Orphan Drug ‘Revestive’ Launch and the Importance of Mastering the Fundamentals and Individualised Care

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NPS Pharma vastly improve quality of life for short bowel syndrome patients with Launch of Revestive

Dr Thierry Darcis, NPS Pharma talks us through the launch of orphan drug ‘Revestive’ for the treatment of rare disease short bowel syndrome. 

His presentation on short bowel syndrome explored the unique and systematic approach provided by NPS to meet unmet needs of the orphan and rare disease industry.

Many may of heard of short bowel syndrome, believing it to be a common disease. It is in fact an ultra rare disease with an estimated figure of 1.4 patients per million in the EU. 

Given a microcosmic view of the work NPS are involved in, this particular case study demonstrated that while short bowel syndrome can be easily diagnosed, the disease requires a multidisciplinary routine in order to manage day-to-day. Having developed ‘Revestive’, which was approved by the EMA in 2012 for adult patients as the first long term, major treatment advance in 40 years, Dr Darcis commented that as a result of this discovery and launch, a handful of those suffering from short bowel syndrome have experienced a reduction of parental nutrition and IV fluid intake. Therefore giving back a balance of living a real life.

An integrated, patient-centric strategy is most important in educating, mobilising and informing those suffering with the disease, as well as relatives. As is the focus of developing strong relationships with patient advocates and doctors. And this extends to other orphan drug launches aside from those for short bowel syndrome.

The importance of mastering the fundamentals very much comes down to:

  • Planning clinical trials properly for both market access and regulatory purposes
  • Formulating reimbursement plans, data and evidence demonstrating an appropriate value proposition

NPS is a biopharmaceutical company specialising in rare diseases which only delivers what we call ‘first in’ class/’best in’ class therapies.

I’d like to thank Dr Darcis for his participation in this years Congress. If you’d like to hear more from this years World Orphan Drug Congress speakers, please visit  http://bit.ly/WODCEU2014.

 

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