Connect with us


5 questions for the future of cancer treatment

This policy brainstorm white paper is part of Global Policy Lab: Decoding Cancer.

A transformation..

This policy brainstorm white paper is part of Global Policy Lab: Decoding Cancer.

A transformation is underway in cancer care, with both drugs and treatment strategies increasingly personalized for individual patients. The genetic profile of a tumor can tell us which medicines are most likely to work. Teams of specialists will collaborate to work out the best course of treatment for a specific tumor. Therapies like CAR-T even promise to re-engineer patients own cells to fight their specific cancer.

Health systems are not transforming at the same rate. High costs and novel approaches to treatment are confounding old systems that werent designed to pay for them.

The transformation also means operating with a higher degree of uncertainty: Its urgent to give patients a fighting chance, but sometimes drugs are so new, or tested in such small populations, that we dont really know what were getting — or if the results were measuring are actually those most important to patients and their families.

POLITICO Global Policy Lab has been looking at how to move the cancer care system ahead of the science, including ways to make sure advances dont leave some patients behind. In this brainstorm white paper, we lay out the key questions facing policymakers, industry leaders and society as the fight against tumors gets increasingly personal.

The problem: Breakthrough cancer drugs are expensive: They cost a lot to discover, and in many cases, theyre designed for just a small sliver of patients, driving developers to seek high prices. Health systems are struggling to adapt — and cost isnt the only factor. Cancer experts increasingly look to fight tumors together in teams, and new therapies like CAR-T can only be provided in specialized centers. Insurance systems arent necessarily set up to pay for these approaches.
The question: How can health systems provide access to innovative new therapies? What can we learn from the current experience with the newly approved CAR-T treatments Kymriah and Yescarta?

The problem: Regulators are changing their approach to approving cancer drugs for patients with no other options, allowing treatments to enter the market based on smaller studies, though often with more dramatic initial results. The idea is that “real world data” will help us reassess those drugs in the future. For the health systems and insurance funds that have to pay for these drugs, however, determining a fair price amid so much uncertainty is a challenge.
The question: How does the drug price negotiation process need to change to accommodate experimental drugs?

The problem: Some view artificial intelligence as an increasingly important tool for diagnosis and treatment of cancer. But winning the trust of patients, and perhaps more crucially, the doctors whose livelihoods could be transformed by AI, could be a barrier. Likewise, concerns about data protection and confusion about ownership of patient information stand as barriers to personalized medicine that relies on genetic data.
The question: How do we find the right balance of embracing data and AIs potential without undermining trust, privacy and quality of care?

The problem: Theres a growing consensus that specialized cancer centers — where doctors have a lot of experience and can collaborate with experts across different fields of medicine — are key to improving outcomes. But studies also show that living far away from a cancer hospital can lead to later diagnosis and lower-quality care. This tension is especially acute for treating rare and childhood cancers, where there may only be a few experts in Europe.
The question: What are the unique sources of disparities in cancer research, treatment and long-term care, and how do we address them? What are the key barriers to treating rare and childhood cancers, and how can we overcome them?

The problem: “Patient-centered care” has become a buzzword, but its definition is fuzzy. While many clinical studies focus on improving survival, the effect of new drugs on quality of life is often an afterthought in the development process. As new treatments lead to longer lives, many cancer survivors find they face discrimination and long-term health effects.
The question: How can patients priorities be measured and incorporated throughout the treatment process, and beyond?

POLITICO Global Policy Lab is a collaborative journalism project seeking solutions to pressing policy problems. Join the community.

Original Article

Continue Reading


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…)

Continue Reading


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…)

Continue Reading


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…)

Continue Reading