loans. For these individuals, seeing the price of insulin jump from $4.34 to $12. 92 per milliliter has meant rationing the lifesaving drug to the bare minimum – a deadly decision for some.

As a response to the growing demand for insulin and skyrocketing prices, the World Health Organization (WHO) has proposed a two year prequalification pilot project, which will allow pharmaceutical companies to produce generic insulin to be evaluated by WHO for efficacy and affordability. These types of pilot projects have been previously deployed to improve the accessibility of life saving drugs for malaria, HIV, and tuberculosis. These efforts have led to an increase in production and market competition leading to reduced costs for individuals.

Currently, the major producers of insulin, Eli Lilly, Novo Nordisk, and Sanofi have welcomed the prequalification program, vowing to be a part of the solution not the problem. According to WHO, companies in several countries, including China and India, have already expressed interest in the pilot project. This shift in insulin production would allow companies producing insulin domestically to enter the global market. As WHO-certified suppliers, these new competitors could dramatically drive down the price of insulin and improve accessibility on a global scale. Despite this positive global outlook, there are still some hurdles to cross for Americans to obtain these generic insulin products. The main one being that the pharmaceutical market is regulated by the FDA and the review process can be expensive for smaller companies. Nonetheless, Americans are fighting back to reduce the cost of insulin and other life savingdrugs, prompting lawmakers, presidential candidates, and the President to prioritize reduced drug prices for Americans. These mounting pressures will hopefully lead to a faster solution for this life or death situation.

(Donald G. McNeil Jr., The New York Times)

Will Microneedle Patches Be the Future of Birth Control?

In 2018, the The Lancet reported that between 2010 and 2014 44% of all pregnancies in the world were unplanned. Despite medical advances in sexual and reproductive health, new contraceptive methods are needed to expand accessibility and improve reliability for women. In the United States, the establishment of the Affordable Care Act (ACA) and health policies such as the Federal Contraceptive Coverage Guarantee, which requires private health plans to include coverage for contraceptives and sexual health services, has improved family planning for women of reproductive age. Despite the social and economic benefits of improved family planning and enhanced accessibility, conservatives continue to challenge these beneficial health policies. Unfavorable changes to these policies could result in major barriers for women to access some of the most effective, yet pricier forms of contraceptives such as intrauterine devices (IUDs) and implants. Studies show these long-acting forms of birth control are up to 20 times more effective in preventing unintended pregnancies than shorter-acting methods such as the pill or ring. Thus, new long-term contraceptives with reduced cost barriers would be essential in reducing unintended pregnancies and enhancing economic benefits on a global scale.

To address this issue, researchers at the Georgia Institute of Technology and University of Michigan in partnership with Family Health International (FHI) – a non profit human development organization, have developed a long-acting contraceptive administered by a patch containing biodegradable microneedles. The patch is placed on the surface of the skin and the microneedles painlessly come into contact withinterstitial fluid resulting in the formation of carbon dioxide bubbles, which allow the microneedles to detach from the patch within 1 minute of application. The needles themselves do not introduce a new contraceptive hormone, rather they provide levonorgestrel (LNG), which is regularly used in IUDs and has been deemed as safe and efficacious. After dissociation from the patch the needles slowly release LNG into the bloodstream. 

Thus far, the pharmacokinetics of the patches has been tested on rats and a placebo version has been tested in humans to test the separation process between the patch and the needles. The in vivo animal studies indicate the patch is able to maintain LNG concentrations at acceptable levels for more than one month and the placebo patch was well tolerated among study participants with only 10% reporting transient pain or redness at the site of patch application. Lastly, the researchers analyzed conceptions and acceptability of this new contraceptive method among American, Indian, and Nigerian women compared to oral contraceptives and monthly contraceptive injections administered by a physician. The results indicate women overwhelmingly preferred the microneedle patch method over the daily pill (90%) or monthly injections (100%). The researchers expect the patch to be simple to mass produce and a low-cost contraceptive option, which will reduce cost barriers and improve accessibility for women. Although the results of the study are promising, additional studies will have to be completed to address some of its limitations. Future studies will have to increase the number of animals used in the study and the number of human participants. Additionally, the release profile for LNG will likely need to be extended beyond 1-month to truly address the need for new long-acting forms of contraceptives. Finally, clinical trials will have to be completed to test the efficacy and general reliability of this method at reducing unintended pregnancies. If the microneedle patch is approved, it would be the first self-administered long-term birth control to enter the market, which could ultimately lead to enhanced accessibility for women with limited access to health care.

(Claire Bugos, Smisothian) 

Science Policy Around the Web November 15th, 2019

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By Mohor Sengupta PhD

Image by David Mark from Pixabay

Rollout of Johnson & Johnson Ebola vaccine begins in Congo

To the African countries reeling with the second deadliest Ebola outbreak in history, two back-to-back launches of new vaccines may be a beacon of hope. On Tuesday this week, Merck’s vaccine Ervebo was pre-qualified by the WHO, meaning that it was declared safe for use. This occurred merely 48 hours after the European Commission granted conditional marketing authorization for the vaccine. The speed of this decision-making sheds light on the gravity of the situation at hand. 

The current outbreak in the Democratic Republic of Congo has killed more than 2,200 people. The previous Ebola outbreak, and the deadliest in living memory, rocked West Africa in 2013-16, claiming 11,300 lives. 

In addition to Ervebo, a new vaccine produced by Johnson & Johnson was approved yesterday. It passed several clinical trials, however it will now be tested for the first time in a real world setting in the village of Goma, on the Rwandan border with DRC. It will be administered to 50,000 people. 

The new vaccine by Johnson & Johnson is aimed at complementing Ervebo. While the later requires a single shot, the Johnson & Johnson vaccine will require two shots spaced at 8 weeks. Ervebo is being used as “ring-vaccination”, a strategy in which close contacts of Ebola-infected individuals will be vaccinated.  

(Reporting by Fiston Mahamba; Writing by Hereward Holland; Editing by Anna Pujol-Mazzini and Mark PotterReuters)

Written by sciencepolicyforall

November 15, 2019 at 4:35 pm

Science Policy Around the Web November 12th, 2019

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By Allison Dennis PhD

Image by Edward Lich from Pixabay 

Your DNA Profile is Private? A Florida Judge Just Said Otherwise

In a game changing decision for law enforcement, a U.S. judge approved a warrant that allowed a police detective to search the DNA records of all the users included in the GEDmatch database. This comes after GEDmatch changed its policies in May to only grant law enforcement access to the users who had chosen to opt-in to such uses. However, the warrant overruled the choice of 1.1 million users out of the 1.3 million included in the database who had not opted in. The value of these databases to law enforcement stems from their use in identifying a DNA profile through the combination of rich genealogical data with DNA profiles of distant relatives. As more potential relatives are included in the search, the likelihood a match is found increases. Theoretically there is a 60% chance that someone of European ancestry living in the U.S. will be found to have a relative in a database of 1.3 million profiles.

The U.S. Department of Justice has enacted a policy to limit the use of forensic genetic genealogy to solving violent crimes and identifying human remains. Further, under this policy, law enforcement must demonstrate that traditional crime solving methods have been exhausted before turning to DNA databases for clues. 

Many feel the recent judge’s decision may be a critical step towards law enforcement potentially gaining access to the much more valuable Ancestry.com and 23andMe databases, which each contain the profiles of 15 million and 10 million users. Both companies maintain a strict posture in resisting access to their customers data for law enforcement purposes. However, it may take either company formally challenging a warrant or a defendant claiming a violation of their fourth amendment rights to draw a clear line between genetic privacy and forensic genetic genealogy. 

(Kashmir Hill and Heather MurphyThe New York Times)

Google’s ‘Project Nightingale’ Gathers Personal Health Data on Millions of Americans

For more than a year, the second-largest health system in the U.S., Ascension, has been sharing the health records of millions of their patients in secret with Google. Known as project Nightingale, the collaboration was established to allow Google to use Ascension’s vast collection of medical records to design new software while helping Ascension improve patient care and generate revenue. This type of data sharing is allowed by the Health Insurance Portability and Accountability Act (HIPAA) of 1996. Under HIPAA provisions, patients and doctors do not need to be informed of such arrangements if the disclosure of data to a third-party is “only to help the covered entity carry out its health care functions.”

Earlier in November, Google announced its acquisition the fitness tracking company Fitbit. Many have speculated that Google’s underlying motivation is to gain access to data that could be analyzed to provide advertisers with more educated guesses about a potential customer’s health status. However, Google has stated that “Fitbit health and wellness data will not be used for Google ads.” The sharing of Ascension patients’ data and identity with advertisers would be strictly prohibited by HIPAA. Other Google ventures including their partnership with the drugmaker Sanofi to develop a healthcare innovation lab, announced in June of 2019, reflect their interest in developing personalized approaches to medical treatment. While it is clear that the medical community sees long-term potential in capitalizing on Google’s expertise in artificial intelligence and secure storage of data in the cloud, it remains to be seen the direct benefit these partnerships will have on improving individual patient’s health.

(Rob Copeland, The Wall Street Journal)

Written by sciencepolicyforall

November 12, 2019 at 4:41 pm

Science Policy Around the Web November 8th, 2019

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By Mary Weston PhD

Image by PublicDomainPictures from Pixabay 

Scrubbing Your House Of Bacteria Could Clear The Way For Fungus

While it may seem that a ‘cleaner’ environment would mean fewer germs, a new study in Nature Microbiology suggests otherwise. Researchers comparing the diversity of microorganisms in increasingly urbanized environments found that fungal diversity was actually higher in urban homes. They examined four types of housing, ranging from thatched huts in a Peruvian rainforest community to city apartments in Manaus, the capital of the Brazilian state of Amazonas. 

One of the study’s coauthors, Dr. Laura-Isobel McCall, suggests several reasons for why this may occur. Many antibacterial cleaning products specifically target bacteria, which could free space for fungi and other microorganisms to grow. Fungi have thicker cell walls, which may make them more difficult to destroy. Also, urban homes tend to block light and trap CO2, which could be creating a favorable setting for fungi to grow. 

In general, these results indicate that urbanization has large effects on the human skin microbiota, as well as the surrounding chemical and microbial environment. This lack of bacterial diversity could be problematic, as some of them may be helpful to humans. The researchers also discovered many more synthetic chemicals in city apartments, which can originate from cleaning products, building materials, medications, and personal care products (such as shampoo and deodorant). The effects of increased exposure to synthetic chemicals are not well known.

While this study was conducted in areas of Brazil and Peru, the findings may have a broader significance. Justin Sonnenber, a microbiologist at Stanford University, asserts that “My guess is that this gradient they’ve established for these fungal communities is largely representative of what’s happening all over the world”.  

(Pien Huang, NPR)

Trump administration sues HIV prevention drug maker for patent infringement

On November 6, the Trump administration sued Gilead Sciences for patent infringement over Truvada and Descovy, drugs that are crucial in preventing the spread of HIV. The suit asserts that some of the relevant patents are owned by the government because scientists from the Centers for Disease Control and Prevention (CDC) developed the breakthrough drugs. 

The Department of Health and Human Services (HHS) argues that Gilead has repeatedly refused to obtain licenses for the use of 4 CDC patents, but are making billions of dollars from the drugs. HHS secretary Alexander Azar says that while the government recognizes Gilead’s role in selling the anti-HIV medications to patients, the company “must respect the US patent system, the groundbreaking work by CDC researchers, and the substantial taxpayer contributions to the development of these drugs.”  The lawsuit argues that “Gilead’s conduct was malicious, wanton, deliberate, consciously wrongful, flagrant, and in bad faith.”

Gilead disagrees with these allegations, arguing that the government patents are invalid and the work performed by the CDC was “obvious and proposed by others.” Gilead asserts that “The fact remains that Gilead invented Truvada and funded the clinical trials that led to its 2004 FDA approval for use in combination with other antiretroviral agents to treat HIV.”

Truvada and Descovy are PrEP (pre-exposure prophylaxis) drugs, an HIV prevention method for people who are at a high risk of acquiring HIV. When taken daily, these medications reduce the risk of getting HIV from sex by 99% and 74% for those who inject drugs. In his 2019 state of the Union address, President Trump established a goal of ending the spread of HIV in America by 2030. Lowering the price of PrEP, which can cost around $21,000/year, would significantly advance those efforts and some hope that the Gilead lawsuit itself may result in a price reduction.

(Peter Sullivan, The Hill)

Written by sciencepolicyforall

November 8, 2019 at 10:57 am

Science Policy Around the Web November 5th, 2019

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By Silvia Preite

Source: Flickr

Natural measles infection impairs the preexisting immunity to other pathogens

Measles is one of the most contagious human infectious disease, causing over 100,000 deaths worldwide every year. Despite the availability of a safe and effective vaccine, the number of new measles cases is growing fast, with a 30% global increase between 2017 and 2018. Misguided vaccine safety concerns are leading to under-vaccination that, together with extensive international travels of people around the world, contributes to this sharp increase in measles cases. As October 2019, there are more than 1250 confirmed cases of measles in the U.S. alone – the highest number reported since 1992. 

The harmful consequences of a Measles infection go beyond the infection with the virus itself. Epidemiological studies have associated measles outbreaks with increased morbidity and mortality to secondary unrelated reasons. Two recent studies published in Science Immunology and Science shed lights on this phenomenon: the authors showed that measles suppress the body’s immune system, reducing the ability to respond to other infections. 

Scientists analyzed a cohort of children from an Orthodox Protestant community in the Netherlands that have been not vaccinated by their parents for religious reasons. A total of seventy-seven of these children partook in the study before and after a measles outbreak in 2013. The blood of children pre-measles contraction contained antibodies (proteins produced by immune cells called B cells) that protect against common pathogens. However, after a natural measles infection, between 20 and 70% of these antibodies were lost. The immune system becomes “ignorant” again to viruses that it had encountered in the past. This “amnesia” of the immune system increases the risk of infections and slows down the ability of our immune system to fight pathogens such as influenza. 

Strikingly, children receiving vaccination against measles did not display such suppression of acquired immunity. These data further support the importance of widespread vaccination strategies to protect against measles but also to maintain a proper herd immunity to other pathogens.

(Petrova et al., Science Immunology, 2019 AND Mina et al., Science, 2019)

Written by sciencepolicyforall

November 5, 2019 at 4:27 pm