maliqun61 schreef op 19 oktober 2018 06:23:
[...]
Tja, wie het kleine niet eert .......
Neem bijvoorbeeld Schotland (geschat aantal HAE patiënten 102), kijk vooral naar de afwegingen.
conestat alfa 2,100 units powder (and solvent) for solution for injection
(Ruconest®) SMC No 745/11
Pharming Group NV
6 July 2018 / Published 13 August 2018
The Scottish Medicines Consortium (SMC) has completed its assessment of the above product and advises NHS Boards and Area Drug and Therapeutic Committees (ADTCs) on its use in Scotland. The advice is summarised as follows:
ADVICE: following a full submission assessed under the ultra-orphan medicine process conestat alfa (Ruconest®) is accepted for use within NHSScotland.
Indication under review: For treatment of acute angioedema attacks in adults and adolescents with hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency.
Conestat alfa was associated with a significantly shorter time to relief from symptoms of HAE attack compared with placebo during controlled phase III studies.
This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of conestat alfa. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.
This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting
Patient and clinician engagement
A PACE meeting with patient group representatives and clinical specialists was held to consider the added value of conestat alfa as an ultra-orphan medicine in the context of treatments currently available in NHS Scotland.
The key points expressed by the group were:
• The frequency of HAE attacks can vary significantly between and within individual patients with severity ranging from mild to life threatening. Patients with frequent attacks may be confined to bed or home, while those with infrequent attacks may be affected by the unpredictability in their timing and severity. These situations can be significantly disruptive to normal daily and family living, and to work and education and can have a substantial negative impact on quality of life.
• Conestat alfa provides an additional effective option for the treatment of acute HAE attacks and increases patient choice.
• The availability of an additional C1 inhibitor, including the availability of a non-plasmaderived product, would help mitigate the risk of potential supply shortages which have occurred with other plasma derived products eg immunoglobulins. Any shortage in supplies of C1 inhibitors could put patient lives at risk.
• The currently available C1 inhibitors (Berinert® and Cinryze®) are plasma-derived products and conestat alfa would offer an alternative animal-derived product for patients who wish to avoid plasma-derived products. This could be for religious reasons or to avoid the theoretical risk of human blood borne pathogens. Although no case of infection transmission has been reported to date, the availability of conestat alfa would offer patients an alternative choice.
• The shelf life of conestat alfa is longer than for other available C1 inhibitors and this may be an advantage where supplies are held in remote centres. It may also be an advantage for patients with infrequent attacks who keep a supply of C1 inhibitor at home for emergency use or for travel.
Pharming heeft bovendien de betreffende patiëntenvereniging niet omgekocht.
Additional Patient and Carer Involvement
We received a patient group submission from HAEUK, which is a registered charity. HAEUK has received 85% pharmaceutical company funding in the past two years, with none from the submitting company. Representatives from HAEUK participated in the PACE meeting. The key points of their submission have been included in the full PACE statement considered by SMC.
Conclusie:
After considering all the available evidence, output from the PACE process, and after application of the appropriate SMC modifiers, the Committee accepted conestat alfa for use in NHS Scotland.
Toestemming in Wales (een nog kleiner geschat patiëntenaantal: 61) zal zéér binnenkort volgen met waarschijnlijk dezelfde argumentatie.
Je zou ook kunnen zeggen: veel kleintjes maken één grote.
Ook kleine kinderen worden groot.
www.scottishmedicines.org.uk/media/36...