Companies Forum

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We launched our CRDN Companies Forum initiative at #CRDN2016  where it was met with much interest and a number of company representatives attended a lively breakout session.

Discussion meetings on the challenges and barriers facing companies working with rare diseases, are now in running 3 times annually and our first formal meeting took place on Friday 20th October 2017, at Robinson College, Cambridge prior to our annual summit.  

The meetings will be conducted under the Chatham House Rule  for representatives from pharma and biotech companies operating in the rare disease space interested in understanding the regulatory environment for orphan medicinal products.  October’s meeting involved a two hour workshop designed to explore the process of achieving regulatory approval for an orphan medicinal product. This covered issues such as orphan  designation and how to get it, scientific advice and protocol assistance, planning a clinical development programme when there are only a few patients across Europe, marketing authorisation and post marketing surveillance etc

The meeting was chaired by Alastair Kent OBE, former Chair of CRDN and ambassador for Genetic Alliance UK and the workshop led by Jordo LLinares, MD SSc, head of Scienntific and Regulatory Management, Human Medicines Evaluation Division, European Medicines Agency.

A discursive workshop took place exploring the help and support available and the hill companies have to climb if they are to be successful in bringing an orphan drug to market.  Providing an insight into the way that the Regulator approaches its task, and in so doing enabling companies to be better prepared when submitting an application for orphan designation and/or developing a clinical development plan intended to lead to a marketing authorisation.

Our first Companies Forum Meeting of 2018 will be held on March 1 from 9.30 – 1pm with lunch at the Double Tree Hilton by the riverside in Cambridge. We will be joined by guest speaker Sheela Upadhyaya, Associate Director of the Highly Specialised Technology (HST) Programme at NICE. She will provide an overview of NICE’s work in relation to rare diseases and its HST programme, which is used to evaluate some medicines for rare disease patients. Within the Strategy, NICE is responsible for implementing the commitment to ‘ensure that there are appropriate procedures for evaluating the costs and benefits of treatments available for patients’. Medicines evaluated through the HST programme are for very small patient groups. HST evaluations seek to balance the potential value of a medicine with the uncertainties associated with its efficacy (as a result of data gathered from small populations).

For further information see the Companies Forum concept paper and membership options.

If your company is interested in becoming a member of the Companies Forum   please contact Kay Parkinson, for more information.