What will be their fate?

The NICE evaluation meeting had taken place in December ( see Relucant Advocate story here) so aHUS families in England entered January 2014 with the uncertainty of their fate and what NICE would decide and tell us later that month.

Unsurprising as we learned by then we soon found out we would not find out in January as NICE announced the following to participants: :

Dear Consultees and Commentators  

Highly Specialised Technology Evaluation

Eculizumab for the treatment of atypical haemolytic uraemic syndrome (aHUS) [ID703]

Following the Highly Specialised Technology Evaluation Committee meeting on Wednesday 11 December 2013; 

• If an Evaluation Consultation Document (ECD) is produced, this will be sent to consultees and commentators in the week commencing 17 February 2014 

• If a Final Evaluation Determination (FED) is produced, this will be sent to consultees and commentators in the week commencing 10 March 2014″

The two documents needed some explanation.

1. Evaluation consultation document (ECD) if produced
The Evaluation Committee make its provisional recommendations in the ECD. An ECD will be produced only if the recommendations from the Evaluation Committee are restrictive. A restrictive recommendation will be one that is more limited than the instructions for use that accompany the technology. Consultees and commentators have four weeks to comment on the ECD. The ECD is also made available on our website so health professionals and members of the public can comment on it.

2. Final evaluation determination (FED) produced,
The Evaluation Committee considers the comments on the ECD if produced, then makes its final recommendations in the FED on how the technology should be used in the NHS in England. Consultees can appeal against the final recommendations in the FED.

So if an ECD is issued it would not be the end of the matter. If it was to be an FED it would be the end , subject to appeal.

We subsequently heard that any ECD would be released in the week commencing 24 February.

That made us think.

As part of our involvement in NICE aHUSUK had to sign a confidentiality agreement and it prevented us revealing anything in the public domain about what we had been told us for 7 days after we had been told.

The earliest we could say anything to aHUS families  about the result of an ECD would be 3 March,  3 days after Rare Disease Day 2014.

The Rare Disease community was wary about NICE is involvement in these decisions. Would a “not re ommended” decision not be palatable in the Rare Diseaes community at a time of heightened awareness

A negative result would not go down well on Rare Disease Day. Better for Government for it  to come  after. March 10th for a Final Evaluation Decidion would mean nothing would be heard by Rare Disease Day.

The wait continued it looked like our fate certainly was that it would not end soon.