Are drug companies not manufacturing certain cures?

[mrblue92]

From a conversation with a friend today:

“There are several medications that are not made for rare diseases that only children get…because they are not profitable and in our society, we can not force a business to make them. I just wish that I could remember some of the diseases…”

Does anyone know of such diseases? (I’m not so much concerned about juvenile disorders, either… any examples would be appreciated.)

Given the high cost of drug development in the first place, it seems unlikely they’d develop a drug they couldn’t market… Could these “cures” have come from independent or university research labs? I have to wonder if this is just an easy slam against the monolithic, greedy pharmaceuticals, or if there’s a grain of truth to it.

[mandielise]

Yes, actually I have a friend with PK (he’s phenylkenoric - I know I spelled it wrong). Basicall, someone with PK can’t eat any type of protein: dairy, meat, beans, nuts, etc… They’re allergic to one specific amino acid and since amino acids are the building blocks of all proteins, they have to eat a VERY small amount of protein a day.

Most people with this disease die young - in fact, my friend is the oldest person with this problem in Louisiana, and he’s only 22! Since the body needs protein to build muscles and cells, and to perform most chemical reactions, a substitute has been manufactured which contains all the necessary amino acids other than phenylaline, they one they can’t have. My friend has to drink this stuff (which appears like milk, but I’m told it tastes MUCH worse) twice a day. Well, they just stopped making it because of costs.

There IS a substitute that my friend will have to take, but it will cost a lot more, taste much worse, and really mess with his system. Imagine taking the same exact medicine twice a day every day for your entire life in the place of eating much of anything. Then one day, they take it away from you and tell you to adapt to something else. My friend’s doctor said this could really hurt him, but there’s nothing they can do cause the drug companies are greedy b*stards!

Most drugs in America are invented, if you will, through tax payers money. The Government uses our money to create these drugs, then hands them off to private companies for free. These companies charge substantially more to Americans than they’d dream of charging in any other country in the world (why do you think I get some of my stuff from Canada?). They didn’t even create the stuff, but they rob us blind! It’s ridiculous!.. IMHO of course!

[astorian]

Do a google search on the phrase “Orphan Drugs,” and you’ll find out all there is to know about the subject.

To oversimplify, there are certain drugs that are very expensive to make, and which benefit a very small number of people. Sometimes, those very few people need the drugs desperately (or stand to beneit tremendously), but there’s no way for anyone to manufacture them at a profit… or even at an acceptable loss.

[Kamandi]

*Originally posted by mandielise *
**Most drugs in America are invented, if you will, through tax payers money. The Government uses our money to create these drugs, then hands them off to private companies for free. These companies charge substantially more to Americans than they’d dream of charging in any other country in the world (why do you think I get some of my stuff from Canada?). They didn’t even create the stuff, but they rob us blind! It’s ridiculous!.. IMHO of course! **

Can we get a cite for this?

“IMHO of course!” does not cut it in General Questions.

[Boyo_Jim]

*Originally posted by mandielise *
**…Most drugs in America are invented, if you will, through tax payers money. The Government uses our money to create these drugs, then hands them off to private companies for free. These companies charge substantially more to Americans than they’d dream of charging in any other country in the world (why do you think I get some of my stuff from Canada?). They didn’t even create the stuff, but they rob us blind! It’s ridiculous!.. IMHO of course! **

That’s just not true… except for the part about them robbing us blind, that is.

I’d like to point out the some of these threads are using the word “cure” instead of a more correct term - “treatment”. If these orphan drugs “cured” people, they wouldn’t need to continue taking the drug. It’s an important distinction to keep in mind when considering the economics of manufacturing these drugs.

[Yarster]

Former drug discovery researcher checking in:

I used to work at a company called Ligand Pharmaceuticals, a public company (NASDAQ: LGND) that has a handful of drugs on the market used to treat obscure forms of leukemia and rare skin lesions. As someone who worked in drug discovery, on a weekly basis, we came across numerous ‘leads’ in our screenings against all types of receptors.

Depending on who you talk to, about 1 in 10,000 to 1 in 50,000 will make it to the pharmacy shelf as a real drug after 10+ years and several $100 million dollars. All things being equal, no one compound is likely to exceed, and the longer it takes to fail in studies, the more it will cost you. Therefore, it only makes sense to go after the biggest target markets.

It’s not like we test a chemical compound and instantly know it’s a cure for some obscure disease but suppress that knowledge. We just don’t screen against receptors for obscure diseases to begin with because it’s a losing proposition from the beginning in most cases. All of Ligand’s drugs started out as potential candidates for big money targets like breast cancer, acne, psorasis, and other such conditions, but as you go through animal trials, you realize it treats a specific form of the disease better, so in a way you back into this specialty, rather than targeting it from the start.

We regularly found whole classes of compounds we couldn’t use that didn’t match any of the biological targets we were interested in at our company. That said, we then outlicensed them to other companies so we could still make money on them.

I don’t believe anyone is suppressing a true ‘cure’ for any disease. The Orphan Drug Act does encourage pharmaceutical companies to test for more obscure drugs by giving them a longer patent for those they develop. That said, however, I still think drugs that do come out in this category find their way there by serendipity rather than planning.

And I wouldn’t blame the pharmaceutical companies too much there. They get their money from venture capitalists and the public just like every other business. If someone came to you as an investor and said, you have two choices: you can fund a project with a 10% chance of success that if it succeeds makes $1 Billion/ yr or a 10% chance of success but will only make $250,000/ yr. which would you choose. Yep, you pick breat cancer over Wilson’s Disease every day of the week and twice on Sunday.

[pldennison]

Most drugs in America are invented, if you will, through tax payers money. The Government uses our money to create these drugs, then hands them off to private companies for free.

Nothing in either of these two sentences is true.

[Nametag]

Government research is not given away free. From an NIH Website:

Federal Technology Transfer Act of 1986, PL 99-502
The Act amends the Stevenson-Wydler Act by codifying a number of changes that impact on the technology transfer process such as (1) scientists and engineers are now responsible for, and evaluated their abilities to get technology transferred out of the laboratory. (2) Inventors from government owned and operated laboratories (GOGO) are required to receive a minimum of a 15% share of any royalties generated through patenting or licensing…

[WV_Woman]

I grew up with a kid who had a really really weird, rare disease. (I never did know the name of it but it has something to do with his body not making an important enzyme.) This thing is so rare that like, maybe 40 people in the world have it.

Anyhow, he gets his meds for free from the company that makes it.

[mrblue92]

Well, had she given me the phrase “orphan drugs”, I might have saved you guys the trouble… but it’s always interesting to see the different perspectives. (And yes, “treatment” is definitely a better word than “cure”…)

FYI, the section of the FDA that is concerned with this particular area is called the Office of Orphan Products Development (OOPD). So in the case of these relatively unpopular drugs (presumably ones you couldn’t justify building even a small factory for), it seems likely that gov’t money is paying for most of the research and subsiding production. Oddly enough, I suggested to my friend that subsidies seemed like a good idea before I got the responses…

From the FDA’s site:

Since it was created in 1982, the OOPD has been dedicated to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. To locate such products, the OOPD interacts with the medical and research communities, professional organizations, academia, and the pharmaceutical industry, as well as rare disease groups. The OOPD administers the major provisions of the Orphan Drug Act (ODA) which provide incentives for sponsors to develop products for rare diseases. The ODA has been very successful - more than 200 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than ten such products come to market. In addition, the OOPD administers the Orphan Products Grants Program which provides funding for clinical research in rare diseases.

Thanks, everybody.