Major areas of debate for the orphan drug industry

orphan drug congress, rare diseases, rare partners, premium price, clinical developmentOrphan drug development to treat rare diseases is a field of real concern and importantly change for the pharmaceutical industry. With over 6000 known rare diseases, many of which are exceptionally severe, it is simply a necessity to create a new paradigm where orphan drug development can occur quickly and reach the patients in need in a timely fashion.

I was fortunate to liaise with the speaker faculty for the World Orphan Drug Congress 2012 and ask them what they deemed the major areas of debates for the industry, and if one thing could be changed to speed up development, hypothetically what would that be?

Marco Prosdocimi, Managing Director of the not-for-profit Rare Partners srl shared his thoughts to me recently.

"The model based on high cost and high return based on premium price in the present economic situation is at risk. Patient associations will help? Regulatory authorities will help? Can a different model be envisaged? Clinical trials should be performed in a shorter time frame. Ethical Committee analysis should be centralised and surrogate end points should be further studied and possibly accepted for registration. Indeed the number of patients is small and no country, in general, can provide adequate number of patients. At this point, small companies are in trouble – for them clinical trial organisation is very difficult. International grants should be made available in order to allow clinical trial completion in a shorter time frame. Furthermore, clinical trials involving a large number of countries should be supported by health authorities and patient organisations. Finally, Ethical Committee analysis should be centralised and surrogate endpoints should be further studied and widely accepted for registration "

I went on to ask why Marco had elected to speak at the World Orphan Drug Congress 2012 in Geneva – "First, I want to disseminate the ideas that were leading us to the establishment of Rare Partners srl, namely a company with a non profit mission. Second, it will be an ideal place to have an idea of what is going on almost everywhere. Third, I may find companies interested in our projects or scientists willing to propose to us new collaborations. [Delegates] will have a rather complete view of a very complex field in 2 days…few of them will find a company willing to work in their project or a scientist with a project that fits a company plan"

Marco Prosdocimi will speak on the opening day of the congress within the Clinical Development and R&D track, focusing on the "Potential for repositioning to act as a gateway to openly innovate in the rare disease field for a non-profit company". The presentation will focus on:

  • Developing orphan drugs from an altruistic viewpoint using an innovative business model, based on collaboration with academia, foundations and established companies
  • Is there room for new players, such as non-profit companies and charities, in the drug development arena, making "non-commercial" rare diseases more attractive?
  • Can repositioning be a suitable strategy for non-profit companies endowed with drug development expertise?

With over 60 speakers confirmed to lead the debate at this years World Orphan Drug Congress, we are ensuring there will be the content and focus you need for your business. See how the content will come together across the three tracks by downloading the brochure.

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