Let me say up front, this may be the most contentious blog I’ve written. So, beware.

The definition of an orphan drug is that the market for said drug is less than 200,000 patients. This number of patients is considered to be not sufficient to be financially successful for drug companies to develop drugs. Orphan drug status means that one FDA designated company can develop and market specific drug targets for those patients. See; http://www.fda.gov/orphan/oda.htm for more information.

There are however lots of drugs that are still not being developed. I think we need a new definition of orphan drug to be able to get more useful drugs to the patients who need them. The solution may not be just increasing the size of the target market, but might be to extend the life of the drug. This could be achieved by increasing the length of time that a patent is valid. Alternatively the FDA could designate a drug an orphan drug to protect it for a longer period of time. They could choose to not allow copycat drugs to enter the market. One might argue that the copycat drugs are generics and the generics are just as good as the brand name but cheaper. Yes, that may be true for drugs already on the market. But what is happening is that companies chose not to take compounds to market because of the perceived risk of competition from generics.

I am not proposing a monopoly for any company or drug, and am in favor of a free market economy, but I am also in favor of providing opportunities to enhance the health of our nation. The current market is not treating the general population adequately and these suggestions can improve things.

What if a new indication of a drug about to go out of patent was discovered? The drug company would NOT try to develop the drug for a new treatment because it would cost time and money that would not be recouped. Further, scientists and physicians often do not want to do the years of research needed to get the drug approved for a new indication. So the treatment remains unused and is thereby orphaned.

Yes drug companies will make more money but more drugs for underrepresented diseases can be developed. In the example of a drug coming off patent, the company could be allowed appeal to the FDA to extend the orphan drug status of a previously developed drug and make it profitable for the company to do the development needed to get a new application for that drug. There are many cases where it looks like a drug might have a second application but it is not investigated by drug companies because the patent is dying.

Sub categories of such diseased such as wake up stroke or drugs targeted to be in ambulances or for disasters could be better developed. Drugs in ambulances can expire or go bad because of heat and cold cycles and that means they are not used or have drastically decreased profits. Disaster drugs often sit unused and therefore new ones may not be developed because of a weak market.

Numerous examples where drugs could be developed but companies choose to not go that route can be found. The federal government does not make drugs and cannot mandate a drug company to make and market a drug. Therefore companies need to have incentives to develop drugs for less profitable arenas.

Solutions to these problems do not need to cost the taxpayers’ money, but rather allow companies in these cases to be able to recoup their expenses with these drug development projects. Solutions could be:

1. Extend patent life.
2. Protect from or decrease costs of litigation.
3. If a new indication for a drug is discovered extend patent life for that drug. Especially for that indication. The FDA would need to approve generics for the old indication but forbid generics to be used for the new indication.
4. Restrict new drugs that unfairly compete for a period of time.
5. Optimize regulatory steps such as FDA required trials, reimbursement, or accelerate the process and allow longer periods of time for marketing.

I realize that I have not explained number 5 above. What I mean is that it takes the average drug 10 to 15 years to get to market. That is a huge expense and with a patent only being valid for 20 years the period of time that a drug can make money is very short. So if drugs can reach the market in 5 to 10 years, that is more than enough time to prove safe and effective and will increase the time where money can be made off that drug.

For anyone who is offended or concerned by all the discussion of profitability for drug companies please ask yourself one question. Who else is going to make and sell drugs? These are companies and this is a free market society. If you want more and better drugs than make it appealing for drug companies to get them to you. Pharmaceutical companies are not charities and charities are not manufacturers of drugs. But we have sadly legislated pharmaceutical companies out of business – almost. It takes one billion dollars to get a drug to market with only about 5 years of marketing for that drug to break even. What product makes $200,000,000 per year for the first five years after its launch? The answer is not much, but that is what is needed for a new FDA approved drug.

Our current system is broken and tainted. The result is helpful therapies are not being made, sold or developed to treat treatable diseases.

In the interest of full disclosure let me say that I am a Professor of Neurology at the University of Cincinnati. My research is into the causes, treatments and diagnosis of stroke and related neurologic emergencies. I am one of the founders of a very small biotech company that is working to make diagnostic devices for stroke patients. Recently, I wrote and published a book entitled, My Ambulance Education. A huge theme in the book is “helping people” and that theme is the desired endpoint of the blog above.