 | Few pharmaceutical companies are as close to their patients as Shire’s HGT division, and this gives a special character and commitment to the work they do |
Shire has one of the richest and most exciting product pipelines in the biopharma sector, ranging from VYVANSE™ (lisdexamfetamine dimesylate), our next generation treatment for ADHD, and our ulcerative colitis drug LIALDA/MEZAVANT™ (mesalazine) to the new specialist medicines we’re developing for celiac disease and the treatment of scarring. These are the sorts of drugs that will continue to ensure Shire is successful, but there’s another aspect to our product development work, and one that goes to the heart of the unique character of Shire – the employees’ commitment to bringing medicines to patients who need them.
In our Human Genetic Therapies division, in particular, many of the treatments we develop are what’s known as ‘orphan drugs’. Drugs like this treat conditions that are so rare that it wouldn’t be cost-effective for companies to develop new therapies without external incentives. But even if these diseases are uncommon they can still have a devastating effect on individual patients and their carers. Hunter Syndrome affects only 1 in 100,000 children, and then only boys, but for those who do get it this is a terrible and life-limiting condition, and one that until recently had been untreatable.
But now at last there is some hope. ELAPRASE (idursulfase) was developed by our HGT team to replace the enzyme lacking in Hunter patients with a biologically generated form of the same chemical. We launched it in the US in 2006, and by January 2007 we were awaiting final approval from the EU to market it there. By that stage Shire was already in advanced discussions with authorities in Italy, Austria, Belgium, Spain and France to ensure that the drug could be made available as soon as possible after the official go-ahead came through. Many Hunter sufferers had already taken part in the clinical trials and the HGT team had come to know them and their families personally. Their absolute priority was to get these patients the one drug that could help them, and fast.
The efforts the team made were quite extraordinary. Our general manager in France began meeting key decision-makers even before she’d officially joined the company, and managed to secure patients early access to ELAPRASE through a special temporary authorization. In Belgium the team successfully appealed against the Ministry of Health’s initial refusal to pay for the drug, while in Austria we had to put together an early access program in a country that had never undertaken such an initiative before. As Mark Rothera, Shire HGT regional and marketing director, Europe, says “I’ve worked in this industry for 18 years and I’ve never seen such a remarkable example of a whole regional team working together with a single aim in mind: to do the best for patients. Most patients were able to get access to the drug anything from 3 to 12 months earlier than normal, and by the end of 2007 ELAPRASE was available in 12 EU countries and we’d exceeded market expectations by well over 20%.”
The same commitment is making itself felt in North and Latin America, where ELAPRASE has been available in the latter territory since mid 2007. Here Shire people are working hard to raise awareness of the disease and help patients get access to the drug. To cite only one example, it was only after seeing a newspaper article mentioning some of our material that one mother in Ohio was able to get the right diagnosis and treatment for her sons.
Across the world ELAPRASE is now approved for use in 34 countries, with new approval expected this year in Mexico. And in each new market our commitment will be the same: to bring these patients new hope.
CR Report 2007