Friday, June 11, 2010

FDA: Protecting us to death

Today former FDA deputy commissioner Scott Gottlieb argues that:
“The time from lab to market for new drugs keeps getting shorter, but bad government policies threaten to reverse this trend ... News from this week's gathering of the American Society of Clinical Oncology … underscores how good we have become at turning new scientific principles into superior medicines.

Bristol Myers's drug Ipilimumab, the first treatment to extend the lives of patients with advanced melanoma skin cancer, is based on science that is 30 years in the making. Pfizer's drug Crizotinib, which shrank some of the most resistant and fatal forms of lung cancer, was developed as a result of science done over the last decade.”

Given that most drug research leads to no marketable product, it’s great that a few drug companies are able to shepherd their discoveries through the torturous regulatory system. However, my big brother warns that “pharamascolds” threaten that progress. Gottlieb agrees:
“Like all fragile ecosystems, the critical path for translating basic scientific principles into effective medicines is susceptible to outside forces. Lately, these are policies that shrink the incentives that drive the capital investment needed to underwrite these long and risky endeavors, or growing regulation by the Food and Drug Administration that makes it harder to get treatments to market.

Most ominous, the journey from lab to treatment is at risk from activists' and regulators' growing suspicion of the collaboration between the academic researchers who uncover basic science and the drug industry that is able to design and manufacture medicines. Yet that hand-off from researcher to manufacturer was behind Ipilimumab, Crizotinib and many of our best cancer treatments. …. Now Congress is endeavoring to investigate scientists who get National Institutes of Health research grants and also collaborate with industry.”

Gottlieb claims the future will bring even quicker development of useful medicines. However:
“Severing the links between the academic researchers that firm up basic science and the industries that craft medicines is the surest way to reverse this trend.”


Cheap cancer drug 'could save the sight of tens of thousands of older people'

A drug initially developed to fight cancer could provide a cheap way to save the sight of tens of thousands of older people, according to a new study. Doctors found that Avastin improved the sight of patients suffering from the leading cause of blindness in the elderly, a condition called age-related macular degeneration (AMD).

The drug could cost as little as £350 annually, much less than the £10,000-a-year price tag of the current therapy.

Developed to treat bowel cancer, Avastin is not yet licensed as a therapy for sight problems. However, based on early trials, the National Institute for Health and Clinical Excellence (Nice), the Government’s drugs rationing body, has started to look at whether it would be effective for AMD.

A new study by researchers at Moorfields Eye Hospital in London suggests that it would. The drug significantly improved the sight of most patients and did not appear to cause any harm to the eyes, it found.

Avastin works by targeting a protein involved in the formation of new blood vessels. This is crucial in AMD, which is caused by damage to the back of the eye triggered by leaking from abnormal blood vessels. More than 250,000 people in Britain currently suffer from the condition, with around 25,000 new cases diagnosed every year.

Campaigners fought a long battle with Nice to have access to Lucentis, which was initially deemed too expensive for the NHS.

Avastin is also expensive when it is used as a cancer drug, and it has been down for use as a bowel cancer treatment on the NHS by NICE, which said that at £21,000-a-year its costs outweighed its benefits.

However, the drug can be used much more cheaply to treat AMD. Doctors need only a fraction of the amount required to fight cancer, and treatment need only be given around seven times a year, bringing the costs down to around £50 a time, or £350 annually.

However, the trial, which is published in the British Medical Journal (BMJ), did not directly compare the effectiveness of Avastin to Lucentis.

Adnan Tufail, a consultant ophthalmologist at Moorfields, who led the latest study, said that before patients were moved on to the cheaper drug further trials were needed to ensure that Avastin was at least as successful in treating the condition.

Barbara McLaughlan, from Royal National Institute of Blind People (RNIB), said that patients should continue to be treated with Lucentis until "robust evidence" was available on the safety and effectiveness of Avastin.


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