Orphan Drugs

It's a bit of an odd name, but basically an "orphan drug" is one that is used to treat a rare disease. by definition a rare disease is suffered by few people and this means that there is not the scope for pharmaceutical companies to make large profits through volume sales. Because of this, various countries have changed their law to allow companies to make more money out of the restrictive market.

The problems of the Orphan Drugs laws is highlighted by this letter in BMJ:

The original purpose of this legislation, passed in 1999, was to encourage drug companies to conduct research into rare diseases and develop novel treatments. However, as the rules are currently enacted, many drug companies merely address their efforts to licensing drugs that are already available rather than developing new treatments. Once a company has obtained a licence, the legislation then gives the company sole rights to supply the drug. This in turn allows the company to set an exorbitant price for this supply and effectively to bar previous suppliers of the unlicensed preparation from further production and distribution. 

The signatories give an example:

One example of the effect of these rules is the drug 3,4-diaminopyridine (3,4-DAP). We have been using 3,4-DAP for more than 20 years to treat two rare diseases, Lambert-Eaton myasthenic syndrome and congenital myasthenic syndrome; both cause disabling muscle weakness of the limbs, body, eyes, and face, together with swallowing and breathing problems, which can be fatal. The drug improves muscle strength and is used either because other treatments haven’t worked well enough or to avoid using drugs that can have serious side effects. Expert clinicians take the responsibility for informing patients about the drug and prescribing it. It has an excellent safety record. Until now 3,4-DAP has been produced by a small drug company on an unlicensed basis and costs between £800 (€945; $1285) and £1000 per patient per year.

The company BioMarin has now been issued with a licence to supply the drug (marketed as Firdapse) throughout Europe and has priced its product at £40 000 to £70 000 per patient per year—a 50-fold to 70-fold increase. BioMarin merely had to demonstrate that its drug works, using data generated from the unlicensed version. It has simply produced a slightly modified version (amifampridine) that meets regulatory standards and has been allowed to set the price at an exorbitant level with no clinically relevant advantage. 

The details sound very similar to those that I have given before on this blog about Avastin/Lucentis use for Wet Age Related Macular Degeneration, which Andrew Lansley uses as a model for his new Value Based Pricing scheme.

Understandably the clinicians are worried that this rampant profiteering by drug companies will have a detrimental effect on their patients. They point out that some PCTs refuse to pay the inflated prices, and presumably this will get worse when the budgets are handed over to GP consortia (who will have smaller risk pools than PCTs). Lists of Orphan Drugs can be found on the FDA website.

The signatories conclude:

Legislation on orphan drugs, far from encouraging the development of new treatments for orphan diseases, is severely limiting the availability of existing treatments. We believe that the Medicines and Healthcare Products Regulatory Agency and Department of Health should not just state the rules but should act now to progress the issue of unfairness upwards, so as to instigate change.

These figures are shocking and it seems that things are likely to get worse if Lansley has his way.