What Is Open Source Pharma (and Why Should You Care)?

Andy Updegrove writes: Humanity today is almost completely dependent on huge pharmaceutical companies to create the drugs we need. But these companies focus exclusively on drugs that can be sold at high prices to large populations — in other words, to patients in developed nations. This means that those who live in the emerging world that suffer from the remaining 'neglected diseases,' like Malaria and drug resistant TB, have no one to depend on for relief except huge charities, like the Gates Foundation. They also have no way to afford many of the patented drugs that do exist. But there is another way, modeled on open source software development, which relies on crowd sourced knowledge, highly distributed, volunteer efforts, and advanced open source tools. That methodology is called Open Source Pharma, and it has the potential to dramatically drive down drug development while saving millions of lives every year.

Comments

Actually no

[rsilvergun]

most of the (expensive) basic research is still done on the public dime. Then big pharma comes in, runs a few (cheap) clinical trials, patents the whole shabang and blamo, new drug. You didn't think mega corps actually paid for things like us little people, did you?

Re:Actually no

[ShanghaiBill]

Then big pharma comes in, runs a few (cheap) clinical trials

Clinical trials are not "cheap". They are usually the most expensive part of bringing a new drug to market.

Re:Actually no

[LetterRip]

Clinical trials are not "cheap". They are usually the most expensive part of bringing a new drug to market.

The expensive trials are when we have a drug that treats the problem extremely well, the new drug appears to offer little or no benefit, and thus they have to offer people ridiculous sums of money to be recruited into the trial, and they have to have a enormous number of people enter the trial to show an effect size.

For trials where there is no effective treatment, and the new treatment should be highly effective, the cost of trials is quite modest.

Re:Actually no

[the gnat]

For trials where there is no effective treatment, and the new treatment should be highly effective, the cost of trials is quite modest.

The problem is that a lot of the proposed new treatments turn out not to be highly effective, but companies don't find this out until they've already sunk tens or hundreds of millions of dollars into it. So the cost of every new FDA approval needs to be balanced against all of the bets that failed.

This is true even for diseases where there isn't an effective treatment - clinical trials for Alzheimer's drugs (a sure money-maker, if they worked) have been notoriously failure-prone.

Re:Actually no

[RandCraw]

Yes. Clinical trials are famously expensive -- no less than $100 million US for any drug that is not fast tracked, which reduces development time (and cost) by no more than half.

In general only untreatable mortal diseases like cancer or infection can fast track a drug. The other 95% of drugs go through probably 5 years of compound identification, tuning, and testing, then 5 years of preclinical trials in multiple animal species, then another 5 years in humans before approval. (Yes, that's about 15 years.)

The last phase of development (clinical trials) is unavoidably very expensive (80% of the overall cost). And no amount of free software or volunteerism is going to change that appreciably. Even now, all patient participants in clinical trials are already volunteers.

The cost of a new drug lies in planning, testing, write up, peer review, and great gobs of regulatory oversight and process. Open source cannot change that.

Re:Actually no

[RandCraw]

I know more about nonhuman studies than clinical, but according to the US HHS (who runs FDA), the breakdown of costs are these:

- $15k/patient for phase I
- $20k/patient for phase 2
- $25k/patient for phases 3 and 4

The cost of the average trial:

- phase 1: $4 million
- phase 2: $13 million
- phase 3: $20 million
- phase 4: $20 million

Some phase 3 trials can be larger and last longer than average, like 20,000 patients over 5 years. Obviously at the average cost of $25k/patient, such a trial would cost $500 million, well over the average. In fact, a long study can greatly increase the per patient cost as well.

Because multiple trials are run in each phase for each drug, these trial costs are multiplied.

The principal cost in any trial are the medical procedures (~25%): drug administration, tests (lab, imaging, biopsy, etc), exams, etc. These are repeated multiple times on each patient during each trial to monitor changes in both efficacy and safety.

Here's a thorough accounting from US HHS:

http://aspe.hhs.gov/report/exa...

These costs are set by FDA regulatory standards and the medical laws of each country where the trial is performed. Of course if you want approval for your drug in another country, you must comply with all their rules as well, often repeating studies using their residents (e.g. Japan).

This 2012 Forbes article by Avik Roy offers further insight on why clinical trial costs are rising:

http://www.forbes.com/sites/ar...

Pharmas must play by these rules, but they don't write them. Lawmakers do that.

Re:This won't be allowed to happen

[ScentCone]

cannabis cures CANCER

Man, the stoners really will pitch any ridiculous meme they can latch on to, won't they?

Re:This won't be allowed to happen

[ScentCone]

I have Chinese neighbours and they're showing me new (to me) stuff all the time

You mean like how grinding up the horn of a rhinoceros and eating it will fix erectile dysfunction because, you know, horns are sort of phallic looking, and if there are only a few of the animals left in the world, it's a sure sign that their horns must be really really effective? Yeah, that's how Chinese medicine operates. It's almost entirely placebo effect, and ... shocking! ... Chinese people die of cancer every day.

Sorry but I'm going to have to cite Dara O'Brian

[Sycraft-fu]

"Oh herbal medicine has been around for thousands of years. Indeed it has and then we tested it all and the stuff that worked became 'medicine' and the rest of it is just a nice bowl of soup and some potpourri."

https://www.youtube.com/watch?...

Your statement about pot curing cancer also has to be one of the stupidest, most easily disproven thoughts I've seen in awhile. Turns out that when people get cancer often they need help managing pain and apatite and marijuana DOES help with those, so a good portion use it. Guess what? They don't get cured. I've had two people close to me who got cancer and died, both who use marijuana to manage symptoms.

You dumbass potheads do more harm to getting it legalized than any of your opponents could by making shit up. The more you lie about what it actually does, how it actually works and the actual risks (yes there are risks, everything has risks) the less people are going to listen to you about the real benefits.

Grow up.

Pharma development is hard and expensive

[hawkeyeMI]

Here's approximately how the drug development process works, to the best of my knowledge. I'm not in pharma but I've discussed some research projects with pharma folks and done a few projects on contract with them. I am not a pharmacologist or chemist. I am involved in medical device development and clinical trials for that purpose.

1. Pick a target condition (based upon need, existing treatments, size of market, whatever)
2. Based upon pharmacology, look at possible pathways to address said condition
3. Develop/find compounds that might address those pathways
4. Do whatever possible to narrow down these compounds by screening for safety before doing any trials, animals or otherwise. Select very carefully for screening techniques that won't give you false dangerous results becuase even if the compound is actually safe in the end, if you have a screen that looks bad at this stage the FDA is not going to like it down the road and it exposes you to liability.
5. Do animal trials with the promising compounds and hope some both work and don't cause harmful effects. Depending on the animal model this can get very expensive and time consuming.
6. Do human trials with promising compounds. A well-powered study will be VERY expensive (easily tens of millions of dollars or more), and depending on the condition being targeted, may also be very time consuming. Hope that what worked in animals works in humans, and no harmful effects crop up.
7. Assuming you make it through FDA approval, and they don't make you do more trials or enroll more patients before you get it, now you can produce the drug.
8. Try to sell enough of the drug to recoup your R&D expenses on ALL the compounds you checked out for the condition, all the trial expenses, etc, before the IP protection expires and the drug goes generic.

This "open source" model is neat and it may help a lot, especially in places where you can get away with less regulatory approval, but the way it's done is not because pharma companies are evil. It's because drug development is hard and expensive, and anything less than a blockbuster drug carries a high risk of never recouping the R&D expenses.

I think there's a lot of hubris to the idea that it can be done so much better this other way, but I will be happy to be proven wrong, because it really is a problem that needs solving.

Re:Pharma development is hard and expensive

[RandCraw]

I do work in pharma, and your sequence of steps sums up the process nicely.

I'd add that for every drug that succeeds, roughly another 20 fail, often after 5 or 10 years of development and costs incurred. That's why the estimated development cost of each new drug is widely acknowledged to be a minimum of $1 billion US (though most cite $2B as the norm). However after you include the cost of all failed drugs, the cost of producing each drug that succeeds effectively rises to between 4 and 5 billion. This is why each new drug needs to be a big selling blockbuster. It has many mouths to feed.

Obviously open software and volunteerism has their work cut out for them if they are to make drugs affordable. But I *would* be curious to know where their advocates believe these forces could have significant impact. It'd have to be in the clinical trial phase, where 80% of cost is incurred.

(BTW, to compute the net average cost of each new drug, you divide pharma company annual R&D budgets by the number of approved drugs/year. Matthew Herper of Forbes has covered this topic extensively, as has pharma chemist chemist Derek Lowe in his blog "In the Pipeline").

Re:Pharma development is hard and expensive

[Ungrounded Lightning]

Obviously open software and volunteerism has their work cut out for them if they are to make drugs affordable. But I *would* be curious to know where their advocates believe these forces could have significant impact. It'd have to be in the clinical trial phase, where 80% of cost is incurred.

For diseases with too small a community of sufferers to pay off that several-billion-dollar price tag, the alternative drug development/deployment system will have to cover all the steps, one way or another, because a pharma company would normally not be able to profitably perform the steps necessary to develop, test, obtain approval, and deploy. a new drug or treatment. Ditto for new uses of existing drugs or common compounds (such as DMSO) that are beyond IP protection. (Even if they COULD get IP protection on a new use, it would be virtually impossible to enforce.)

Big Pharma is out of the loop unless treating an "orphan disease" falls out as an off-label use of something new they've developed for a more pervasive condition, or there's big-time PR to be had.

Once omething like Open Source Pharma is up and running, Big Pharma companies might be able to harvest some PR benefits, and help out sufferers of orphan diseases, by announcing signs they find that some compound might help some particular orphan disease that they can't afford to pursue, and sign off on others proceeding further with it. That could help Open Source Pharma by relieving them of the costs of the initial search for possibilities (and also distract them from competing with the Big Pharma companies on potential new cash-cow drugs.)

Re:There's more to it than developing the drugs.

[currently_awake]

Reasons for government to do all drug research: #1-drug companies do research for profit only, unprofitable drugs don't get developed no matter how many lives it would save. #2-high cost and risk of developing new drugs. #3-developing a cure is less profitable than a treatment, so corporations would only make the treatment. The drug companies should only do production and distribution.

Re:This won't be allowed to happen

[Applehu Akbar]

Investigation of folk botanicals as new soutrces of medicine is routine in pharma and always has been. That's how willow bark tea became aspirin. How many liters of tea would be the equivalent of one 325mg pill, anyway?

Meanwhile, how many endangered species are your Chinese neighbors making disappear in their fruitless search for the elusive senior boner? Viagra saves species that Greenpeace can't be bothered with.

Re:This won't be allowed to happen

[ScentCone]

Let me guess. That person also works from home and makes $10,000 a month using one simple trick, right?

Re:This won't be allowed to happen

[the gnat]

The Chinese have known about ALL of these things for six thousand fucking years

And the Chinese also had the same god-awfully poor standard of living and short life expectancy as everyone else, until they adopted modern standards of sanitation, public health, and medical care.

Re:There's more to it than developing the drugs.

[ArmoredDragon]

Reasons for government to do all drug research: #1-drug companies do research for profit only, unprofitable drugs don't get developed no matter how many lives it would save. #2-high cost and risk of developing new drugs. #3-developing a cure is less profitable than a treatment, so corporations would only make the treatment. The drug companies should only do production and distribution.

Ok I'll need you to take off your rose tinted government issue glasses for a minute. Consider the fact that the US has a smaller GDP than the combined EU, and the EU governments take in more taxes than the US government.

Now ask yourself, why is it that the world's most advanced medications always come from for profit corporations in the US, and nowhere else? Why is it that the US is by far the most popular destination for medical tourism, even though in the US, hospitals are owned by private, for profit corporations?

Clearly because government run medicine is so much better, right?

Re:There's more to it than developing the drugs.

[davester666]

You only live those 40 years if you happen to be rich enough to afford it.

Re:There's more to it than developing the drugs.

[sjames]

No, you died because you couldn't afford it. Or you were left unnecessarily bankrupt and died years early because THAT kept you from getting decent healthcare.

There's profit and then there's obscene profiteering.

Re:There's more to it than developing the drugs.

[sjames]

Too bad you're wrong. India and Singapore are the hot medical destinations. Mexico is popular for Americans who need expensive dental work. There ARE people who travel to the U.S. for medical care, but more people travel FROM the U.S. to get medical care.

I have no idea where you got the idea that all of the drug development happens in the U.S.

Re:There's more to it than developing the drugs.

[sjames]

OH, you're a KOOK! You should have said so and saved me the trouble of replying.

Re:There's more to it than developing the drugs.

[sjames]

No, it should have been offered at an affordable price.

It's funny how they magically find a way to do that when the drug is at all optional.

Re: There's more to it than developing the drugs.

[koomba]

I think rtb kind of has a point, some medicine costs are just obscene, particularly in a hospital setting. My dad is an ER pharmacist, so I get to hear all kinds of stuff about insane drug prices. One example: someone came in recently that had been bitten by a dog, and they didn't know if it had rabies or not. He didn't have insurance. And with rabies, you can't just wait and see if symptoms appear, if you have contacted it, the antidote must be given long beefier symptoms appear, or you could be in trouble. And the price for the antidote paying with no insurance? $40,000. So hmmm, just pray the dog didn't have it? Blow 40k incase it did? No good choices there.

Re:There's more to it than developing the drugs.

[sackvillian]

Clearly because government run medicine is so much better, right?

The US pays its doctors some of the highest salaries in the world, publishes the most and best medical research in the world, and also charges its patients the most in the world.

You can find the best and worst care in the US. For the rich who want the best care -- American or not -- the US is their destination of choice. It's just that the rest of the developed world gives a damn about providing decent care to the vast majority of citizens who are not rich. By focusing on that, they take care of the rank and file and still leave the opportunity for the richest to travel abroad to pay through the nose for better care, so nobody really suffers.

And as the poster below points out, medical tourism is not exactly the best metric of your system's quality. India and Mexico aren't exactly shining models of medical care.

Re:There's more to it than developing the drugs.

[pepty]

#1. If the drug would theoretically save many lives (in the US) at all, it would be profitable. #2. So Congress, with its great respect for science, will be a great arbiter for deciding which drug research projects should get billions of dollars? #3. NO. This is a common fallacy. A cure for any serious ailment would be an absolute goldmine for a pharma. They would price it much higher than any treatment and drive out almost all competition in the market (the ones selling treatments, not cures) in months. Plus, they would get all of their revenue NOW, as opposed to having to sell it for a decade or more.

Want to book $10B in revenue in five years? invent a good diabetes treatment. Want book two trillion dollars in revenue? Invent a cure for type II diabetes. Seriously. It would have a stated price of $50k+, sell for an average price of ~$25K, and easily sell 80 million doses in five years.

Re:There's more to it than developing the drugs.

[pepty]

Year in, year out, about 25% of new drugs (NCEs) are invented by public research. 75% are invented by pharmas/biotechs.