The challenges of ‘orphan’: can drug development cope with rare diseases?

Thursday, June 9th, 2011

Scarcely a day goes by that the Clinuvel team is not contacted regarding the latest conference: everything from monitoring to manufacturing to marketing is analysed, discussed and disputed across the globe in conference form. It’s impossible – and unwise – for small companies to try to attend all these forums, but it is equally impossible to work in the space without looking outside the office door: there is much to be learned from what others have done or not done to succeed in an industry fraught with failure.

And indeed there was much to take on at the recent World Orphan Drug Summit, held in Frankfurt last week. (more…)

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At what cost, expertise?

Thursday, May 26th, 2011

Drug development is time consuming, expensive and fraught with risk. Setting aside commercial considerations, the scientific rational for the use of a drug and its introduction into the clinic requires a deep level of scientific and medical understanding in an environment which is continuously changing. The less common the drug class or clinical indication, the less likely one is to find individual experts who have the capacity  to properly evaluate whether a drug is safe and effective for use in a clinical setting. Here one needs to learn while progressing, learning on the job due to lack of precedent.

If one identifies scientific experts in the field of interest, they are likely to be highly specialised physicians and basic scientists; often these experts will want to be involved on a potential breakthrough by accessing the prospective and novel therapy in their field of expertise. (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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FDA Basics website reworks new drug approvals data

Friday, March 18th, 2011

We’ve spoken often about drug approvals by regulatory agencies, particularly approvals for new drugs, or new molecular entities (NMEs). A quick – and useful – data update on this front has just been posted by the FDA to their FDA Basics website (a project we blogged about in 2010).

The FDA has posed itself two questions which it seeks to answer, head over the their website to see the results:

Is it true FDA is approving fewer new drugs lately?

Is the U.S. really slower than Europe in approving new drugs?

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New drug approval rates

Friday, February 25th, 2011

Novel drug development is a business which is not well understood; perhaps one which is not well explained. To be able to launch a novel drug proposition, one needs tenacity, expertise and a talented team to succeed. Unlike a ‘follow-on’ product where an abundance of safety data on the molecule in one or other formulation or therapeutic application exists, those few companies working with new drugs must clear all the necessary stringent barriers from the outset.

This makes novel drug development a risky endeavour, amplified by a level of uncertainty even when it is believed that the necessary regulatory hurdles have been cleared and the drug development process seems complete. By looking at several new molecules being developed for obesity (Lorcaserin, Contrave and Qnexa particularly), one can see how uncertain the outcome remains, even when so much work and so many years have been invested; others have written extensively about these cases and I encourage avid readers to review the publications. (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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The Fitzpatrick Scale

Wednesday, October 13th, 2010

The Fitzpatrick Scale (also known as skin type or phototype) is a system for classifying skin based on the amount of melanin naturally present in skin and its response to ultraviolet (UV) radiation exposure. It was developed in 1975, by Dr Thomas B. Fitzpatrick, a dermatologist at the Harvard Medical School. In the years since, the categories have been revised and refined and are still widely used today. (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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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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