from clinical experts, Patient Groups and, where relevant, the output from a Patient and Clinician Engagement (PACE) meeting, and will make a decision on routine use of the medicine in NHSScotland. SMC will then review information provided by the company as well as other sources of evidence e.g. This should include relevant new evidence for example: results of further controlled studies, observational, registry or other real world data gathered during initial availability of the medicine within the ultra-orphan pathway.
#Define orphan full#
The company will be asked to provide a full updated submission following the period of data collection. Guidance to support this phase of the ultra-orphan pathway is available on the Scottish Government website. The previous ultra-orphan process (pre April 2019) is now closed for submissions and resubmissions.ĭata collection can begin at an early stage in parallel with SMC initial assessment. Submissions received from April 2019 onwards will be assessed using the new approach. Further information is available in SMC guidance to submitting companies – supplement for medicines for extremely rare conditions (ultra-orphan medicines). SMC will use a broad framework to appraise ultra-orphan medicines which takes account of the nature of the condition, impact of the medicine, value for money, impact beyond direct benefits and on specialist services, and costs to the NHS. This will highlight uncertainties within the available evidence-base and will help to inform the data collection stage of the ultra-orphan pathway.Ĭompanies should complete the New Product Assessment Form (NPAF) for Ultra-Orphan Medicines. Thereafter if a product has GB marketing authorisation from the MHRA if there are eligible patients in NHSScotland and if no submission is forthcoming, SMC will move to issue Not Recommended advice.Īn initial assessment of the clinical and cost-effectiveness of the medicine will be conducted. Ultra-orphan validation decisions will expire after 2 years. Confirmation that the medicine is eligible for the ultra-orphan pathway is required before the company makes a submission for SMC initial assessment. SMC will review the proforma and will confirm whether or not the medicine has been validated as ultra-orphan within around eight weeks (deadline for receipt of proformas is the first Monday of the month). (Note that in the event of a high number of proformas being received we may prioritise to take account of the date and time received and actual / estimated date of product availability in the UK). Companies are encouraged to seek confirmation that a medicine meets the ultra-orphan definition at an early stage, and ideally prior to receiving an EMA Committee for Human Medicinal Products (CHMP) opinion, by completing an ultra-orphan proforma.
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