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Science Policy Around the Web November 26th, 2019

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By: Andrew H. Beaven, PhD

Source: CDC

How Best To Use The Few New Drugs To Treat Antibiotic-Resistant Germs

Bacteria have existed for 3.5–4 billion years, and their survival demonstrates remarkable environmental plasticity. One consequence of their plasticity is that bacteria are able to rapidly become resistant to antibacterial drugs (drugs meant to help humans and animals stave off infection).

Combatting antibiotic drug resistant bacteria (so-called “superbugs”) has been pinpointed as a major, modern global health concern. A new Centers for Disease Control and Prevention (CDC) report published November 14, 2019 estimates that more than 2.8 million treatment-resistant infections and 35,000 annual deaths occur in the U.S. alone. Notably, the development of new antibiotics has lagged, therefore, scientists are recommending new methods to use old drugs. These include: limiting doses for healthy people and allow the body to do its work; flooding the body with multiple drug types at once; or sequentially changing drug types every 12 to 24 hours.

Given all we know about this global health concern, why is the market not being flooded with new antibiotics? Largely, because it is not lucrative for pharmaceutical companies to chase a moving target. Additionally, antibiotics are only used for short-term ailments (as opposed to those used for chronic illness) and many antibiotics remain unused in an effort to minimize new drug resistance. To help promote new antibiotic research, U.S. Senators Bob Casey (D-PA) and Johnny Isakson (R–GA) introduced the Developing an Innovative Strategy for Antimicrobial Resistant Microorganisms Act of 2019 (DISARM Act of 2019; S.1712) to the Senate on June 4, 2019. The goal of the act is to strengthen American antimicrobial research and improve the development pipeline. While the act was called “essential and timely” by the President of the Infectious Diseases Society of America, it has not passed the Senate at time of publication.

(Richard Harris, NPR)

As SpaceX Launches 60 Starlink Satellites, Scientists See Threat to ‘Astronomy Itself’

On November 11, 2019, the private American aerospace company SpaceX, founded by Elon Musk in 2002, launched its second Starlink satellite payload rocket into outer space. Starlink is a prodigious project that aims to provide “high speed internet access across the globe,” specifically allowing internet access to “locations where access has been unreliable, expensive, or completely unavailable.” The Starlink webpage states that they will provide near-global internet coverage by 2021 through their satellite constellation (a network in which satellites work together to provide continuous coverage).

Even with the relatively few Starlink satellites currently in place, astronomers have already noted significant impact on their work. The primary point of concern is that the satellites are very bright, and astronomers say that even if the satellites are darkened, they will have a profound effect on Earth-based astronomy. Additionally, astronomers worry that Starlink will pollute radio wavelengths used to probe deep space and permanently pollute low-orbit space with “space junk.” SpaceX says they are attempting to minimize the effects Starlink has on the scientific community and that the project is moving ahead. Indeed, Mr. Musk has requested the Federal Communications Commission to allow 30,000 more satellites than 12,000 that were already approved. If successful, SpaceX would have eight times more satellites in low-Earth orbit than currently in orbit.

Other companies, such as, Amazon, Telesat, and OneWeb, are following close behind launching similar satellite constellations. Using outer space for private gain yields an important, unanswered question – who can profit from outer space? Megan Donahue, president of the American Astronomical Society acknowledges that “international space law is pretty wide open,” and that it for now the public must trust corporate good will. Currently, a set of United Nations treaties and principles as well as resolutions have laid out guidelines on the peaceful usage of space, but few exact rules are in place.

(Shannon Hall, The New York Times

Written by sciencepolicyforall

November 26, 2019 at 3:14 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

Should Vaccines be Mandatory?

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By: Juan I. Moliva, Ph.D.

Image by Angelo Esslinger from Pixabay 

The anti-vaccination movement has been gaining traction across the United States (U.S.) and the world. According to the Centers for Disease Control and Prevention (CDC), the number of measles cases in the first five months of 2019 surpassed the total number of cases per year for the past 25 years, with the number of children unvaccinated for preventable diseases quadrupling since 2001. The CDC also found an increase in the number of vaccine exemptions for kindergarteners across the U.S. While the numbers vary across the country mostly due to laws set in place by each state, in Mississippi, one of three states that do not grant exemptions due to religious or philosophical reasons, the rate of kindergartners with an exemption to one or more required vaccine was 0.1%. Compare that to Oregon, a state that grants exemptions to vaccines due to religious or philosophical reasons, where the rate of vaccine exemptions for kindergarteners is at 7.6%. As the number of individuals choosing not to get vaccinated or choosing not to vaccinate their children continues to increase, the debate as to whether vaccines should be mandatory has taken front stage.

Simply stated, a vaccine is a weakened or killed version of a pathogen that is delivered to the body with the sole goal of stimulating an immune response to protect you against the disease you are being vaccinated against. How is this accomplished? Your immune system has a subset of cells with “memory” that remember the pathogen and if you are later exposed to the pathogen these “memory” cells will eliminate the pathogen before it can make you sick. In the U.S. and most of the world, the general scientific consensus on vaccines is that they are safe and critical for maintaining healthy communities. The U.S. Senate Committee on Health, Education, Labor, and Pensions (HELP) strongly defended the case for vaccines. Chairman Lamar Alexander (R-TN) said, “The science is sound: Vaccines save lives – the lives of those who receive vaccines and the lives of those who are too young or vulnerable to be immunized.” Like any medication or medical procedure, vaccines do not come without risk, but the advantages vastly outnumber any disadvantages.

Not only do vaccines protect your body from dangerous pathogens, they also protect the community via a concept known as herd immunity. When a high enough percentage of the population is immune, pathogens have a harder time spreading. We rely on this principle to protect those who cannot receive a vaccine. However, vaccine opponents continue to argue that mandating vaccines is a slippery slope towards losing body autonomy. I have spent the last eight years studying vaccines, from basic research to pre-clinical testing through clinical trials and licensing. I have recently come to embrace the belief that the sole exemption for a vaccine that has been proven to be effective is a medical one – the case where you cannot receive a vaccine because your body responds negatively to the vaccine; this includes a weakened immune system that can be caused by chemotherapy or radiotherapy, a congenital condition that leads to an impaired immune system, or a history of serious adverse events related to vaccination such as an allergic reaction to one or more components in the vaccine.

The case against anti-vaxxers surrounds the choices they make on behalf of their children and how those choices affect their children and those in the community. If parents started claiming religious or philosophical exemptions against child safety seats or against buckling their children while driving, would we be arguing in their favor? Seatbelts and safety seats have been proven to save lives by preventing ejection from the vehicle after impact. Vaccines have been medically proven to save lives by preventing death or disability from infectious pathogens after exposure. While we expect not to be involved in a car accident, we rely on seatbelts in case we do. Vaccines work the same way; we do not want to be exposed to deadly pathogens but being vaccinated against these pathogens helps ensure we do not get sick if we do. Childrenare incapable of deciding if they want to be vaccinated as some vaccine regimens are initiated within the first year of life, thus their welfare is entrusted upon their parents. 

In the U.S. we have laws about providing proper nutrition, schooling, clothing, cleanliness, shelter, and many more that protect children’s welfare. Providing vaccines on time should be included as a basic necessity and be treated no different than other child welfare offenses. If an unvaccinated child contracts a preventable disease and is significantly affected to the point of disability or even death, the parent(s) should be directly responsible. A guardian refusing to vaccinate their medically eligible child should be treated like what it is: child endangerment. Take for example the recent case of a six-year-old in Oregon that nearly died of tetanus after sustaining a cut while playing outdoors. The child’s parents had refused to vaccinate the child with DTaP, a safe vaccine intended to protect against tetanus. The boy was held in an intensive care unit for 47 days, undergoing multiple intravenous infusions to alleviate the high fever, hypertension, and tachycardia caused by the infection. All at the cost of over $800,000 for a disease that could have been easily prevented with vaccination. Despite the episode, the boy’s parents continue to refuse the vaccine.

Although bipartisan support exists for vaccination at the federal level, it is unlikely federal law eliminating philosophical and/or religious exemptions would come to pass as public health policy is traditionally left to the states. State legislatures are beginning to introduce laws that will help limit the increase in numbers of preventable disease cases due to lack of vaccination. New York state recently passed legislation forbidding religious and non-medical exceptions to vaccines. After signing the bill, Governor Cuomo (D-NY) said, “While I understand and respect freedom of religion, our first job is to protect the public health.” Washington state, plagued by surging number of measles cases, also recently banned personal and philosophical exemptions to the MMR vaccine, which protects against measles, mumps, and rubella. Merely going to a public park or a grocery store can place unvaccinated children or immunocompromised individuals at risk of contracting a preventable disease. Such was the case of a six-year old boy with leukemia that died of measles after being exposed by his intentionally unvaccinated siblings. Senator Bill Cassidy (R-LA), a former physician, stressed: “If you are such a believer in liberty that you do not wish to be vaccinated, there should be a consequence and that should be that you cannot infect other people.” While I agree that autonomy over one’s body is important, the government has the right to require a reasonable level of vaccination for all citizens as a matter of public health. 

In the U.S. where freedom and personal liberty are valued above all else, mandatory vaccination would have a lengthy and challenging uphill battle. Additionally, data suggests that mandatory vaccination does not necessarily increase vaccination coverage. Instead, the U.S. could increase vaccinations via financial incentives similar to the system in place in Australia whereby parents receive monetary compensation when their children meet vaccination requirements. However, the most successful approach might be akin to the one the CDC implemented over sixty years ago to encourage stricter vaccination requirements by the states. By providing educational support, the CDC engaged parents, policymakers, and state and local health officials at every level to increase vaccination coverage. Education is one of the most effective ways to counter vaccination noncompliance. Specifically, educating people on the alternatives to vaccines – the morbidity and the mortality associated with lack of vaccination – was shown to be the most effective strategy. Educating why vaccines are so crucial is the most powerful weapon to combat vaccine non-compliance. 

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Written by sciencepolicyforall

August 14, 2019 at 10:00 am

Posted in Essays

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Disparity in the Global Supply and Demand for Vaccines Against Rotavirus

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By: Lawrence Wang

Image by Arek Socha from Pixabay 

Vaccines are extremely important for individual and global health. These life-saving biologics are especially important for children in developing countries, who suffer disproportionately from vaccine-preventable infectious diseases. One of these infectious diseases is rotavirus, which causes severe diarrhea and kills around 200,000 young children and babies per year1. Effective vaccines against rotavirus are responsible for saving countless lives and reducing suffering around the world.

In November 2018, the major pharmaceutical company Merck canceled a long-term commitment it made with GAVI (The Global Alliance for Vaccines and Immunization) to provide its rotavirus vaccine, RotaTeq, to four countries in West Africa (Burkina Faso, Cote d’Ivoire, Mali, and Sao Tome) for $3.50 a dose. Simultaneously, Merck increased RotaTeq shipments to China where a course sells for $40, nearly 12 times more than in West Africa2. Merck reported that the reason for this termination was “supply constraints” and an “unprecedented increase in global demand” for Rotateq3. Vaccines that were earmarked for West Africa were instead diverted to China, where they would protect millions of Chinese children while leaving millions of West African children vulnerable.

This situation exemplifies the disparity in the global supply of, and demand for, vaccines. Many vaccines are produced by large pharmaceutical companies like Merck that are based in wealthy nations (e.g., Merck’s headquarters are in New Jersey, USA). In general, developed countries have more agency in purchasing and providing vaccines for their citizens at higher prices. Developing countries, on the other hand, usually lack the money to pay for vaccines and are consequently reliant on free or subsidized vaccines. Profit-driven companies such as Merck are thus incentivized to sell their limited supply of vaccines to countries that can afford to pay more.

GAVI and UNICEF (United Nations Children’s Fund) are organizations that work to address disparities in vaccine access between industrialized and developing countries by buying and delivering vaccines to poor countries around the world4,5. However, they still rely on pharmaceutical companies to produce the vaccines that they distribute. When Merck notified GAVI and UNICEF that it would stop supplying discounted RotaTeq doses to the four West African nations, their only recourse to address the unexpected vaccine supply shortage was to help the affected countries switch to anot­­her rotavirus vaccine produced by a different company2.

This potentially problematic situation was avoided by a multilateral collaboration between aid organizations, governments, and manufacturers. Specifically, other pharmaceutical companies like GlaxoSmithKline (GSK), Serum Institute of India, and Bharat Biotech stepped in to fill the dearth left by Merck by pledging to supply their rotavirus vaccines (Rotarix, Rotavac, and Rotasiil) to the four West African countries. These countries are expected to switch to these alternative vaccines (which are actually cheaper than Merck’s) in 2020 after their supplies of RotaTeq run out6.

While this story ostensibly has a happy ending, it also highlights inherent problems in the way vaccines are supplied to the world, specifically poorer nations. These nations are reliant on aid organizations like GAVI and UNICEF to provide them with free or discounted vaccines, which are in turn dependent on profit-driven pharmaceutical companies to produce and supply the vaccines. Why can’t these multinational pharmaceutical companies, which have vast resources at their disposal, just produce more vaccines when there is a shortage?

The answer to this question is multifactorial. Firstly, vaccine production is complicated and only multinational pharmaceutical companies have the sufficient expertise, infrastructure, and manpower to produce high-quality vaccines that are safe and effective at scale. Thus, even these companies need advanced warning to produce millions of vaccine doses. Secondly, the prices for vaccines have been increasing in recent years, mostly due to high fixed overhead costs and exclusive licensing practices that preclude competition7. Thirdly, vaccines represent only about 2-3% of big pharma’s trillion-dollar production portfolio and are not the most profitable part of their portfolios8. Thus, pharmaceutical companies have little incentive to pick up the slack. How, then, can the costs of vaccines be reduced and who is supposed to step up to make vaccines when big pharmaceutical companies fall short of their obligations?

One potential solution to reduce the cost of vaccine production is for smaller pharmaceutical companies to produce generic vaccines. Generics are alternative versions of patented drugs and vaccines that are supposed to work just as well as the original formulation. In fact, India has become one of the world’s leading producers of generics and is a major source of vaccines and drugs for Africa. The issue with this solution is that the generics manufacturing industry is fraught with regulatory issues such as poor quality control and lack of oversight9. Africa is most starkly affected by these issues because manufacturers ship their lowest-quality drugs to the continent. Some of these products are completely counterfeit, with no active ingredients, while others have only a fraction of the active ingredients listed10. Without fixing quality control and oversight of generics manufacturing, this is not a viable long-term solution to provide vaccines to poor countries.

Another potential solution to increase vaccine coverage in Africa specifically would be to empower the continent to manufacture its own vaccines. Though Africa is home to nearly 17% of the world’s population, it produces less than 1% of the world’s vaccines and spends millions purchasing vaccines from foreign entities. For instance, African governments imported $900 million worth of basic vaccines for children in 201411. GAVI and UNICEF could partner with pharmaceutical companies to spearhead capacity building in specific African countries so they could produce their own vaccines, instead of making them dependent on vaccines from third parties. Apart from providing vaccines, this strategy would potentially build local biomedical capacity, provide jobs, and incentivize highly educated African expatriates to return to their home countries12.

Unfortunately, global vaccine stakeholders are hesitant to invest in building vaccine production capacity in Africa. For instance, profit-driven pharmaceutical companies tend to invest in healthy markets that will yield stable returns on their investments. Before investing in building a manufacturing facility, these companies must first ensure that the vaccines they produce will be bought for a certain price at a certain time12. Besides, It will be an uphill battle if Africa cannot produce vaccines more cheaply than other countries like India, which has already optimized infrastructure for producing vast quantities of vaccines cheaply13. Overall, there are many economic barriers that prevent Africa from developing its own vaccine manufacturing infrastructure.

In conclusion, the recent story revolving around providing rotavirus vaccine for West African countries exemplifies the complex challenges in supplying vaccines for the world, especially developing countries that contain some of the world’s most vulnerable populations. Despite the relatively vast resources that global organizations and companies like GAVI, Merck, GSK, and UNICEF have at their disposal, the demand for life-saving vaccines continues to outstrip the supply of vaccines. The bottom line is that vaccines are a vital component to the health of people and nations, and access to these medicines ought to be a universal human right.

  1. https://www.who.int/immunization/diseases/rotavirus/en/
  2. https://www.npr.org/sections/goatsandsoda/2018/11/01/655844287
  3. https://www.fiercepharma.com/pharma/amid-china-launch-merck-cites-supply-limits-plan-to-scale-down-rotateq-shipments-africa
  4. https://www.gavi.org/
  5. https://www.unicef.org/
  6. https://www.npr.org/sections/goatsandsoda/2019/05/31/726863111/it-looked-as-though-millions-of-babies-would-miss-out-on-a-lifesaving-vaccine
  7. https://www.who.int/immunization/programmes_systems/procurement/market/en/
  8. https://www.theatlantic.com/business/archive/2015/02/vaccines-are-profitable-so-what/385214/
  9. https://www.statnews.com/2019/07/22/indian-pharmaceutical-industry-drug-quality-charges/
  10. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4105729/
  11. https://www.dw.com/en/africa-wants-to-manufacture-its-own-vaccines/a-46151389
  12. https://www.frontiersin.org/articles/10.3389/fpubh.2019.00056/full
  13. https://www.thehindu.com/sci-tech/health/trusting-the-science/article25861183.ece

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Written by sciencepolicyforall

August 7, 2019 at 2:45 pm

Where to have a baby? Newborn screening is not created equal.

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By: Emily Wires, Ph.D.

Image by PublicDomainPictures from Pixabay 

In 2017, there were an estimated 3,855,500 live births in the United States, one of whom was my son. Within the first days of life, infants are tested for heritable disorders that can hinder physical and/or cognitive development. Unfortunately, the same set of potentially life-saving tests are not equal for all 3,855,500 infants. 

Since its conception in the 1960ss, newborn screening has been recognized as an essential preventative public health initiative. Serving as a frontline for early detection, conditions included on the screening panel are often considered rare and early intervention is essential to improved quality of life. The Newborn Screening Saves Lives Act was signed into law in 2008 and sought to bring public awareness among parents and health professionals as well as improve newborn screening programs.   

Currently, the panel of tested disorders varies state to state — yes, that means your baby’s health trajectory can be influenced by state lines. When considering birth location, most expecting parents grapple with the pros and cons of hospital versus birthing center versus home birth, but many are naïve to state regulations. Newborn screening standards, specimen collection, and follow-up procedures are implemented by state health departments. Contact information for newborn screening laboratories and follow-up program are available for individual states. Variations in statutes often extend beyond the panel of testable conditions, spilling over to funding for both screening programs and treatments, laboratory standards, and parents’ rights of refusal. To address discrepancies among states, the Department of Health and Human Services (HHS) has provided a Recommended Uniform Screening Panel (RUSP) that includes 34 core conditions and 26 secondary conditions and serves as a suggestive resource for state governments to incorporate as part of their screening programs. RUSP inclusion criteria stipulates a condition be identifiable 24-48 hours following birth, screening tests for the condition are available and validated, and the condition has been shown to benefit from early detection. Conditions included within RUSP are recommended by the Advisory Committee on Heritable Disorders in Newborns and Children along with the HHS Secretary. Despite these recommendations, not all states adhere to RUSP suggestions. 

Orphan drugs are intended to serve the rare disease community but are often commercially underdeveloped owing to their limited profitability. The Orphan Drug Act, passed in 1983, has since served as a legislative springboard set to improve the lives of individuals with rare diseases by incentivizing and accelerating drug discovery. Rare diseases are just that– rare, and because rare diseases are defined as disorders that affect less than 200,000 Americans at one time (with a sizeable proportion belonging to pediatric populations), pharmaceutical companies often expect to incur financial loss developing orphan drugs, which often means a treatment is not pursued. Collectively, however, rare diseases comprise a large healthcare need (an estimated 30 million people in the US are living with a rare disease) and have garnered increased public interest through the work of patient advocacy groupsresearch institutionsgrant funding opportunities, and public events such as Rare Disease Day

So, herein lies the dilemma. Are we essentially running a relay race but missing the handoff? Newborn screening assesses the presence of heritable conditions during a critical development period that otherwise may be missed and parallels an incentivized impetus for drug discovery, yet many states are dropping the proverbial baton regarding panel legislation. Consider, Spinal Muscular Atrophy (SMA) and its recent headlines in the rare disease community. SMA is a debilitating (and deadly), heritable disorder resulting in the loss of motor neurons and subsequent muscle atrophy. In 2016, the Food and Drug Administration approved Spinraza as the first-ever treatment of SMA. The American Academy of Neurology 2019 annual meeting recently reported data from 25 infants diagnosed with SMA at less than six weeks of age who received Spinraza prior to observable symptoms. By 14-34 months, none of these infants required permanent ventilation, all were able to sit and swallow without support, and most of them could walk on their own. However, despite this monumental advancement, not all states include SMA in their newborn screening panel. 

How do we become better for the rare disease community? Admittedly, a loaded question and likely multifaceted in approach. One potential approach would be to federally incentivize universal newborn screening across states. Screening implementation conditions typically vary due to financial constraints, frequency of disorder within state’s population, and treatment availability. California and Pennsylvania are currently the only states that with rare disease legislative caucuses. These bipartisan committees are comprised of individuals dedicated to bridging awareness among policy makers, scientists, patients, and the public. However, there are still discrepancies even among states with caucuses (California tests for 63 conditions, while Pennsylvania tests for 38), attesting to the critical necessity of a universal screening panel. 

Science is making vast strides in the detection and treatment of rare diseases. It is time for public health and policy makers follow suit in an appropriate manner, especially when time may be a crucial factor for our youngest rare disease patients

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Written by sciencepolicyforall

August 2, 2019 at 2:38 pm

Science Policy Around the Web – April 12, 2019

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By: Saurav Seshadri, PhD

Photo by Elijah Hiett on Unsplash

With Vertex, NHS back at the pricing table, CF advocates ratchet up the pressure

petition demanding coverage of the cystic fibrosis (CF) drug Orkambi in the UK has now garnered over 100,000 signatures, and must therefore be considered by Parliament for debate.  The milestone is the latest development in the struggle between Orkambi maker Vertex Pharmaceuticals and the British government, which began soon after Orkambi was approved in 2015.  The main point of contention is the price of the drug.  The UK’s National Institute for Health and Care Excellence (NICE) has refused to recommend Orkambi at Vertex’s asking price of £104,000 ($136,000) per patient per year, but Vertex has rejected the UK’s offer of £500 million for 5 years’ access, leaving both parties at an impasse.

The UK is not the first country to clash with Vertex over pricing. Several health agencies have refused to pay for Orkambi on the grounds that it is only marginally effective; some now face lawsuits, as programs like Medicaid are required to provide available drugs for qualifying patients.  While Orkambi is not as effective as Vertex’s first drug Kalydeco, it can be prescribed to more patients (up to 50% of those with CF).  Life expectancy for patients with CF is less than 40 years, and many patients are children, so even small improvements can be life-changing.  However, as with the multiple sclerosis drug Ocrevus, NICE seems unlikely to relent; on the contrary, UK Health Minister Matt Hancock recently accused Vertex of ‘hold[ing] the NHS to ransom’ and ‘profiteering’.  

For its part, Vertex is unlikely to compromise on the price of its best-selling drug, which brought in $1.26 billion in 2018.  CEO Jeffrey Leiden insists that this revenue is critical to the company’s continued investment in CF research.  Ironically, this stance may be pushing the UK closer to a measure that would jeopardize all future medical R&D efforts: invoking ‘Crown’ use, which allows the government to sell a patent without the consent of its owner. While the idea has gained support among some British lawmakers, and has been used in the past (to make Pfizer-owned antibiotic tetracycline available in the 1960s), it would face legal challenges that could render it ineffective.  But with public pressure mounting, especially after Vertex recently admitted to destroying almost 8,000 packs of Orkambi amid the standoff, inaction may not be an option for much longer.

(Eric Sagonowsky, FiercePharma)


Why some low-income neighborhoods are better than others

A recent study, published in PNAS, builds upon a body of evidence that while race can influence upward mobility (with white children having a 4-fold higher chance of moving from the lowest to highest income brackets than their black peers), environmental factors also play a major role.  Previous work demonstrated that the neighborhood in which a child grows up has a large effect on their future success, with better outcomes for children raised in low-poverty neighborhoods, regardless of race.  However, black children are significantly less likely to live in such neighborhoods.  To combat racial inequality, it is critical to understand which aspects of poverty impact long-term socioeconomic progress. 

The new study is based on the Opportunity Atlas, and pulls together data from tax returns, Census surveys, police reports, prison admission records, and blood tests conducted by the health department. The data tracks a cohort of children born in 1978-1983 (age 31-37 in 2014), living in 754 Census tracts in Chicago.  The authors report that even after controlling for other variables, a large proportion of the racial disparity observed in adults can be explained by three factors: violence, incarceration, and lead exposure during adolescence.  Since these factors were highly correlated with each other, the authors combined them into a single ‘neighborhood harshness/toxicity’ factor; this variable proved to be a much stronger predictor of income, incarceration, and teen pregnancy than more traditional factors, such as poverty or college education rates.   

That these elements impair social mobility is perhaps not surprising, as exposure to both violence in the community and high levels of lead have both been linked to cognitive impairment. But the magnitude of the effect is striking: for example, according to their model, toxicity exposure could account for 60% of the difference in incarceration rates between black and white men in their sample, and a 10% increase in teen births among black women.  While the authors acknowledge they cannot establish causality, they conclude that ‘Chicago’s residential segregation is disproportionately exposing its black children to neighborhoods that are hazardous to their development’.  Recently elected mayor Lori Lightfoot ran on a platform that includes stopping violence, expanding affordable housing, and ‘investing in our neighborhoods’.  Insight into the mechanisms that perpetuate inequality can only enhance these policies’ power to improve the trajectories of vulnerable kids.      

(Sujata Gupta, Science News


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Written by sciencepolicyforall

April 12, 2019 at 5:21 pm

Science Policy Around the Web – January 11, 2019

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By: Mary Weston, Ph.D.

1600px-aedes_albopictus

Source: Wikimedia

A Virus Even More Dangerous Than Zika to Pregnant Women

According to a recently published paper, Rift Valley Fever Virus (RVFV), a mosquito-borne virus found in Africa and the Middle East, may be even more damaging to fetuses than the Zika virus.

RVFV largely affects livestock, causing death and 90%-100% abortions in cattle. In humans, RVFV infections cause anything from mild flulike symptoms to severe liver problems. In 2016, a study associated the development of RVFV infection while pregnant with an increased likehood of miscarriage, but nothing further was known. However, a new study published last month in Science Advances shows that RVFV may severely harm human fetuses if contracted by women while pregnant.

The paper investigated how the virus affects pregnant rats, finding that 40% more pups died compared to uninfected controls and all surviving offspring contracted the virus. Further, the infected mothers’ placentas contained more virus than any other tissue. Upon testing human placenta tissue, they discovered that RVFV infects specialized cells that supports the region of the placenta where nutrients flow in, an area typically resistant to viral infections. According to the Dr. Amy Hartman, the infectious disease specialist at University of Pittsburgh who led the study, “Zika must take the ‘side roads’ into the placenta to infect a fetus, while the Rift Valley fever virus can take the ‘expressway.’”

Given that RVFV is carried by the same mosquitos found in Europe and America, there is a risk the virus could spread beyond Africa and the Middle East. Currently, there are no human vaccines or treatment for Rift Valley Fever and the World Health Organization has classified the disease as a potential public health emergency. Last week, the Coalition for Epidemic Preparedness Innovations launched a call for proposals to develop human vaccines against RVFV and Chikungunya virus, providing $48 million to finance up to eight projects

(Emily Baumgaertner, New York Times)

 

Prescription Drug Costs Driven By Manufacturer Price Hikes, Not Innovation

A new report published in Health Affairs argues that the rampant cost increase of many prescription drugs in the US is primarily due to price inflation, not the entry of new products or improvements to existing therapies.

The study compared pharmacy claims from the University of Pittsburgh Medical Center Health Plan and pricing data from First Databank, a company that collects prescription drug sales data, over the period of 2008-16. They found that the average costs of brand-name oral drugs annually increased 9.2%, while brand-name injectable drugs increased an average of 15.1% every year, five to 8 times the rate of general inflation. For example, the Health Care Cost Institute cited that the cost of insulin doubled from 2012-16.

The costs of generics and specialty drugs also increased during these time periods, but the authors determined that was due primarily to new product entry. During 2008-16, many blockbuster brand-name medications, such as Lipitor, lost their patent protection. There is typically lag time between becoming a generic and the time required to file generic applications. Thus, initial prices of generics are more closely matched with brand-name prices until more competition enters the marketplace, which factored into the report’s observed increase in generic pricing.

William Shrank, the chief medical officer of the UPMC Health Plan and an author on the study, argues that since rising costs are not improving treatments, policy makers may want to get involved. “This observation supports policy efforts designed to control health care spending by capping price inflation to some reasonable level,” he says.

Total US spending on prescription drugsin 2017 was $333 billion, a 0.4% increase from 2016, but a 41% increase compared to $236 billion in 2007. Additionally, according to a 2017 Commonwealth Fund study, US residents pay more for medications than any other high-income countries. Recently, efforts towards lowering/regulating prescription drug costs has received bipartisan support and this new report may help further those proposed regulations.

(Alison Kodjak,NPR)

 

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Written by sciencepolicyforall

January 11, 2019 at 4:44 pm