Overdue FDA reform to come?

The recent news that Sen. Ted Kennedy has brain cancer sharply focuses national attention on the tragedy of all forms of cancer. The senator has a malignant glioma so difficult to treat that half of those diagnosed with it die within a year, and nearly all are dead within two years.

There are many promising new cancer treatments in the pipeline, but under current Food and Drug Administration (FDA) regulations, almost no one gains access to them, no matter how dire the need or how compelling the evidence that the drugs work.

Most people receiving a terminal cancer diagnosis die before the most promising treatments in the pipeline reach them. Why? Because those tragic events occur on the wrong side of the magical moment when someone at the FDA puts an approval letter on a fax machine declaring the drug they needed - and never got - is "safe and effective."

Congress now has an opportunity to address this problem thanks to Sen. Sam Brownback (R., Kan.) and Rep. Diane Watson (D., Calif.), who recently introduced the Access, Compassion, Care, and Ethics for Seriously Ill Patients Act. The Access Act, said Mr. Brownback at a press conference introducing the bill, provides that "terminally ill patients whose medical needs are unmet by currently available options would be granted access to promising, investigational treatments."

As Ms. Watson observed at the same press conference, "The activism of the AIDS community in the 1990s expedited the marketing to the general public of promising antiretroviral drugs. Today it is my understanding that many AIDS drugs do not have to go through the controversial and questionable Phase III testing with placebo controls. Sadly, the expedited approval of promising new drugs for cancer patients and patients with other life-threatening diseases does not receive the same attention or expedited approval."

We know from personal experience - having received similar diagnoses for our spouses - what Mr. Kennedy and his family face, and it is our hope that he be given access to any promising treatment that can give him the best chance of extending his life. We support that access even if he gets it only because of who he is, a Kennedy and a U.S. senator. Our national shame is that humane access to effective drugs is not available to all with terminal illnesses.

Among the promising new therapies that should be available to Mr. Kennedy is a vaccine being developed at Duke University. The vaccine trains the body's immune system to kill malignant tumor cells, but to leave healthy cells alone. It is safe and effective, increasing average survival for patients with malignant gliomas like Mr. Kennedy's from 14 months to 33 months, based on the results of clinical trials presented in Chicago at a conference on cancer recently. Although the number of patients treated so far is small, the magnitude of the estimated survival difference and the strength of the underlying science makes it very unlikely that the positive effect is due to chance.

But the vaccine faces additional years of randomized trials in which a few hundred patients will get, or not get, the vaccine before the FDA considers approving it. Given what we know about the vaccine, the ethical problems with such trials are obvious, but the FDA will rigidly demand them.

In the meantime, the thousands who won't get into the trials will die waiting. What will we learn? That the survival advantage is a month or two less, or more, than the 19 months already estimated. That's it.

Mr. Kennedy's situation, identical from a regulatory standpoint to the plight of hundreds of thousands of other Americans, shouts to the heavens the humane necessity of urgent reform in the drug approval process to make it work better for people who have serious and terminal diseases.

The Access Act creates a new approval mechanism called Compassionate Investigational Access (CIA) for patients who can't wait. Patients receiving a CIA drug must suffer from a serious or life-threatening disease, be out of approved options and unable to gain access to a clinical trial, provide informed consent, and allow the collection of clinical data from their experience with the drug so we will all know more about the safety and efficacy of new therapies before they are approved for wider use.

The Access Act also improves the FDA's accelerated approval mechanism that has helped to preserve the lives of HIV/AIDS sufferers, but has never been adequately applied to other serious diseases like cancer.

Before his diagnosis, Mr. Kennedy was working on legislation to reinvigorate the war on cancer. The Access Act belongs in that package. Some of his friends in the Senate, including Democrat Bob Casey and Republican Arlen Specter of Pennsylvania, co-sponsors of the Access Act, will support him.

The unavoidable reality for people with a terminal disease is that good cancer drugs are held up behind a one-size-fits-all regulatory wall. The Access Act offers Congress, and Mr. Kennedy, a way to help break the regulatory logjam for all of us.

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What is wrong with Britain's proposed new polyclinics? Try the death of GP care

Britain may get more opening hours and new equipment but the doctor-patient relationship will suffer

Those mourning the absence of the English football team from Euro 2008 have a new spectator sport to distract them: the battle between health ministers and the medical profession over polyclinics, the Department of Health's brave new vision for healthcare. Doctors argue that the Government is adopting a Steve McClaren approach, with ill-conceived tactics that will end in tears; while politicians accuse the medics of acting like prima donnas - interested only in themselves, while currying favour with patients by kissing the NHS club badge.

The debate boils down to this. Last year's review by Lord Darzi of Denham into healthcare in London suggested the development of new facilities to increase the range of services for patients - polyclinics. The Government is mightily impressed with this idea and wants to run with it the length and breadth of England. The medical profession isn't, and wants to trip it up. Shiny new facilities, extended opening hours, multiple services under one roof? What's not to like?

Quite a lot, in fact. Open-all-hours surgeries may appeal to time-pressed, worried-well commuters. But to the most needy users - young families, the chronically sick, the elderly - geographical convenience is more important. Centralised services may make impressive buildings and economic sense, but are little use to Zimmer-frame-hampered patients.

Another disadvantage is the loss of continuity. It may be difficult to establish doctor-patient rapport in a polyclinic, given the number of staff, their shift patterns and the facelessness of the service. Cynics say that continuity is less sacred than the medical profession would have you believe - and, if the worst of a patient's problems is an ingrowing toenail, they may have a point. But for patients with multipathology - and, in our ageing society, that is a significant constituency - continuity is key. With it, patients have a clear point of reference, someone with his finger on the physical and metaphorical pulse; without it, there is a real risk of duplication, omission and disintegrated care.

"Care" is a killer word in the polyclinic debate. A clear message from countless patient-satisfaction surveys is that, while GPs may not always be able to cure, they do care. Whether this key facet of general practice will be retained in the new era remains to be seen. There are certainly concerns that the threat of privatisation - which many believe goes hand in hand with polyclinics - could make staff and managers focus more on income than illness.

Of course, ministers have ready answers to these objections. To a degree, they have a point: the new plans might work well for some people in some areas. They would argue that much of the opposition is simply the reflex rant of a profession notorious for its resistance to change - although this is not surprising if the change involves being coerced into new working patterns or environments, or seeing your lovingly nurtured patient list swallowed up by the corporate clinic down the road.

But the Department of Health might do well to look behind the rhetoric to ask why we medics - backed by the BMA, the Royal College of General Practitioners, the King's Fund and many patients - are quite so sceptical. Partly, it's change fatigue. Doctors emerge from one set of time-consuming reforms to find that another pile has landed on their desks - each reinventing the wheel and distracting from patient care. Then there is the feeling that we are being bullied. GPs are still licking their wounds from the fight with the Government over extended opening, in which they were given a choice between a slap round the face and a doing-over with a baseball bat.

These latest changes feel as if they are being imposed from above. Then there is a nagging suspicion that the polyclinic is just another bright idea. One which, like NHS Direct, walk-in centres, choose and book, computerised records, smacks of expensive, focus-group-driven initiatives, pandering to those with plenty of health wants but few genuine health needs.

Most of all, though, the medical profession's fire is fuelled by anxiety that the precious fundamentals of traditional healthcare - personalised care, continuity and patient advocacy - are in jeopardy. The polyclinic is perceived as a threat to the heart and soul of general practice.

A brief scroll down the GP forums confirms this - "One-to-one GP care will be lost forever", "The structure of family medicine in this country is being destroyed" and "This is the death knell for general practice". This depth of feeling won't be appeased by any semantic re-jigging of the concept - politicians should be less fixated on gratuitous innovation and more appreciative of what they have already. Otherwise, they will score an own goal that will relegate traditional general practice to the status of the English football team: talented, sorely missed and criminally redundant.

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