Guest blog for Genetic Alliance
Jill Prawer of LPLD Alliance was invited to write a guest blog for Genetic Alliance UK. See what she wrote here.
The European Medicines Agency has just updated its European Public Assessment Report on Glybera (Alipogene Tiparvovec). You can read it here.
“Glybera is used to treat adults with lipoprotein lipase deficiency who have severe or multiple attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet” and “only for patients whose disease has been confirmed by appropriate genetic testing and who have detectable levels of the lipoprotein lipase enzyme in their blood”.
This designation is very limited and will not encompass everyone even within the small number of patients with LPLD.
On May 26th LPLD Alliance attended a scoping workshop at NICE which was the beginning of the process to assess whether the therapy will be paid for. The task of the workshop was to ensure that the right questions will be asked should Glybera go through to the next stage of the process.
It is thought that this decision will be made around the end of August/beginning of September.
Patient Advisory Board
On 13/14 March a group of patients met in the St Pancras Renaissance hotel, courtesy of Akcea Therapuetics, to discuss what it is like to live with LPLD. Some themes that emerged were how alone we felt in managing the condition, how the extreme restriction of fat makes ‘normal’ life difficult, and how our levels of energy are low compared to our peers who don’t have LPLD. For nearly all of the patients this was the first time they had met another patient.
Patients came from Holland, Belgium, Portugal, London and Luton.
After much negotiation, Andres the Chef at the hotel provided us with a fat-free feast! Dishes included hors d’oeuvres of prawn lollipops, sushi and spring rolls to name a few, and our mains included risotto, kedgeree, couscous and much much more. Plus some interesting deserts and snacks.
Everyone very much enjoyed meeting one another and we all went away with a better recognition of how difficult it can be to live with LPLD.