Every year hundreds (perhaps thousands; nobody knows the real figure) of new, lab-tested drugs are abandoned.

There’s often nothing obviously wrong with these potential medical treatments.

In the laboratory, they have been shown to melt away tumours, make brain plaques disappear, puff up weakened bones with calcium, help mice and dogs live twice as long as usual.

Of course, most of these treatments are likely to turn out to be useless (or even dangerous) in humans.

But a few could save lives and ease the suffering of millions.

We won’t know unless we do further research.

But that research is not happening.

The problem is financial

According to the Tufts Center for the Study of Drug Development, the leading reason for drugs to fail human safety tests (the earliest phase of human experimentation) has nothing to do with whether or not the drugs are safe.

It’s because the scientists run out of cash and so cannot complete the early tests and move on to the next phase. 

Of the few drugs that get through to later experiments, in which researchers are starting to look for efficacy, almost a further third are dropped for financial reasons, not because they’re ineffective. 

In the brutal language of the pharmaceutical industry, this gap between lab discovery and the start of the large-scale, late-phase human experiments is called the “valley of death”.

The valley of death doesn’t cost much to cross. 

In many cases, between £2m and £5m is ample; without that money, no discovery stands a chance. 

Context: £2m is the price of an Aston Martin Valiant. It’s what it costs to rent Richard Branson’s Necker Island for two and a half weeks.

Industry and government policymakers are flummoxed by how to bridge the valley of death.

There are only two broad ways to fund clinical trials:

Profit-making (venture capitalists, biotech, big pharma)

or altruism (charities, government finance).

For venture capitalists, the pressures of the market exclude ideas that require patience to investigate, or seem too risky because they’re unconventional. 

For the altruistic crowd – statutory and benevolent funders – money is tight; the focus must be on drugs that will benefit the most patients with the least risk of failure.

Many brilliant potential medications are daring, crazily unfashionable, require decades of dull, careful nurture, or are out of patent and cheap as chips.

Products like these don’t appeal to greed or cash-strapped benevolence.

Let’s introduce self-interest

At the moment patients for promising medical trials are selected on a strictly ethical basis.

That’s good. Civilised.

But how about allowing wealthy people to buy places on clinical trials?

If we allow this, the wealthy person’s self-interest could help them and bring forward new treatments that benefit everyone.

This could generate billions of pounds of money for research 

It won’t get drugs all the way to market – the full process can cost anywhere from £50m to hundreds of millions of pounds – but it will help to pick out the products worth a further look.

Meanwhile the people in the trial might be cured.

There’s already a good number of drugs rescued from the valley of death by public intervention, including:

• Kalydeco for cystic fibrosis
• Gleevec for leukemia
• Herceptin for breast cancer
  
  

   

This is not “pay to play”

Medical ethicists rightly howl at schemes in which participants have to pay to take part in clinical experiments.

They’re called “pay to play” trials and, outside poorly regulated countries, they are expressly forbidden.

Wretched and desolate patients drain their pension funds, bankrupt their families and sell their houses to join studies based on flimsy research for drugs that don’t work, and could kill.

Charging patients to participate is the preferred finance technique for deluded zealots and quacks, because no respectable funding body will touch their sloppy research.

The cost to the patient can be anywhere between £7,000 and £250,000.

Ironically (as every con artist knows) this prohibitive cost is part of the appeal of pay to play.

Something so valuable must be good, thinks the patient.

(A few years ago, in the weirdness of Florida, the “young blood project” saw wealthy old people hooked up to blood extracted from teenagers, at a cost of $285,000 each.)

But there are several assumptions in this grim picture of pay-to-play clinical research, whereby desperate patients are fleeced by substandard operators.

What if you remove some of them? Take the assumption that pay-to-play trials charge all participants.

Instead this idea bills only one participant 

Everyone else participates for free (as is usually the case in a respectable study),

but this one patient is charged the most absurd sum of all: the cost of the entire trial, that £2m to £5m.

If such a funding tactic were run by a well-regulated body, on peer-reviewed research, and it is impressed on all participants that new drugs carry huge risks as well as possible benefits, then the usual ethical objections to pay to play disappear.

This would work best for small studies, for rare (and frequently underfunded) diseases.

But what idiot is going to agree to pay the full cost of a clinical study just to ensure that nobody else has to pay a penny?

How about: anyone who can afford it. 

Approximately half a million people in the world are worth more than £10m

They don’t want to die.

They have people they love, just like the rest of us; they don’t want them to die either.

If these rich people could spend a measly 20% of their wealth to rescue a piece of quality research that may give them an outside chance to save their own lives, or the life of their mother, husband or daughter, they’d do it like a shot.

As for the poorer patients? 

That’s a quid pro quo:

without them there could be no clinical study, because the research would have only one participant, which is statistically useless.

To rescue lost drugs, the mega-rich and the poor have to work together, for the weal of all.

The rich patient is shackled to benevolence.

We know this works, because we’ve tried it

This idea is the brainchild of Alexander Masters.

More than a decade ago, Alexander’s best friend, editor and co-writer, Dido Davies, was dying from pancreatic neuroendocrine cancer (the same disease that killed Steve Jobs).

Alexander heard about a potential treatment in Sweden, flew out to meet the astonishingly open-minded and supportive Prof Magnus Essand, and got him to agree to a deal:

If Alexander secured the money that he needed to run a trial, he’d make Dido a participant. 

Alexander didn’t have £200 to spare, let alone £2m, but he hunted it down.

He found an oilman with the disease (living, as it happened, in a mansion just down the road from the Brocher, in Geneva) and got him to give  more than two-thirds of the money, also in return for participation.

The rest was raised, with considerably more effort and much more skill, by crowdfunding, through the tireless work of Dominic Nutt, the second founding member, and a third campaigner, Liz Scarff.

They had successfully rescued a drug and started it on its precarious journey to market.

Tragically, Dido died on the day the campaign announced it had secured the money.

The oilman – a lovely person, called Vince Hamilton – also died before the study could begin. 

As Alexander says “I lost interest in fundraising for stupid, boring life-saving ideas that don’t save lives in time.”

That was in 2013.

In the 12 years since, several researchers, trawling through the outer reaches of bioethics literature, have come across a paper about the idea – jokingly referred to as “the Plutocratic Proposal” that Alexander and Dom  published in the Journal of Medical Ethics, and got in touch to see if this could save their underfunded drug.

These fleeting collaborations with expertise – sometimes no more than a Zoom call long – have enriched the idea.

What began as one counterexample to the idea that all pay-to-play schemes are inherently unethical is now a set of five different proposals, covering early- and late-stage clinical studies, developed in cooperation with bioethicists, medical scientists and the odd biotech executive.

An Example

One collaboration almost led to money.

An Oxford Professor got in touch. 

He had a potential treatment for Stoneman syndrome, also known as fibrodysplasia ossificans progressiva, a disease in which muscle and connective tissue are gradually replaced by bone, causing the patient to grow a rigid second skeleton. 

A neglected treatment, a small proposed study, a heart-wrenching story that would make it easy to run a publicity campaign: perfect for the Plutocratic Proposal.

“But only 800 people in the world have it,” warned the scientist.

Alexander did a back of the envelope calculation: 

8 billion people on earth, 800 people … so, one in 10 million … 

Trial needs £2.5m … Donor needs assets of, say, £5m-plus … 

About 2.5 million people in the world are worth more than £5m. 

So, if you take the chance of having the condition (ie 1/10m) and multiply it by that number of eligible plutocrats, you get … one quarter of one plutocrat.

“More than enough!” Alexander pronounced, delighted.

One of the advantages of the Plutocratic Proposal’s use of desperation as a funding force is that you don’t have to worry too much about calculations like this. 

The super-rich don’t need to suffer from Stoneman syndrome themselves to feel desperate to finance research. 

It’s enough that a friend or family member has it, or a friend of a friend. 

Rich people have hearts, and the fact widens the catchment area considerably. 

A quarter of a wealthy plutocrat is as good as a whole.

Read about our Matching Agency to introduce wealthy “plutocratic patients” to scientists with potential solution