Development of novel drugs is truly like no other business: one attempts to address questions that may have never been previously posed – let alone answered – in the pursuit of improving the lives and quality of life of patients. As I eluded to in my recent letter to shareholders, the team is now well into the analysis of results from our erythropoietic protoporphyria (EPP) program; two studies from the US and Europe. This is a complex and time consuming task that requires one to collate and make sense of thousands of data points to answer a seemingly straight forward question: does this trial show that the drug is safe and effective?
Obtaining an answer needs to be understood from the concept of clinical relevance. Put simply, results don’t just need to show that a treatment or intervention has an effect on a disease. Rather, they need to indicate that that effect is relevant to the current clinical understanding, treatment and care for the disease or indication. They need to show that the drug’s effect is having a positive, meaningful impact upon a patient’s prognosis and care. This is a crucial point to consider in the development of protocols and in the careful analysis of results, as it is how regulators will review the results.
Later this year, pending clinical results, we intend to file SCENESSE® (afamelanotide) with the European Medicines Agency (EMA) for marketing authorisation approval (MAA). If successful, this will allow us to make the drug available across Europe. The team’s current work is focused on building a dossier of information which will allow the EMA to objectively review all data on the drug’s safety and efficacy as a prophylactic treatment for EPP.
In analysis, however, numbers are only half the work. One must employ lateral thinking to determine the relevant outcome of a study, followed by a discussion with the relevant medical community to challenge and validate the study results.
Rare diseases are often poorly understood, even in the medical community, and very seldom seen outside of specialist centres. Here clinical relevance is of even greater value as traditional study endpoints are generally of little value and unique, disease specific, endpoints must be developed, quantified and employed. Looking back at when we commenced our EPP program in 2006, we had to first work with the community of patients, physicians and regulators to develop and validate these endpoints before embarking on an expanded clinical program.
In short, the quantitative objective of our EPP studies is to determine whether the drug can prevent or reduce the incidence and severity of reactions in EPP, caused predominantly by light around 408nm in wavelength (blue spectrum). From a clinical relevance perspective, however, we also aim to be able to show whether the drug improves the patients’ quality of life, allows them to expose their skin to sunlight for extended periods of time or alters the UV/light avoidance behaviour ingrained in them since childhood (including so-called ‘shadow jumping’). The addition of objective clinical measurements – such as phototesting – provides further numerical support.
These factors will form the basis of our understanding of whether SCENESSE® provides a benefit to our patients and has a positive and measurable impact on their lives. In the coming weeks we will learn the results from this program and ideally be able to confirm the clinical relevance of the first prophylactic treatment in EPP.
