Dr. Lowe, from In the Pipeline, writes an update on a story we covered earlier this year:
You may recall Marathon Pharmaceuticals, the small company that announced plans to sell a long-used steroid treatment (Emflaza, deflazacort) in the US to Duchenne muscular dystrophy patients. The price was set to go up steeply, since the company was awarded years of market exclusivity by the FDA (under their program to reward orphan-drug indications like this one).
This business model is the same one followed by a number of other small outfits (see that link above for more), and it's infuriating. Generic drugs are off patent, by definition, and they're supposed to be cheap. Taking advantage of regulatory loopholes and perverse incentives to jack their prices up is shameful, unproductive, and expensive.
[...] Marathon itself appears to be about to disappear. And why not? They've turned a quick buck. Endpts, who have been doing a great job on this story, couldn't find anyone who thought that the company had spent more than $70 million on the drug's approval, and it was probably a lot less. So $140 million, plus milestones and royalties, is a nice return. But there's more money coming than just that – the company got a priority review voucher from the FDA for bringing a rare pediatric disease drug to the market, and they can sell that on the open market. I'd guess that it could bring in another $100 million or so
http://blogs.sciencemag.org/pipeline/archives/2017/04/25/marathon-pharmaceuticals-cashes-out
https://en.wikipedia.org/wiki/Deflazacort
The priority review voucher is an incentive program that provides companies, that get a drug approval for a neglected or rare disease, a voucher that entitles them to a more speedy review (under six months instead of ~10 months) for a future drug. This is a great program; however, Marathon Pharmaceuticals deliberately exploited this program by conducting a very small trial using a generic drug (another loophole).
Previous Story: https://soylentnews.org/article.pl?sid=17/02/11/2116252
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Dr. Derek Lowe, from In the Pipeline, writes:
So since drug pricing and FDA regulations are so much in the news, it would seem like the perfect time for a small company to game the system for big profits, right? That's apparently what Marathon Pharmaceuticals believes. They just got approval for deflazacort, a steroid, as a treatment for Duchenne Muscular Dystrophy.
[...] So what's not to like? Well, this drug has been around since the early 1990s. Marathon most certainly did not invent it. Nor did they think of applying it to DMD patients – the biggest clinical trial of the drug for that indication was done over twenty years ago, by someone else. DMD patients in the US were already taking the (unapproved) drug by importing it from Canada. Marathon just dug through the data again and ran a trial in 29 patients themselves, from what I can see. I should note that this is not any sort of cure, nor does it address the underlying pathology of the disease. The steroid treatment makes muscle strength in DMD patients stronger – barely. But even for that benefit, US patients will now have to get it from Marathon at something like 50 to 100 times the former price.
[...] So while I defend the FDA's function of making it tough on new drugs (making them prove safety and efficacy), I cannot stand how loose they are with old generic compounds. The agency hands out extremely valuable rewards like lollipops in these cases – a priority review voucher can be sold for hundreds of millions of dollars
[...] And they're also allowing the likes of Marathon to make the rest of the drug industry look like greedy sociopaths. Marathon, Catalyst, T*ring and all the rest of the people who are pulling these tricks have the word "Pharmaceuticals" in their name, but they are not drug companies. They discover nothing. They do no research. They take virtually no risks. They exist only to play legal games and watch the money roll in.
[...] As for the FDA, the agency probably can't change this on its own, though, even if it wants to – Congress has to act to give them the authority to deny market exclusivity or priority review vouchers under some conditions. Either that, or we should rethink these incentives entirely, because they are (clearly) too easy to exploit for fast bucks.
https://en.wikipedia.org/wiki/Deflazacort
Also at ArsTechnica.
A new and non-viral approach to CRISPR has been used to treat Duchenne muscular dystrophy in mice:
A new version of the CRISPR-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed.
Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups, according to a press release.
The study, "Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair," [DOI: 10.1038/s41551-017-0137-2] [DX] was published in the journal Nature Biomedical Engineering.
[...] Unfortunately, methods of delivering the components of this system, which include an RNA molecule called a guide RNA, a protein called the Cas9 nuclease, and the correct DNA sequence to replace the mutation (via donor DNA), have not been fully developed for human use. A primary technique used to deliver the components of this system relies on viruses, but this technique is plagued by complications and unwanted side effects.
In response, researchers at the University of California, Berkeley have developed a new approach called CRISPR-Gold, which used gold nanoparticles to deliver the components of this system in a mouse model of DMD. This method works by using gold nanoparticles to coat a modified DNA molecule that binds the donor DNA, which in turn is bound to Cas9 and the guide RNA.
This entire system is then coated by a polymer that will interact with a cell membrane and allow entry into a cell. Then, the components of the system are released into the cell as the coat breaks apart upon entry. The guide RNA, the Cas9 nuclease, and the donor DNA can then make their way into the nucleus and correct the mutation.
Also at TheScientist.
Previously: FDA Panel Recommends Rejection of Duchenne Muscular Dystrophy Treatment
Marathon Pharmaceuticals is Part of the Problem
Marathon Pharmaceuticals Cashes Out on Regulatory Loopholes
What is a Muscle Protein Doing in the Brain?
(Score: 2, Disagree) by bradley13 on Wednesday April 26 2017, @05:08PM (6 children)
This is a poster-child example for crony capitalism, i.e., undeserved profits made possible through bought-and-paid-for government regulation.
Less government would be more.
Everyone is somebody else's weirdo.
(Score: 2) by Grishnakh on Wednesday April 26 2017, @05:55PM (1 child)
Government regulation seems to work pretty well overall in western Europe; we never see stories like this over there, and drug prices are really cheap both there and in neighboring Canada.
So why is it the government regulation in the US usually results in crap like this?
(Score: 2) by kaszz on Wednesday April 26 2017, @05:57PM
So why is it the government regulation in the US usually results in crap like this?
Answer: Who pays for politicians? :-)
(Score: 3, Insightful) by DannyB on Wednesday April 26 2017, @05:56PM
How about: Less government CORRUPTION. Government exists to regulate. The problem isn't the regulation so much as the corruption, the crony capitalism that you point out. I'm for less government, as long as it can regulate abusive behavior.
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(Score: 3, Informative) by Joe on Wednesday April 26 2017, @06:08PM
The program was developed to address a very real problem with market-based solutions for drug development. The problem is obvious: the market will only drive development of profitable drugs and will neglect diseases that are either rare (e.g. muscular dystrophy, ALS, Huntington's) or only prevalent in populations that are poor (e.g. dengue, Chagas, zika, chikungunya).
To help address this problem, some professors (from the economics and business departments of Duke University) proposed the priority review system as a subtle way to make developing orphan drugs more profitable to the market by (in a way) coupling their success with that of blockbuster drugs. This program uses the market to set the price of priority review vouchers and it does not increase regulations or review time for any other drugs.
TL/DR: Unprofitable diseases become more profitable (better for neglected patients), blockbuster drugs get to market earlier (better for patients), and no increased regulatory burden.
The loophole, which needs to be fixed, that was exploited in this case was that generic drugs from other countries can qualify if they can be applied to new indications in the US (after acquiring FDA approval). What Marathon Pharmaceuticals did (run a clinical trial and get FDA approval for the drug) is worth something, but it really shouldn't have qualified for the program.
http://content.healthaffairs.org/content/25/2/313.abstract [healthaffairs.org]
https://en.wikipedia.org/wiki/Food_and_Drug_Administration_Amendments_Act_of_2007#Title_XI:_Other_Provisions [wikipedia.org]
https://en.wikipedia.org/wiki/Orphan_Drug_Act_of_1983 [wikipedia.org]
https://en.wikipedia.org/wiki/Neglected_tropical_diseases [wikipedia.org]
- Joe
(Score: 3, Insightful) by Azuma Hazuki on Wednesday April 26 2017, @07:59PM
Oh sure, and by that metric, because some people use guns to commit murders, the solution is less guns, riiiiiiiiiight? :D
THINK before you say something like that. Regulations don't kill people, regulatory capture does.
I am "that girl" your mother warned you about...
(Score: 1) by DannyB on Wednesday April 26 2017, @08:15PM
It is not 'govmint'. It's 'gubmit'. A noun. Here is an example of the proper use of the word in a sentence:
Dem gubmit foaks shore is ignert.
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(Score: 2) by bob_super on Wednesday April 26 2017, @05:50PM (2 children)
My idiotic parents taught me to work hard and be fair.
The news keep telling me about assholes raking in more dough than I'll ever have, by screwing everyone they can and getting away with it on technicalities.
What a world, what a world...
(Score: 2) by DannyB on Wednesday April 26 2017, @05:59PM
Yep. Play by the rules. Get screwed. Government is not doing its job. And IMO it cannot be fixed. It will spiral out of control. It's not what I want. I simply believe it is inevitable. I am able to separate what I want from what I think is going to actually happen. Not a pessimist. And God Forbid not an Optimist! But a Realist.
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(Score: 0) by Anonymous Coward on Wednesday April 26 2017, @06:03PM
Yeah, same here. My parents were big fans of this "real" world where that sort of thing would pay off.
If I were still on speaking terms with them, I'd love to hear what they had to say about the "real" world where shit like this happens.
In fact, if anybody knows how to get to this "real" world, I'd love to know how.