[ed. Interesting, not only for the proposal, but the ethics and procedures involved in getting a new drug to market.]
“But you have missed the bigger idea!” exclaimed Peter Lanciano, grabbing the pepper grinder and banging it on the table. “The problem isn’t how to get my drug into Mr Pepperpot. The problem is how to protect me from being sued if Mr Pepperpot dies.”
It had taken me two years to track Lanciano down. For this meeting I’d broken off my holiday, woken up at three in the morning and flown 1,000 miles across Europe to have breakfast at a London hotel built like a penitentiary. Lanciano is the Executive Director of a small US drug company. In his early 50s, with a Teddy Roosevelt moustache and a lumberjack shirt stretched tight across his broad chest, I believe he can help solve a niggling problem that holds back medical research around the world and makes patients suffer. Every year, an untold number of potential new drugs or interventions, any one of which might go on to improve thousands of lives, are thrown away without being tested in humans. It is a matter of funding, not science: there is not enough money in the public or private sector to run clinical trials on every exciting proposal that comes out of research labs. Thoughtful but hurried (and often arbitrary) judgements are therefore made about which products to save – and the rest of these potentially life-saving therapies are ditched. “There’s tons of promising stuff out there,” says David Stojdl, cofounder of the Californian biotech company Jennerex Biotherapeutics, “and it is dying on the vine.”
I have a simple proposal for a way to rescue this waste. I’m not a scientist or a physician; I have no medical training. I’m a biographer and an illustrator, and until a couple of years ago I’d never heard of clinical trials. But I know my idea works because I’ve already tried it once, to rescue a promising anticancer therapeutic that was about to be thrown out in Sweden. The general version of my proposal has now received backing from a select group of university research departments and a clutch of experts on medical ethics, and has the interest of one of the world’s largest law firms specialising in the life sciences. If the scheme can be made to work on a larger scale, it will open up the possibility of millions (I think, billions) of pounds of extra money for clinical trials, especially for rare and difficult-to-treat diseases – the ones that traditional funders are reluctant to support. (...)
I began thinking again about the fundraising we’d done. Why not extend the principle of selling trial places, to raise money for other Uppsalas and other diseases: not just neuroendocrine cancer, or just cancer, but any illness? There are over 12 million millionaires in the world – any one of these would want to buy a place on a trial if it might purchase relief or stave off death. Every one of them has people they love for whom they’d pay good money to get an extra chance. Why not set up a charitable or private body that would arrange these ‘sales’?
My first thought was that it would be run like a dating agency. (...)
“But you have missed the bigger idea!” exclaimed Peter Lanciano, grabbing the pepper grinder and banging it on the table. “The problem isn’t how to get my drug into Mr Pepperpot. The problem is how to protect me from being sued if Mr Pepperpot dies.”
It had taken me two years to track Lanciano down. For this meeting I’d broken off my holiday, woken up at three in the morning and flown 1,000 miles across Europe to have breakfast at a London hotel built like a penitentiary. Lanciano is the Executive Director of a small US drug company. In his early 50s, with a Teddy Roosevelt moustache and a lumberjack shirt stretched tight across his broad chest, I believe he can help solve a niggling problem that holds back medical research around the world and makes patients suffer. Every year, an untold number of potential new drugs or interventions, any one of which might go on to improve thousands of lives, are thrown away without being tested in humans. It is a matter of funding, not science: there is not enough money in the public or private sector to run clinical trials on every exciting proposal that comes out of research labs. Thoughtful but hurried (and often arbitrary) judgements are therefore made about which products to save – and the rest of these potentially life-saving therapies are ditched. “There’s tons of promising stuff out there,” says David Stojdl, cofounder of the Californian biotech company Jennerex Biotherapeutics, “and it is dying on the vine.”I have a simple proposal for a way to rescue this waste. I’m not a scientist or a physician; I have no medical training. I’m a biographer and an illustrator, and until a couple of years ago I’d never heard of clinical trials. But I know my idea works because I’ve already tried it once, to rescue a promising anticancer therapeutic that was about to be thrown out in Sweden. The general version of my proposal has now received backing from a select group of university research departments and a clutch of experts on medical ethics, and has the interest of one of the world’s largest law firms specialising in the life sciences. If the scheme can be made to work on a larger scale, it will open up the possibility of millions (I think, billions) of pounds of extra money for clinical trials, especially for rare and difficult-to-treat diseases – the ones that traditional funders are reluctant to support. (...)
I began thinking again about the fundraising we’d done. Why not extend the principle of selling trial places, to raise money for other Uppsalas and other diseases: not just neuroendocrine cancer, or just cancer, but any illness? There are over 12 million millionaires in the world – any one of these would want to buy a place on a trial if it might purchase relief or stave off death. Every one of them has people they love for whom they’d pay good money to get an extra chance. Why not set up a charitable or private body that would arrange these ‘sales’?
My first thought was that it would be run like a dating agency. (...)
Wealthy people financing clinical trials is not new: this type of private funding already exists all over the world if all you want to do is make money. As Savulescu said, “If I were a venture capitalist, I could invest millions of dollars in funding the development of a drug, hoping to make hundreds of millions of dollars if it’s successful. So why shouldn’t I be able to pay the same money for the same development, to have a chance of saving my life? It is completely ludicrous.” (...)
In medical parlance, there are typically three phases of trials a drug has to pass before it can be sold commercially. Phase I tests the tolerable dose range and safety in healthy volunteers or, in the case of serious diseases such as cancer, in sick patients. With a few exceptions, the doses used are too small to offer medical benefit. In phase II the research team tests efficacy, and uses the information from phase I to provide potentially therapeutic treatment at the optimal safe dose. This second phase of trialling can be divided into two parts: IIa, which is open to all suitable patients and has no placebo wing, and IIb, in which placebos and randomisation are introduced. Phase III tests whether the drug is better than the best already available – this is abominably expensive, involves hundreds of people and is not worth thinking about unless you’re a multibillionaire.
O’Connor’s objection therefore restricted the Dating Agency to brokering phase I or phase IIa trials. That’s not terrible. That’s almost all it was intended to do anyway: get promising preclinical research over the hurdle into early-stage clinical trials, because that comparatively small amount of money is way beyond the reach of ordinary university departments. Uppsala is a combined phase I/phase IIa trial.
O’Connor’s next objection had not occurred to me at all: “What happens if the drug works?”
In medical parlance, there are typically three phases of trials a drug has to pass before it can be sold commercially. Phase I tests the tolerable dose range and safety in healthy volunteers or, in the case of serious diseases such as cancer, in sick patients. With a few exceptions, the doses used are too small to offer medical benefit. In phase II the research team tests efficacy, and uses the information from phase I to provide potentially therapeutic treatment at the optimal safe dose. This second phase of trialling can be divided into two parts: IIa, which is open to all suitable patients and has no placebo wing, and IIb, in which placebos and randomisation are introduced. Phase III tests whether the drug is better than the best already available – this is abominably expensive, involves hundreds of people and is not worth thinking about unless you’re a multibillionaire.
O’Connor’s objection therefore restricted the Dating Agency to brokering phase I or phase IIa trials. That’s not terrible. That’s almost all it was intended to do anyway: get promising preclinical research over the hurdle into early-stage clinical trials, because that comparatively small amount of money is way beyond the reach of ordinary university departments. Uppsala is a combined phase I/phase IIa trial.
O’Connor’s next objection had not occurred to me at all: “What happens if the drug works?”
by Alexander Masters, Mosaic | Read more:
Image: Jean Jullien at Handsome Frank
