Rare Diseases – are there sufficient incentives to develop treatments?

Today is RareDisease Day. Rare Disease Day is sponsored by EURORDIS, Rare Diseases Europe.

While anybody suffering from any illness may receive the sympathy of the entire Kat family, those afflicted by a rare illness must face a particularly difficult struggle.  A sufferer from a rare disease will likely have difficulty obtaining a correct diagnosis. Further, once a diagnosis is received, treatment options may be limited, if indeed there is any treatment available at all.

So what can be done to encourage the development of treatments for rare diseases? Judging from its website, EURORDIS takes the view that appealing simply to the altruism of pharmaceutical companies is unlikely to be effective.  This Kat would tend to also take that view.  Even though Monday was International Corporate Philanthropy Day, no company is going to invest money in research to treat an illness if there is no real prospect of a return.  Some degree of enlightened self-interest is necessary.

(A diversion on points of definition -  in Europe, a rare disease is one of low prevalence, generally one affecting less than 1 in 2000 people.  An orphan drug is a medicine for treating a rare disease).

Patent law sometime seems as immutable as the Laws of the Medes and the Persians.  It is not known for its flexibility.  In particular, the same standards for novelty and inventive step, and, most importantly for our purposes, the same patent term, apply equally to a blockbuster drug with a potential market of billions and to an orphan drug with the tiniest market.

The US was, to the knowledge of this Kat, the first country to introduce legislation to provide incentives for pharmaceutical companies to develop and market drugs to treat rare diseases, with the Orphan Drug Act of 1983.  The IPKat understands that this Act provides marketing exclusivity, tax credits, and fee waivers.

Within the EU, the Orphan Drug Regulation, which came into force in 2000, aims to provide incentives for pharmaceutical companies to research rare diseases by providing a 10 year market exclusivity period when such drugs are licensed.  During that period a generic version of the drug cannot be put on the market. Fee reductions are available for the marketing authorisation process.  Further, the regulatory process is likely to be far smoother when seeking approval in relation to a condition for which no treatment currently exists.

However this Kat, who is of a patent-y disposition, wonders whether the patent system itself can be used to provide an incentive to a pharmaceutical company to develop treatments with limited markets.

Many commentators, for many different reasons, have questioned whether a uniform patent term is correct given the hugely different industry models that the patent system attempts to support.  A uniform term is still with us probably because it is too difficult to try to assign different terms to different industry areas. Where would the lines be drawn? Who would decide? It is a great idea in theory, but how could a practical system be worked out?

This Kat’s feline ancestors told him that there used to be in UK patent law a concept of inadequate remuneration. Under the UK 1949 Patents Act, a patentee could apply to extend a patent term on the basis that it had not been adequately remunerated by the patent.

Could some system along these lines be worked out for orphan drugs? Would it be practical to have a system whereby, if the estimated or actual market for a particular drug was below a predetermined amount, then a patentee would be presumptively entitled to a certain extension of patent term? Would such a system give a pharmaceutical company the confidence to fund the research in the first place?  Commercially, certainty is usually prized above all.

These mew-sings on today’s theme are not new or revolutionary, and make use of established principles.  Do our dear readers have any comments?