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Teaching Big Pharma to share

This article is part of the Global Policy Lab: Decoding Cancer.

Researchers want to save the pharm..

This article is part of the Global Policy Lab: Decoding Cancer.

Researchers want to save the pharmaceutical industry time and money on clinical trials. But that means teaching companies to work together and convincing them to pool patient data, benefiting not just themselves but also their competitors.

Its not an easy sell.

“In the vast majority of cases, the company wants to be in control,” said Denis Lacombe, chief of the European Organisation for Research and Treatment of Cancer.

Lacombe is basically in a race against Big Pharma to build a public biological database before companies create their own private ones.

The stakes, for patients, are high.

Innovations in so-called precision medicine, in which treatments are tailored to individuals, has made patient data critically important. The more it is walled off in corporate troves, some cancer experts like Lacombe warn, the more the potential of using big data is undermined and the harder it becomes for patients to find relevant experimental treatments.

EORTCs SPECTA platform is a collection of genomic and other biological and clinical data. When a new treatment is available to be tested, the data can be searched to identify patients who could participate.

Thats something that will be increasingly important as personalized medicine drives pharmaceutical companies “to search for specific patients with specific [genetic] alterations,” Lacombe said.

That should be an opportunity for the pharma industry, said Lacombe. But it hasnt worked out that way.

The industrys been slow to engage with SPECTA. Lacombe is concerned that instead, companies are starting to collect peoples genomic data on their own, without having a particular treatment or trial in mind.

“There is a risk that the commercial sector will develop a … parallel route to access DNA,” he said. Its “suboptimal, if not unethical, that companies collect biological data and put it in a commercial silo.”

The EORTC isnt the only one trying to collect patient data.

A parallel effort with a more utopian vision is based in the neutral Alpine haven of Switzerland. MIDATA cites the philosopher John Rawls and his idea of “Property-Owning Democracy” in its promotional material. Its founder, Ernst Hafen, predicts that it could make even more money than Google or Facebook — though it would all be reinvested for the good of society.

People who contribute personal information to MIDATA — whether its their genome or smartphone steps log — would remain the owners of their personal info and get to sign off on how its used. Theyd also become shareholders in MIDATA, able to vote on how it conducts business and spends its profits — earned in part by pharma companies who pay to recruit study participants.

MIDATA is starting slowly, with some academic partnerships and some companies interested in how patients are faring with their migraine and multiple sclerosis drugs. But Hafen insists that MIDATA is the future, “a citizen-empowered way of changing the way science is done.”

If patient data is to be collected in public data bases, citizens might have to do the powering. But there are signs the industry is slowly coming around.

Signs point to the pharma industry increasingly being open to sharing data and cooperating on trials, but theyre not eager to talk about it publicly yet. Requests for comment from both the European Federation of Pharmaceutical Industries, the sectors Brussels lobby, and individual companies yielded no one willing to discuss these new initiatives.

Collaboration has never been the pharmaceutical industrys strong suit. While industries like aviation and semiconductors have been working together for decades, “the pharmaceutical industry has been lagging,” said Dalvir Gill, CEO of TransCelerate BioPharma, a non-profit initiative trying to change that.

Over the past seven years, TransCelerate has looked for ways to coordinate clinical trials. They have created a common set of forms for different companies trials. It also created a network for companies to procure comparable drugs from each other directly for use in trials, rather than having to buy the longer-standing treatment from third parties to test against a new drug.

Ten companies signed on to TransCelerate initially, up to 19 now. Its required companies to change their mindset, Gill said.

Theres a “vast amount of work that happens in the development of drugs that is not competitive,” Gill said. “The R&D people get it,” he added, but in the commercial divisions of a company, “theres a lot more convincing that needs to happen there.”

Regulators also need convincing, Gill noted — when companies cooperate, it raises a different set of eyebrows.

“Our biggest barriers are typically not running afoul of antitrust,” Gill said. “Whenever you have 19 companies in a room doing something,” its going to draw scrutiny.

With lots of new cancer drugs coming out for increasingly narrow subsets of cancer patients, so-called platform trials — in which multiple new drugs are tested at once — are seen as a key way to speed up the process and avoid duplication.

But companies are deeply resistant to the idea of having their new idea compared (possibly unfavorably) to someone elses.

The I-SPY 2 trial for breast cancer, non-profit platform trial that has resulted in three drugs winning approval, showed drugs can be tested at the same time without being pitted against each other. Instead theyre tested against the existing standard of care.

Brian Alexander, a Harvard radiation oncologist preparing to launch a platform trial for brain tumors called GBM AGILE, said that the precedent has made it easier for him to convince companies to let him test their drugs.

“They werent as worried anymore because once you see two New England Journal [of Medicine] papers in the same edition, both coming from arms from I-SPY 2, and theyre not compar[ed to] each other… then [drugmakers] were like, Oh, ok, that isnt that much of an issue,” Alexander said.

Hes expecting to announce the first drug to be tested in the new GBM AGILE trial next week, but he said that overcoming companies fear of sacrificing control remained a major challenge.

“To feel like theyre turning over the late stage development of an asset to this other entity — thats a bit of a tough sell,” Alexander said.

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The organization helping to bring new drugs for rare diseases to market

A research team has reason to celebrate after the Food and Drug Administration granted it approval o..

A research team has reason to celebrate after the Food and Drug Administration granted it approval on Friday to begin a clinical trial for a new pediatric brain cancer drug, one that might have ended up overlooked by pharmaceutical companies.

The lead researcher on the team, Dr. Teresa Purzner has already beat impossible odds. The neurosurgeon and mom of three managed to get the approval in record time and with little money thanks to the help of a team of scientific altruists called SPARK.

The development of new medications in the United States is driven by pharmaceutical companies; researchers at universities rarely bring their discoveries to the bedside. For every 10,000 potential new medicines sitting on laboratory shelves around the country, only one will ever reach patients in need, according to the National Institutes of Health. Why? Because the process can take 10 to 15 years, costing upwards of a billion dollars per drug.

As a result, the number of new medications approved by the FDA has remained stagnant at about 31 per year over the past 10 years. The majority of these medications are similar to already existing ones, and many target diseases for which there are large markets — like hypertension and high cholesterol — and therefore, a return on investment.

Enter SPARK, a non-profit program created in partnership between Stanford University and volunteers from the biotechnology, pharmaceutical, and investment industries, which helps academic researchers bring their discoveries to patients. Since its founding, SPARK has given special consideration to projects typically neglected by pharmaceutical companies, including rare diseases and diseases affecting children.

Purzner put her neurosurgery practice on pause to study medulloblastoma, a type of childhood brain cancer. Compared to diseases like hypertension and high cholesterol, which affect millions of Americans, medulloblastoma is rare, affecting only 250 to 500 children every year.

“Theres something especially poignant about seeing children —beautiful, wonderful, innocent things — and seeing the impact of the therapies we are giving them. The medications, the radiation therapies impact their cognition, their quality of life and their ability to function as independent adults in the future,” Purzner said in an interview with ABC News.

Purzner had a clear goal: to find a targeted therapy that could shut down the basic biochemical pathway responsible for the development of this cancer, and she did. She tested the potential drug in mice with good results, and she just received FDA approval to test it in clinical trials, which she will do through the Pediatric Brain Tumor Consortium. She did it all in five years and for a price tag of $500,000.

“To get from my initial findings in the lab to the point where the Pediatric Brain Tumor Consortium picked it up for clinical trials would have never happened without the help of SPARK… they gave me a clear pathway and made me believe it was possible,” said Purzner.

Every year, SPARK provides 10 teams with funding and expert mentorship to promote efficient and cost-effective drug development. (more…)

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Widowed father works with congresswoman on legislation to prevent maternal deaths

Sitting in the hospital room, mother and newborn baby were sound asleep.

“I was overjoyed. I reme..

Sitting in the hospital room, mother and newborn baby were sound asleep.

"I was overjoyed. I remember thinking my family is complete," Charles Johnson told ABC News.

But then he looked down and saw his wife Kiras catheter turn pink and then red with blood.

April 12, 2016 was supposed to be a joyous day for the Johnson family, but it turned into a "nightmare."

Ten hours later, Kira Johnson died as a result of internal bleeding following a cesarean section.

Now, two years later, Johnson is raising two children on his own and advocating to rectify the country's maternal health policies and regulations to prevent anyone else from sharing the same tragedy. Johnson took to Capitol Hill to share his wife's story before members of Congress, working alongside a congresswoman who experienced her own personal difficulties during pregnancy.

Charles and Kira Johnson welcomed their first son Charles V. in 2014. He was delivered via C-section. Two years later, the Johnson family relocated from Atlanta to Los Angeles and learned they were expecting their second baby boy.

"Kira and I had always wanted two boys," Johnson said. "I was excited."

The Johnsons decided to have Langston delivered at Cedars Sinai medical center, a non-profit hospital that is currently ranked as the eighth best hospital in the country by U.S. News and World Report.

Charles Johnson said his wife was in exceptional health and that she took all the necessary prenatal measures to ensure their second child would be born healthy. Since their first son was born via C-section, the doctor suggested the same for their second. (more…)

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States look to breathalyze convicted drunk drivers to reduce fatalities

This story is from Kaiser Health News

On Jan. 1, California joined the majority of states that ha..

This story is from Kaiser Health News

On Jan. 1, California joined the majority of states that have laws requiring drivers with drunken-driving convictions to install breathalyzers in vehicles they own or operate.

Researchers, public health advocates and political leaders believe these laws are helping reduce alcohol-related road deaths.

The gadgets, known as ignition interlock devices, are mounted on the steering wheel of a vehicle and prevent it from starting if the drivers blood-alcohol reading is above a predetermined level.

In California, the breathalyzers are mandatory only for repeat offenders. Five other states — Georgia, Indiana, Massachusetts, Montana and Ohio — have similar laws. Thirty-two states and D.C. require the devices even for first-time offenders.

The advent of such laws across the United States in the past 15 years has been accompanied by some good news: Deaths involving drunken driving are only about half of what they were in the early 1980s, though they have ticked back up in recent years. The long-term decline is largely attributable to greater public awareness, stricter seat belt enforcement and the establishment in 2000 of a nationwide legal blood-alcohol threshold of 0.08 percent — far below the 0.15 percent standard commonly used before then.

State Sen. Jerry Hill (D-San Mateo), the author of the California law, said breathalyzers in cars will make roads safer than under the current law, which generally relies on license restrictions and suspensions.

“Weve seen people on a suspended license continue to drive and continue to cause destruction,” said Hill, who lost his best friend to drunken driving in the 1980s.

There is some evidence that the breathalyzers have an impact. Nationally, from 2006 to 2016, ignition-locking breathalyzers prevented 2.3 million attempts to drive by people with a blood-alcohol level at or above 0.08 percent — the legal threshold for driving under the influence — according to a 2017 report by the advocacy group Mothers Against Drunk Driving.

Emma McGinty, an associate professor at the Johns Hopkins Bloomberg School of Public Health, found that laws requiring interlocks for all DUI offenders were associated with a 7 percent drop in the rate of fatal crashes caused by drunken drivers. Another study found that laws covering all offenders were associated with 15 percent fewer alcohol-related fatalities compared with states that have less stringent laws. (more…)

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