https://www.pfic.org/...mmend-reimbursement-community-action-needed/#
Siehe auch hier: https://www.england.nhs.uk/wp-content/uploads/...-2021-March-2022.pdf
Ganz interessant sich da einzulesen - erklärt eventuell auch warum die Patientenrekrutierung so langsam voran geht zu Beginn. Auch wenn die Patienten wissen und überzeugt sind, weigern sich trotzdem die Krankenkassen, weil ihnen Odevixibat zu teuer ist -.-
Und wie wichtig die Studien und das label ist, nicht nur gegen den Juckreiz, sondern auch disease modifying zu sein.
Die interessanten Sachen sind leider geschwärzt, wie beispielsweise die Kosten. Zur Patientenpopulation habe ich folgendes gefunden: However, Albireo’s analysis of published data, informed by UK clinician input, indicates that UK prevalence of PFIC is 300-250 patients, with
incidence of 8-12 patients per year
We learned today that the National Institute for Health & Care Excellence (NICE), the drug decision-making body for England and Wales, put forth a decision not to recommend reimbursement of Odevixibat which is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. A similar decision is anticipated in France.
In Frankreich ist UDCA sogar für PFIC 3 Patienten zugelassen, was vermutlich die Preisdiskusion erschwert (siehe Anhaltspunkte in UK).
First-line treatment for PFIC is typically off-label ursodeoxycholic acid, or UDCA, which is approved in France only for PFIC type 3
Entscheidung über eine Kostenerstattung für Odevixibat in England / UK fällt wohl am 26 Januar?
https://www.nice.org.uk/guidance/gid-hst10043/documents/html-content-2
Im Folgenden noch ein paar Auszüge aus den Dokumenten:
https://www.nice.org.uk/guidance/gid-hst10043/...onsultation-document
The cost-effectiveness estimates are all much higher than what NICE normally considers an acceptable use of NHS resources within the context of a highly specialised service. So, odevixibat is not recommended.
The clinical experts noted the need for a treatment that, in addition, both improved growth and
preserved liver function. The committee recalled that cholestyramine is effective at lowering serum bile acid levels, but that it can be poorly tolerated (see section 4.3). It concluded that people with PFIC and their families would welcome odevixibat as a treatment for the condition.
They also highlighted that improvements in growth and liver function were important outcomes to people with PFIC and their families but had not been included in the company’s modelling.
However, it recalled that there was no evidence presented forthe rarer types of PFIC or comparing odevixibat with PEBD. In addition, there is no data on the anticipated NHS dosing schedule, and no agreed criteria for dose escalation after 3 months if there is not an adequate response. The short follow-up period in the clinical trial means that there is uncertainty about the longer-term outcomes such as time to, and need for, liver surgery and overall survival rates. The committee agreed that odevixibat likely reduces serum bile acid levels and pruritus in people with PFIC1 and PFIC2, but that its effectiveness in clinical practice is uncertain.
Using these assumptions, the cost-effectiveness results for odevixibat compared with standard care were considerably higher than the threshold normally considered an effective use of NHS resources in a highly specialised technology. The committee acknowledged that odevixibat is a high-cost technology and that uncertainties remained about the clinical evidence. However, none of the company or ERG scenarios were within the threshold considered a cost-effective use of resources for highly specialised technologies. So, the committee did not consider that odevixibat had the potential to be cost effective. As such, it could not be considered for amanaged access agreement. The committee concluded that it was unable to recommend odevixibat for PFIC.
https://www.nice.org.uk/guidance/gid-hst10043/documents/1
Key issues, clinical effectiveness (Seite 68)
Key issues, cost effectiveness (Seite 69)
https://www.nice.org.uk/guidance/gid-hst10043/...onsultation-document
Hier die Einschätzungen beteiligter Patientenkreise
Im Q32021 Call wurde das ja ähnlich angesprochen:
We've launched in Germany, we're actively in discussions with U.K., France and Italy, the next largest markets in Europe.
In den USA sieht es ähnlich aus, dass Odevixibat auf jedenfall nicht sofort SoC und First line treatment ist, so wie ich es verstehe (beispielhaft https://www.hca.wa.gov/assets/pebb/sebb-moda-ump-preauthreqs-part2.pdf ):
Odevixibat (Bylvay) may be considered medically necessary when the following criteria are met:
A. Member is three months of age or older; AND
B. Documentation of member’s weight, measured within past three months, is provided; AND
C. Medication is prescribed by, or in consultation with a hepatologist or gastroenterologist;
AND
D. A diagnosis of progressive familial cholestasis (PFIC) when the following are met:
1. Other causes of cholestasis have been ruled out (e.g., drug toxicity, hepatitis A,
sclerosing cholangitis, Alagille syndrome); AND
2. Diagnosis is confirmed by a molecular genetic test; AND
3. Member does not have PFIC type 2 with ABCB11 variant resulting in nonfunctional
or absent bile salt export pump protein (BSEP-3) as confirmed by a molecular
generic test; AND
4. Member does not have decompensated cirrhosis or prior hepatic decompensation
events (e.g., variceal hemorrhage, ascites, hepatic encephalopathy); AND
5. Provider attestation of presence of moderate to severe pruritis; AND
6. Treatment with ALL the following has been ineffective, contraindicated, or not
tolerated:
i. Ursodiol; AND
ii. Bile acid sequestrant (e.g., cholestyramine, colesevelam); AND
iii. Rifampin; AND
iv. Opioid antagonist (e.g., naltrexone); AND
v. Serotonin reuptake inhibitor (e.g., sertraline)
--> D.h. zusätzlich zu der eindeutigen Identifikation von PFIC müssen zudem die Medikamente i) bis v) als nicht wirksam getestet werden
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