Background and Importance Biological therapy has supposed a great therapeutical progress on immunomodulated diseases. Nevertheless, some pathologies have no labelled indication. Therefore, medication access on special situations are essential and more frequent.

Aim and Objectives The objective of this study is to analyse the request on immunomodulate therapy in special situations among last years.

Material and Methods Retrospective study performed in a tertiary hospital between January 2017-December 2021. Off-label (OL) and compassionate use (CU) requests on selective immunomodulatory drugs received by the Pharmacy and Therapeutics committee were included (P&T).

Data collected: number, type and drugs requested, indication, clinical department, and approvement by P&T. A temporal evolution on the number of requests, drugs and clinical departments was analysed. On those which showed an increase, an exhaustive analysis was performed.

Results A total of 95 requests were identified, 78 (82.1%) OL and 17 (17.9%) CU, representing a 17.3% (95/549) of all kind of requests to the P&T. Twenty-one drugs and 42 different indications were identified. Eighty-seven (91.6%) were approved; six were denied due to lack of evidence and two due a lack of funding by the national health system.

Main drugs requested ustekinumab (18 (18.9%)), dupilumab (15 (15.8%)), rituximab (14 (14.7%)), tofacitinib (9 (9.5%)), tocilizumab (7 (7.4%)), adalimumab (5 (5.3%)).

Requesting clinical departments dermatology (48( 50.8%)), digestology (20( 21.1%)), rheumatology (18( 18.9%)), nephrology (5( 5.2%)), internal medicine (2( 2.1%)), pneumology (1( 1.1%)), coronary unit (1( 1.1%)).

An exponential increase was observed among OL and CU during the study period (requests/year: 8/2017, 12/2018, 14/2019, 21/2020, 40/2021; y=5.2269e0.3778x, R2=0.9559).

The main growth was observed in dermatology (y=1.99e0.4277x, R2=0.768) and digestology (y=1.5x-0,5, R2=0.9375).

Indications requested by dermatology: atopic dermatitis (15 (31.3%)), hidradenitis suppurativa (8 (16.7%)) folliculitis decalvans (4 ( 8.3%)), others (21 (43.8%)).

Drugs requested by dermatology: dupilumab (15 (31.3%)), ustekinumab (5 (10.4%)), tofacitinib (4( 8.3%)), mogamulizumab (4 (8.3%)), adalimumab (4 (8.3%)), secukinumab (3 (6.3%)), rituximab (3 (6.3%)), infliximab (2 (4.2%)) and others (8 (16.7%)).

Indications requested by digestology: ulcerative colitis (13 (65.0%)), Crohn’s disease (4 (20.0%)), collagenous colitis (3 (15.0%)).

Drugs requested by dermatology: ustekinumab (13 (65.0%)), tofacitinib (4 (20.0%)), vedolizumab (2 (10.0%)), infliximab 1 (5.0%)).

Conclusion and Relevance

• Dermatology performed half of requests, specially in atopic dermatitis and hidradenitis suppurativa, which have obtained more evidence on their treatment last years.

• The exponential increase on number of requests in special situations, specially off-label ones, reveals the need to increase the resources assigned to evaluation committees.

Conflict of Interest No conflict of interest