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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.

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NHS ‘should not prescribe acne drug’

The parents of young people who have killed themselves and patients unable to have sex are calling f..

The parents of young people who have killed themselves and patients unable to have sex are calling for the NHS to stop prescribing acne drug Roaccutane.

Ed Henthorn said it had caused him erectile dysfunction, psychosis and suicidal thoughts.

And one man who believes his son killed himself after taking the drug said the risks "are just too high".

Manufacturer Roche said "millions of patients worldwide have benefited from taking the drug".

The majority of those who take the drug have a positive experience.

'Pretty overwhelming'

"I used to think about girls… but my feelings, thoughts, just faded away," Ed Henthorn told the BBC's Victoria Derbyshire programme.

He was 19 when he took Roaccutane. He describes his acne as mild but bad enough to want to treat.

After three weeks he started to experience side-effects, including reduced energy and sex drive.

Then he experienced erectile dysfunction.

"That was why I decided to stop taking it," he said. (more…)

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Spina bifida: Keyhole surgery repairs baby spine in womb

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In a UK first, doctors have used keyhole surgery to su..

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In a UK first, doctors have used keyhole surgery to successfully repair the spine of a baby with spina bifida while it was still inside the womb.

Surgeons at King's College Hospital say the procedure is not a cure, but could be the difference between some children learning to walk or not.

Sherrie Sharp and her son Jaxson had the operation 27 weeks into the pregnancy.

Spina bifida was diagnosed after the routine 20-week pregnancy scans.

They showed Jaxson's spine and spinal cord were not forming correctly.

Gaps in the developing spine meant the cord was bulging out of his back and was left exposed to the amniotic fluid in the womb.

This damages the crucial nerves in the spinal cord and could lead to paralysis, a loss of sensation in the legs and problems controlling the bladder and bowels.

The longer the spinal cord is left exposed, the greater the damage.

Sherrie, 29, and from West Sussex, said the news was a shock, but an abortion was a "definite no". (more…)

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Less chemotherapy better for older or frail patients with advanced stomach and oesophageal cancers

Less chemotherapy is as effective at controlling disease for elderly or frail patients with advanced..

Less chemotherapy is as effective at controlling disease for elderly or frail patients with advanced cancer of the stomach or oesophagus (food pipe), and leads to fewer side effects such as diarrhoea and lethargy. These are the results of a Cancer Research UK funded study, presented prior to the ASCO conference today (Wednesday).

“Increasingly were realising its not just age that affects how well someone can tolerate their treatment and we need to do more work to understand how other conditions or aspects of frailty might play a role.” Dr Peter Hall, Cancer Research UK Edinburgh Centre[contfnewc]

Results from the GO2 trial could change the standard of care for patients who cant have full dose chemotherapy due to their age, frailty or medical fitness.

The study, which ran at hospitals all over the UK, coordinated from the University of Leeds, involved 514 people with stomach or oesophageal cancer. Their average age was 76 and the oldest was 96 years old. All were either frail, elderly or medically unfit, and for those reasons would be unlikely to tolerate full-strength treatment, which involves three chemotherapy drugs.

Patients went through a careful medical assessment, then went onto chemotherapy with just two drugs* and were allocated at random to receive them at either full-strength, medium-dose or low-dose. They were then carefully monitored to see how well the cancer was controlled, whether they had symptoms and side-effects, whether they felt their treatment was worthwhile, and what overall effect it had on their quality of life.

The researchers reported that the medium and lower doses of chemotherapy were as effective as the full-strength dose for controlling the cancer. But when the researchers looked at the overall effect of treatment, including quality of life, they reported that it was the lowest dose treatment that came out best.**

Around 15,800 people in the UK are diagnosed with stomach and oesophageal cancers every year***. Almost half (45%) of these people are 75 and over****. By 2035, this proportion is projected to rise to 55%*****, because of the UKs ageing population. This study, is one of few phase III trials in the country that seek to address how to best care for and treat this increasing population of elderly or frail cancer patients.

These findings also open up the possibility of more older and frail patients being able to take part in clinical trials.

Professor Charles Swanton, Cancer Research UKs chief clinician, said: “These valuable results reduce fears that giving a lower dose chemotherapy regimen is inferior and could make a huge difference for patients with stomach or oesophageal cancer who cant tolerate intensive courses of treatment.

“Older or frail patients are often not considered for new drug trials or standard of care therapy as theyre less able to tolerate combination chemotherapy. These trials are critical to provide much needed evidence on the effectiveness of new therapies and combination approaches, helping us develop new treatments for this growing group of patients.”

The researchers also assessed whether there were differences for the patients in the study who were under 75, or less frail, who might be expected to benefit from stronger treatment; but will be reporting that the lowest dose treatment gave the best results for them as well. (more…)

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