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Cambridge Rare Disease Network - Previous Companies Forum meeting details 1

Companies Forum


Summer 2018

The meeting welcomed  guest speaker Dr Emma Harvey, E&RH Ltd  for an excellent presentation and discussion regarding Managed Access agreements in the UK, the perils and pitfalls with reference to the NICE HST process.  

March 2018

We were joined by guest speaker Sheela Upadhyaya, Associate Director of the Highly Specialised Technology (HST) Programme at NICE. Sheela provided 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). Sheela was joined by MHRA – Dr Daniel O’ Connor, Expert Medical Assessor, member of COMP and SAWP who provided a detailed presentation.

October 2017

Our 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. The meeting was chaired by Alastair Kent OBE, former Chair of CamRARE and CEO of Genetic Alliance UK and the workshop led by Jordi LLinares, MD SSc, head of Scientific and Regulatory Management, Human Medicines Evaluation Division, European Medicines Agency.

A discursive workshop took place exploring the hill companies have to climb, and what help and support are available 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.

For further information see the Companies Forum concept paper.