A new campaign is trying to find parents for potential drugs, writes Dick Ahlstrom.
The Government stands accused of ignoring the plight of Irish orphans. It has taken no action on EU regulations, introduced a year ago, to encourage better treatment of them. These orphans are not parentless children, however, but potential therapies for rare diseases, as described in the EU's Orphan Medicinal Products (OMP) Regulation. OMPs are research discoveries that lack commercial sponsors, meaning potentially valuable treatments may never be brought into play to fight disease.
A meeting this week heard how orphan drugs might help to kick-start growth in the Republic's young biotechnology industry. "It is an awareness-raising meeting," said Michael Griffith of the research charity Fighting Blindness. "There is huge potential for OMP legislation. At a time when we are trying to promote biotech, this is an opportunity to do so."
The Irish Platform for Patients' Organisations, Science and Industry and IBEC put together Tuesday's meeting. The platform includes the Irish Research Charities Group, a body that includes Fighting Blindness, the Irish Cancer Society, the Irish Heart Foundation, the Cystic Fibrosis Association of Ireland and Asthma Society of Ireland. "An OMP is a potential therapy for a rare disorder," says Griffith. "Essentially, because it is a rare disorder, it is unlikely to become a commercial therapy unless there are special incentives [for companies and researchers]."
OMP legislation in the US, where it was introduced in 1983, has been a resounding success, he says. It has led to the introduction of 200 therapies for rare diseases and helped the fortunes of the companies that produced them. "There is evidence to show that many biotech companies have grown by producing OMPs," he says. An example is Genzyme Corporation, one of the world's largest biopharmaceuticals companies. Its vice- president for European corporate affairs, Dr Eric Tambuyzer, was a guest speaker at the meeting.
The new regulations have had very little impact in the EU, says Griffith, and no action has been taken in the Republic. This could work to our advantage if Irish biotech researchers and development companies got together to tackle diseases qualifying under the OMP regulations. "We have a chance of getting ahead of the posse," he says. To qualify for OMP status, a disease should have an incidence of less than five in 10,000 population. Estimates suggest there are between 5,000 and 8,000 such diseases in the human population, affecting more than 25 million Europeans.
The key to successful development is the incentives package that can tempt companies into areas where commercial returns might be limited, says Griffith. "The idea is to create certain protections for companies who would be willing to invest in an orphan product."
The US act included seven-year marketplace exclusivity and tax breaks, such as the ability to write off 75 per cent of research costs. The EU went for 10-year exclusivity but couldn't bring in similar tax measures as it has no control over national taxation policies. The assumption was that member states would evolve their own tax breaks, but this hasn't happened.
To qualify as an OMP the product must be genuinely new. Many of the rare diseases are caused by single gene mutations, so new therapies really are a possibility, and Irish scientists may already have discovered them. "I am told by scientists that there is a lot of intellectual property lying around the universities that has not been developed," he says. The meeting was an attempt to make people aware of the potential and move the Government towards developing OMP incentives.
"We see it as something that could push the whole therapeutic side of biotech," says Matt Moran, director of the Irish Bio-Industries Association, which represents the biotechnology industry.