Pharmaceutical development is undergoing a defining shift following the ever-increasing regulatory rigor in the process of evaluating a new product. Our recent announcement reflects the conservative view we hold on 21st century drug development.
During development the innovator often seeks to create a market for the new product, a common concept applicable to various industries. The same holds true for afamelanotide: we identified and developed our lead product in an under-served market of porphyria. Adaptation to chemistry and technology, novel communication platforms, deep knowledge and relationships with academia facilitated today’s entry to market.
Today’s EPP (porphyria) preliminary results mark a significant step in afamelanotide’s 19 years of development.
Clinical trials are complicated and often the results can be even more so. Close monitoring of the interactions between patients, diseases, conditions and drugs for extended periods of time and the various questions posed in order to prove or disprove safety and efficacy of particular treatments result in an accurate yet highly complex mountain of data.
As many of you may know, this week saw Clinuvel hold its Annual General Meeting in the Melbourne Town Hall. As managing director I welcome these opportunities where I am able to meet and talk to the people whom Clinuvel considers integral to the company and its existence; the Board of Directors sees Clinuvel’s investors as unique. In Clinuvel, an investor has to fundamentally believe in the premise of offering pharmaceutical photoprotection, a novel option, an innovative approach to skin disorders.
In January 1983 the United States government passed an Act that has had a profound impact on pharmaceutical development and the individuals whom it was intended to benefit. The Orphan Drug Act (ODA) was lobbied for successfully by the National Organisation for Rare Disorders (NORD) and provides incentives for researching and developing treatments for rare or ‘orphan’ diseases.
Allergan’s lawsuit filed against the FDA presents a relatively novel case in addressing the requirements of drug developers in relation to off-label use of drugs by physicians.
Today, and in the coming weeks I shall review the increasingly complex phenomenon in the pharmaceutical industry called off-label drug use. 
