Clinical trials are research studies which involve people, with the aim of finding new treatment or procedures for specific illnesses.
There are several phases to a clinical trial, which are carried out after a new drug has been licensed. They collect information about safety, side effects and long-term benefits and risks associated with the drug. The development of any drug is very difficult and extremely expensive, but developing a drug for a rare disease (orphan drug) is very complex.
Because there are only a small number of patients, it is harder to recruit people for these clinical trials and information can take a lot longer to collect and collate. In some instances it could take up to 10 years to complete a trial and retaining investment for that length of time is not easy, but overall can offer the drug makers a significant reward.
One drug company has set up centres in five different countries, using bridging studies such as http://www.richmondpharmacology.com/specialist-services/bridging.php in order to recruit enough people to assist with a trial to help treat Hunters disease. Because these groups are so small, their relationship is a lot closer than would be the case in a larger programme. Drug companies, therefore have to work with the test groups and have a quick response to their needs.
Patient support groups with members suffering a rare disease are happy with the progress being made. Drug companies have listened to the people and have taken on board the needs of the patients. Their main problem is the length of time it takes to diagnose a patient who has a rare disease. A study revealed that only one in ten patients with a rare disease receives specific treatment relating to their disease, and the rarer the disease the longer it takes to diagnose.
The introduction of the 1983 Orphan Drug Act has encouraged a large number of biotech companies to develop orphan drugs. The act helped to speed up regulatory approval and provided protection from competitors. The financial implications of finding a new orphan drug are immense, with global sales of orphan drugs expecting to grow more than 11% per year. This will account for more than a fifth of the total drug revenues within the next five years.