Online evolution

Thursday, December 15th, 2011

Earlier this week we unveiled a major update to our online presence with the release of our new-look website and the launch of Mothers & Children, a new site focusing on the pediatric and parental skincare.

Over the past five years the team has worked to continuously improve our online resources, expand into social media and build our presence on our Youtube channel (generating over 100,000 views in the process). The new site, Mothers & Children, is a direct response to what we saw as a key gap in online skincare information: up-to-date, accurate and concise information on caring for and protecting young and maternal skin. This is a new space for us and we’re excited to be able to compliment our extensive Science of Skin resources with this site.

We’re not standing still online. You will notice further changes in the coming months as they emerge from internal development and testing as well as new content being developed across the site.

Let us know your thoughts about our new site via Twitter, Facebook or email.

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EPP results and clinical relevance

Friday, November 4th, 2011

A protoporphyrin IX molecule

Over recent months I have written several times of the need for Clinuvel to prove clinical relevance in our trials with the use of SCENESSE® (afamelanotide) in erythropoietic protoporphyria (EPP). In orphan populations the need to demonstrate how a novel drug assists in their daily activities and improve their lives is at the forefront of the regulators’ minds. And so it should be, after all the objective of the pharmaceutical industry is to develop drugs which address either disease or symptoms adequately and safely. The results the company released yesterday from our Phase II US study of the drug in EPP (CUV030) have given us important data towards demonstrating clinically relevant improvement of patients’ lives. (more…)

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Clinical relevance – the value of patient experiences

Friday, September 30th, 2011

Since 2006 Clinuvel has trialed SCENESSE® in a truly unique group of individuals: patients living with erythropoietic protoporphyria (EPP), a rare genetic blood disorder which causes an absolute intolerance to light.

EPP prevents patients from leading ‘normal’ lives, especially outdoors. It is one of the few diseases that manifest clinically with initially invisible symptoms which cause severe dermal pain for several days. This not only presents a challenge for diagnosis and treatment, but also for generating meaningful clinical trial results – those which are measurable numerically and are used by regulatory authorities to evaluate the efficacy of a drug in a patient population. Here, real life patient experiences during a trial can play an important role in providing clinical relevance and analysing hard data. (more…)

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The importance of clinical relevance

Tuesday, August 2nd, 2011

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. (more…)

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Porphyrias: a disease grouping by cause, not symptoms

Monday, April 18th, 2011

Held biennially, the Porphyrins & Porphyrias conference (P&P) is the world’s largest gathering on the porphyrias – a group of metabolic disorders causing biochemical disruptions in the pathway of the body which synthesizes haem (heme).

As a result of each of these disruptions, the body presents with unique symptoms ranging from skin symptoms and phototoxicity – as those seen in erythropoietic protoporphyria and congenital erythropoietic porphyria – through to acute attacks of abdominal pain, seen most commonly in acute intermittent porphyria. In short, no two porphyrias are clinically identical yet they are discussed as a single group of disorders with a similar cause. As a matter of fact, there are eight variations of porphyrias, each with a specific clinical manifestation of disease. (more…)

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How does indoor tanning cause skin cancer?

Wednesday, April 6th, 2011

Tanning causes skin cancerEarlier today, new stats were published by Cancer Research UK highlighting that two young adult Britons are diagnosed with skin cancer every day; a rate which has tripled since the 1970s. There were several factors highlighted as contributing to this increase, but one was most clearly singled out in the release:

“using a sunbed before the age of 35 can increase your risk of melanoma by 75%”

(more…)

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‘Measuring’ vitiligo: the challenges of clinical and treatment evaluation

Monday, April 4th, 2011

Since our announcement last year that Clinuvel would commence a new program for SCENESSE® (afamelanotide) in nonsegmental vitiligo, the company has received vast interest in the application of the drug in this disease. Of the enquiries that best captured the essence of this program, one stood out: a US based analyst asked how the company intended to objectively measure the response to treatment, the repigmentation of vitiliginous lesions, in its trial. (more…)

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A clinical success: study retention rates

Thursday, February 10th, 2011

We were delighted this week to be able to announce the successful completion of our first Phase II study conducted in the US, a placebo controlled, randomised trial of SCENESSE® (afamelanotide) for patients diagnosed with erythropoietic protoporphyria (EPP) (CUV030). (more…)

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FDA online regulation

Monday, January 17th, 2011

Effective communication is demanded more than ever in present-day drug development. I’ve blogged before about the role of patients in this process and the importance of acknowledging patient driven drug development. In a time of advanced technologies and communications it is my intention that Clinuvel be at the forefront of any developments in this area and that the company seeks new ways to discuss our program, online and off.

We also work in a highly regulated environment: very little of what the company does is without scrutiny from this perspective, either from our own regulatory team or from external organizations established for this purpose. Despite the hightened reviews, there remains a discord in the communications realm, with a lack of regulation surrounding tools referred to broadly as ‘social media’. (more…)

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Compassionate use – navigating the regulatory landscape to ‘do good’

Friday, December 3rd, 2010

As snow begins to fall around Clinuvel’s European office, the team in Australia is preparing for a long hot summer. The seasons are at the forefront of our minds at Clinuvel, since our lead drug SCENESSE® appears to have a dramatic impact on the ability of patients to expose themselves to sun. We try to test the drug under the most extreme conditions, meaning trials must be conducted in spring and summer. As the seasons change, we begin to see more requests and enquiries from the southern hemisphere, in particular from patients with erythropoietic protoporphyria (EPP), seeking access to the drug outside of formal trial programs. (more…)

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