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Update on Scenesse®

As members are probably aware, funding for SCENESSE® in the UK is currently being discussed with NICE (the National Institute for Health and Care Excellence).

NICE considers whether a drug is likely to benefit patients, whether it will help the NHS meet its targets and whether it is value for money/cost effective. Once NICE issues its guidance, NHS trusts must find the money to make a drug or treatment available. It doesn’t give any extra money, or advise how trusts should find the money.

As part of the NICE process, the BPA (along with a number of EPP patients) attended a scoping workshop in March to discuss the proposed evaluation of SCENESSE®. Patient involvement was limited at this stage – further involvement will come at a later stage if the drug is referred for full evaluation. However, we did still have the opportunity to bring up some very salient points regarding EPP.

The meeting started with a brief overview of the NICE process. In summary, after the scoping workshop, NICE, the Department of Health and NHS England will meet to discuss the outcomes of the scoping process and decide whether to take it forward to evaluation. Highly specialised technologies (HST), such as this, then need ministers to formally refer the topic to NICE for evaluation.

In terms of timelines, a referral for this evaluation would be expected sometime between July and September 2016, after which consultees and commentators would be advised of the evaluation period dates. During the evaluation, a few patients would be able to put their personal patient views forward. The BPA will also be able to help by providing data to back up the process, hopefully in the form of surveys which can collect information from larger population numbers.

The process then may pass through another few stages before a final evaluation determination (FED) would be produced. The FED would recommend how the technology should be used in the NHS in England. If there are no appeals, or an appeal is not upheld, the final recommendations would be then issued as NICE guidance, after which point NHS Trusts would be able to provide SCENESSE® via expert centres around England and Wales initially, with Scotland and Ireland hopefully following soon after.

As is evident from the above, the NICE process is lengthy and time-consuming, but we will keep you up-to-date on all developments as they happen.


SCENESSE® ENFANCE – A Paediatric Development Plan for Children with EPP

At the Extraordinary Porphyria Patients Meeting in Rotterdam earlier this year, Clinuvel Pharmaceuticals Ltd., not only discussed aspects regarding the rollout of SCENESSE across Europe in adult EPP patients, but they also introduced their plan for the development of a new treatment programme for children with EPP – entitled “SCENESSE® ENFANCE”.

As soon as Clinuvel start to dose adult patients with SCENESSE®, at multiple locations across Europe, they will begin developing their paediatric version. They estimate that this will take approximately three years and vast amounts of funds to focus on product development, clinical trials, regulatory filings and the ongoing monitoring and follow-up of patients. However, it is very important to note that the successful completion of the development of a paediatric product is dependent upon wide distribution of SCENESSE® as this will allow for the continued monitoring of the safety of SCENESSE® in the adult EPP population across Europe. The data from this will be captured via the European EPP Disease Registry (EEDR).

As above, the BPA will keep you up-to-date on all developments as they happen. You may also like to visit for further information.