dc.contributor.author
Werner, R.N.
dc.contributor.author
Pennitz, A.
dc.contributor.author
Eisert, L.
dc.contributor.author
Schmidle, P.
dc.contributor.author
Zink, A.
dc.contributor.author
Abraham, S.
dc.contributor.author
Schäkel, K.
dc.contributor.author
Wolff, I.
dc.contributor.author
Goebeler, M.
dc.contributor.author
Plange, J.
dc.contributor.author
Sollfrank, L.
dc.contributor.author
Zielbauer, S.
dc.contributor.author
Koll, P.
dc.contributor.author
Amschler, K.
dc.contributor.author
Müller, V.
dc.contributor.author
Nast, A.
dc.date.accessioned
2025-03-27T13:54:17Z
dc.date.available
2025-03-27T13:54:17Z
dc.identifier.uri
https://refubium.fu-berlin.de/handle/fub188/47064
dc.identifier.uri
http://dx.doi.org/10.17169/refubium-46781
dc.description.abstract
Background
In dermatology, a medical speciality with a relatively high number of rare diseases, physicians often have to resort to off-label treatment options. To avoid claims, physicians in Germany can file a cost-coverage request (off-label application, OL-A).
Objectives
Our aim was to investigate the extent to which the current regulations affect patient care.
Material and methods
Prospective cohort study among tertiary dermatology clinics throughout Germany, consecutively including OL-As (05/2019–09/2020) and assessing the follow-up correspondence. We modelled regressions to assess factors associated with cost-coverage decisions and the time needed by health insurers to process the OL-As.
Results
Thirteen clinics provided data on 121 OL-As, two of which applied for on-label treatments. Of the remaining 119 OL-As, 70 (58.8%) were immediately approved and 44 (37.0%) rejected. Including cases with one or more appeals, 87 of 119 OL-As (73.1%) were finally approved and 26 (21.9%) rejected. There was an association of the final approval rate with (1) the class of medication/treatment, with approval rates being significantly lower for JAK inhibitors than for biologics (OR 0.16, 95%-CI: 0.03–0.82); (2) German state, with approval rates being lower in eastern than in western states (OR 0.30, 95%-CI 0.12–0.76); and (3) cost of the intervention (no linear trend). However, none of these predictors was significant in our multiple logistic regression models. The median health insurer's processing time (first response) was 29 days (IQR 22–38). Our analyses showed no evidence of an association with the predictors we assessed. In cases approved, the median time from the decision to file an OL-A to the actual initiation of the treatment was 65.5 days (IQR 51–92).
Conclusions
Our study points to substantial delays and inequalities in the provision of timely health care for dermatological patients with rare diseases, often involving treatments for which there is no adequate approved therapy.
en
dc.rights.uri
https://creativecommons.org/licenses/by-nc-nd/4.0/
dc.subject
biological products
en
dc.subject
Janus Kinase Inhibitors
en
dc.subject
off-label use
en
dc.subject
patient care
en
dc.subject
practice patterns, physicians'
en
dc.subject
prospective studies
en
dc.subject
rare diseases
en
dc.subject.ddc
600 Technik, Medizin, angewandte Wissenschaften::610 Medizin und Gesundheit::610 Medizin und Gesundheit
dc.title
Impact of off‐label use regulations on patient care in dermatology – a prospective study of cost‐coverage applications filed by tertiary dermatology clinics throughout Germany
dc.type
Wissenschaftlicher Artikel
dcterms.bibliographicCitation.doi
10.1111/jdv.18357
dcterms.bibliographicCitation.journaltitle
Journal of the European Academy of Dermatology and Venereology
dcterms.bibliographicCitation.number
11
dcterms.bibliographicCitation.originalpublishername
Wiley
dcterms.bibliographicCitation.pagestart
2241
dcterms.bibliographicCitation.pageend
2249
dcterms.bibliographicCitation.volume
36
refubium.affiliation
Charité - Universitätsmedizin Berlin
refubium.funding
DEAL Wiley
refubium.resourceType.isindependentpub
no
dcterms.accessRights.openaire
open access
dcterms.bibliographicCitation.pmid
35737866
dcterms.isPartOf.issn
0926-9959
dcterms.isPartOf.eissn
1468-3083