For the half-year to 30 June 2014, the IPKat's regular team is supplemented by contributions from guest bloggers Alberto Bellan, Darren Meale and Nadia Zegze.

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Wednesday, 29 February 2012

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?

8 comments:

Darren Smyth said...

The IPKat has received by e-mail this comment from Felix ROZANSKI of CEDIQUIFA http://www.cedi.org.ar/ (website mostly in Spanish), which he is posting with permission. Thank you Felix for your comment.

THANK YOU FOR YOUR REMINDER OF THE RAREDISEASE DAY.

1. MARKET EXCLUSIVITY IS AN IMPORTANT TOOL TO PROMOTE PHARMACEUTICAL RESEARCH IN THIS COMPLEX AREA. UNFORTUNATELY, MANY DEVELOPING COUNTRIES STILL DO NOT COUNT WITH R&D AND QUALITIVE INCENTIVES, AS THEY PREFER THE EASY ROAD OF INFRINGING IP.
TRIPs ARTICLE 39.3 ON HEALTH REGISTRATION DATA IS NOT OBSERVED.

2.PATENTS: TRIP's IS BEING INTERPRETED, IN MANY DEVELOPING COUNTRIES, AS REQUIRING ABSOLUTE WORLD NOVELTY, NOT ALLOWING PATENTS FOR NEW USES, THEREFORE EXCLUDING ANY NOVEL APPLICATION OF AN EXISTING COMPOUND.

THESE INCORRECT INTERPRETATIONS DO NOT PROMOTE R&D NOR QUALITY.THE COMPOUND EFFECTS OF BOTH WRONG TRIP's APPLICATIONS ARE EXAMPLES OF DISINCENTIVES TO R&D. THERE ARE MANY OTHERS.

CORDIALLY YOURS,

FELIX ROZANSKI
CEDIQUIFA

Anonymous said...

Orphan drug exclusivity is working very well and has been proven to do so. There are reports available somewhere on the great Net.

The issue of patent term for pharmaceuticals was the subject of a discussion on this blog several weeks ago. Unfortunately, due to the way the blog works, the discussion disappeared from view.

Such disappearance was, however, to the probable relief of those constantly slamming the pharmaceutical industry. Such critics were all unable to support their arguments and were completely unable to answer the points put forward by myself and others.

Chris Hayes said...

I wonder if it might be possible to use a system such as a petty patent or an innovation patent of limited duration and not-extendable by a PTE/SPC to support the development into specific areas of unmet need?

The regulatory agencies at a European could dictate for which areas should patents would be available.

As I see it the major advantage in such a case would be in the relative ease of enforcement of a patent compared to market exclusivity.

Chris Hayes said...

I wonder if it might be possible to use a system such as a petty patent or an innovation patent of limited duration and not-extendable by a PTE/SPC to support the development into specific areas of unmet need?

The regulatory agencies at a European could dictate for which areas should patents would be available.

As I see it the major advantage in such a case would be in the relative ease of enforcement of a patent compared to market exclusivity.

Anonymous said...

"Inadequate remuneration" under the old law involved mountains of proof and entire mountain ranges of money. You'd need something better than that.

Derek Freyberg said...

Although I'm also of a "patent-y disposition", I think that exclusivity is a better way to handle orphan diseases, as it starts on approval, making the issue of how much patent life has been used before approval irrelevant. And, contra Chris Hayes, exclusivity is self-enforcing from the point of view of the drug developer, since it is (at least in the US) a mandate to the health regulatory bodies not to approve a competitor's application. Not that this would prevent the developer having a patent, and enforcing it if necessary, if the patent would last beyond the exclusivity period.

Darren Smyth said...

TO Derek Freyberg - I think you have won me over in three sentences

Anonymous said...

Market exclusivity, however, only starts upon approval. A solid patent is required to protect any investment up to that point. This is why the combination of exclusivities available to the pharma industry is important.

The last thing anyone needs is a petty patent, so any such need in the pharmaceutical industry is on an immeasurably small scale. I am concerned such petty patents have been mentioned twice in just a few days, but I'm sure such ideas will go back into their box pretty soon.

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