Shock and Awe Drug Pricing Gives Visibility for Rare and Neglected Diseases

If rare, neglected, and for that matter tropical diseases, had a hard time getting visibility in the media, that changed almost overnight with the announcement By Turing Pharmaceuticals of their 5000% price increase for the toxoplasmosis drug they own. The disease occurs widely in the population as well as in HIV/ AIDS patients, as those with a compromised immune system are more susceptible to the parasite.  What follows is a classic scenario as played out by their CEO — company buys drug that was apparently underpriced, unprofitable and takes it into the price stratosphere in line with other rare disease drugs which costs hundreds of thousands of dollars per year for life. Except there are issues that everyone screaming at the CEO fail to understand. This drug is used for a relatively short term (months) and is a complete cure in approximately 80% of patients. Those are very impressive numbers and compared to burden of taking an expensive orphan drug for an entire life time that is common with most rare diseases, Turing’s drug is a very good deal.

Certainly Turing did not go about their decision in a particularly public relations focused manner. Many critics have also focused on the past decisions, social media presence and generally “non-classic CEO behavior”. But then we live in an age were virtually all big pharmas have been fined hundreds to millions of dollars for criminally unethical behavior on a scale that makes the Turing price raise seem like child blowing a raspberry. All perhaps are missing that this type of price increase is not unique. It happened long before Turing and will happen long after. For example Rodelis Therapeutics increased the price of the tuberculosis drug cycloserine 2000% before they were asked to hand it back to the Purdue Research Foundation. The fact that drug companies are clearing their decks of old drugs and clinical candidates is no different to what has been going on for decades. The only difference is that there is a receptive rare disease market, where some companies have justified the rarity of their disease with prices that are more than most people spend on a house.

There have long been calls for transparency on orphan disease drug pricing with many drugs in six figures. But rather than be seen as apologists for Turing we should perhaps take a wider aim and consider that this is indeed an industry wide issue. The behavior of Turing just made everyone take notice and perhaps that was their strategy. Most do not understand what it takes to develop, manufacture and sell a drug. Even a company that buys a drug others have made even 50-60 yrs ago have to continue to ensure it is made to the standards required by the FDA. That alone has a cost which must be recouped before you even think about adding a profit to it for R&D for ongoing work. Even if Turing were to start their own research program they would likely spend many years and hundreds of millions of dollars to a billion dollars or more developing a molecule that stands a small chance of becoming a drug. The general public do not understand what it takes to get a drug to market. Even a repurposed drug, with a new use patent can now have new value above its generic price. The types of me-too drug development we saw in drug companies in the 1970’s buried the idea for once and for all that the industry could be called “ethical’, price hikes like this are no different. We live in an age where drugs cost money, because research costs money. Most pharmaceutical companies are public companies with shareholders and behave identically. Few companies really consider the implications of what they are doing. There are exceptions and they tend to be led by people affected by diseases or who have made it their lives work to change the status quo.

Time will tell if Turing and its CEO will really have a deep and meaningful effect beyond this news cycle but we have lacked a rare disease news moment this year to compete with the ALS challenge of 2014. The very least they could do is shine a positive light on what the challenges are for rare disease R&D and the daily struggles of patients and their families. We have to take this opportunity with both hands. I waded into social media to make some of these points, not to defend but to set the record straight. The CEO was saying things that were not out of place with language from any other pharma CEO except he was being direct, no sugar coating. That may be seen as a weakness, it could be lack of experience on the world stage or just bad advice. We all learn. Unfortunately the twitterverse associates any serious scientific opinion with opposition. When you point out to them what the challenges are for drug discovery in rare diseases then I think people perhaps appreciate that there are bigger problems we need to focus on. There are over 7000 rare diseases but only a few hundred of them have  treatments. That is not something to be proud of and we still have a long way to go. Each time I give a talk, write on Twitter or post a blog, there is a chance that another rare disease parent or family member will reach out and ask for help. If only we could turn all those people with fiery opinions on Twitter and elsewhere into advocates for rare diseases who would donate some time to helping raise money, awareness or even doing research. Then perhaps these screaming protests will have started a movement that is much bigger than a Turing or other small company.

Can we get a 5000% increase in rare disease awareness?
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