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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Study: pharma ads failing FDA guidelines

Thursday, August 25th, 2011

The marketing model for US prescription pharmaceuticals is often debated for its direct-to-consumer (DTC) advertising as the United States is one of the few jurisdictions globally which allows this type of marketing. Yet equally debated is promotion directly to physicians, key decision makers in the prescription process. Physician promotion comes in many forms, ranging from one on one visits with sales representatives to educational sponsorship to the ubiquitous branded pen. (more…)

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Small populations, big therapeutic potential

Monday, May 23rd, 2011

When Andrew Pollack of the New York Times declared that the “world’s largest drug company is thinking small”, he wasn’t referring to reductions in sales force.

Rather, Pollack was reporting on a licensing deal between Pfizer and Israeli biotech company Protalix which built upon a growing global trend: big pharmaceutical companies were making a move into treatments for rare diseases, otherwise known as ‘orphan’ therapies.

In the months that followed Pollack’s December 2009 article, both Pfizer and GSK (two of the world’s largest pharmaceutical companies) launched specific business units focused on R&D for orphan drugs. In their announcements, both companies highlighted the significant unmet medical needs that exist in rare diseases and the potential of therapies that were in development. (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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Pain measurement in clinical studies: The Likert Scale

Friday, August 13th, 2010

surveyWhen assessing new therapeutic goods through clinical trials, researchers must obtain information on the degree of a patient’s physical response to therapy. This data then undergoes detailed statistical analyses in order to determine the safety and efficacy (effectiveness) of the drug or treatment.

Central to Clinuvel’s clinical trial design, and the value of these studies, is measurement of the severity of phototoxic reactions (adverse reactions to light or UV radiation) in trial patients. In order to do this accurately, a symptom severity scale has been developed based on the method of ‘Likert scaling’.

(more…)

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Summary of today’s announcements

Wednesday, July 14th, 2010

Today’s announcement of the results of CUV017 has brought with it a stream of updates from Clinuvel, on the website, YouTube and beyond.

In this post we summarise the days releases and developments.

(more…)

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What makes a ‘gold standard’ in cGMP? (plus a new webcast)

Monday, July 12th, 2010

As previously discussed on the blog, Good Manufacturing Practice, or GMP is a vital consideration for pharmaceutical manufacture and quality control. This means that the processes, equipment, active ingredients, documentation and training are controlled and of a high standard prior to a drug being approved. To be in compliance with GMP, regulations should ensure that the drug substance is of adequate quality, when used in humans and becomes available commercially.

Ultimately, quality protects the public and ensures that the reality of a drug sold or prescribed is an accurate reflection of the claims and ingredients made not only on the label, but also of the exact nature of the formulation that was approved through rigorous clinical trials and analysis.

(more…)

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Jack Wood discusses Clinuvel’s manufacturing partner for SCENESSE®

Friday, July 9th, 2010

Clinuvel Non-Executive Director Jack Wood discusses the selection of SurModics Inc as Clinuvel’s first commercial manufacturing partner for SCENESSE®. Click here to listen.

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Necessary innovation: the vital role of manufacturing in pharmaceutical development

Thursday, July 8th, 2010

This morning Clinuvel announced a significant milestone in its development program for SCENESSE®: the signing of the first manufacturing agreement for the final drug product with SurModics, Inc., a leading provider of drug delivery technologies to the healthcare industry. It has taken over eight years for Clinuvel to reach this milestone. In this time, the company has refined SCENESSE’s active ingredient – afamelanotide – and arrived at a final injectable drug product which controls its release to achieve an optimal photoprotective response in patients.

(more…)

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Orphan drug development legislation and regulation landscape

Monday, May 17th, 2010

Previously we have discussed the shifts which take place in the regulatory centers of the world specifically London and Maryland, which present an opportunity to the industry to respond to expressed need.  Perhaps the greatest concern of every individual is access to health and quality of life.

This implies a strong directive for drug developers to respond to the identified needs of the patients and patient communities. But amongst those with therapeutic needs are some with more unaddressed medical requirements: patients with ‘orphan’ diseases.

Orphan diseases are rare and frequently untreated diseases. The previously referred shift towards patient driven drug development made a few voices heard and legislators in the European Community and United States started to act on behalf of the population. Here the legislators and regulators provided incentives to drug developers by reducing fees, providing exemptions and protection from competitors.

(more…)

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