Healthcare & Pharmaceutical June 2012

Fighting Rare Illnesses and Disorders

Despite some drugs being unprofitable as few people need them, it is our duty to help all patients

Pharmaceutical makers are in an excellent position to do a lot of good—and, I would argue, have something of a duty to do so—above and beyond merely developing and providing medicines that are going to cure illnesses and give people longer, happier and more productive lives.

One critically important area in which I believe the industry can play a part that will truly help a group of people who might otherwise be overlooked is in rare illnesses and disorders.

By their very nature, these diseases affect a small number of people and so, to create a drug that will treat their symptoms and, potentially, save lives is not always a profitable endeavour. But that does not, of course, mean that we should not go ahead and produce these drugs.

There is no universal definition of a rare disease, but it is generally accepted that such a disorder would affect less than one individual in 50,000 people. However, since there are an estimated 6,000 to 8,000 illnesses that are classified as rare in the EU, one person in every 17 people, or 6 to 8% of the population, could suffer from a rare disorder.

In Japan, as in other countries, there is a high variability in the number of patients with rare diseases, with some concerning only dozens of people, while others can go to 1,000 or up to 50,000 cases, which is the definition for orphan categorization in Japan.

Many rare illnesses are inherited and cause severe symptoms in the afflicted. They can result in children dying at a young age or suffering from debilitating disorders that mean they cannot lead a normal life or, in some cases, even move.

They can include complaints such as pulmonary arterial hypertension (PAH), a very rare form of hypertension that, if left unattended, can become life-threatening. But we are now able to present solutions whereby patients can live for many years with their condition.

It is a sobering statistic that, at present, only 1% of all known rare diseases has a cure. Yet, due to the small number of patients impacted by these illnesses, the drugs required to treat them are not always economically viable.

If all pharmaceutical firms were to base their decisions about whether to invest in the development of drugs on their respective corporate bottom lines, patients would undoubtedly be deprived of cures.

It is my conviction that we should develop remedies if and when there is a medical need that is not being met and we are in a position to deliver a cure.

Fortunately, that belief is shared by a growing number of global pharmaceutical makers, and discussion is increasing regarding what can be done to meet the needs of the significant sector of patients.

In February 2010, we established an independent global unit to tackle the issue of developing and delivering cures for rare diseases. In April of the same year, our Japan-based company set up a Rare Diseases Medicine Development Centre here to support the firm’s global activities.

Although it is unusual for a pharmaceutical company to dedicate resources to solving problems associated with rare diseases, we are committed to seeking innovative ways to meet medical needs currently not being met.

We have selected 200 rare diseases that we have committed ourselves to tackling, and for which we intend to provide cures. Our rare diseases unit may not be a huge division in terms of the number of people who work there, but it is staffed by some of the most talented and hard-working experts in their fields from around the world.

One of the advantages of the team, we believe, is that we are constantly seeking out new partnerships with regard to candidate compounds and technologies, so that we might form new alliances in various areas. With all these efforts combined, we aim to speed up delivery of innovative cures for rare disorders.

GSK has engaged into a capital investment and partnership with Japan Chemical Research, based in Kobe, in order to focus from Japan to the world on some of these diseases.