PTR calls for expanded access to off-label drugs

According to the positions of the Polish Society of Rheumatology 2025, patient access to innovative treatment is steadily improving. Reimbursement of biologics and synthetic targeted drugs under drug programs is mainly based on registered indications, but for some drugs and indications also includes indications not specified in the SmPC, i.e. off-label. These are in line with current medical knowledge and recommendations.

In addition to access to innovative treatment, the availability of classic drugs should also improve. Currently, the following drugs are available in Poland:

• methotrexate (p.o., s.c.),
• azathioprine,
• chloroquine,
• cyclophosphamide,
• cyclosporine,
• mycofenolate mofetil,
• leflunomide,
• sulfasalazine.

According to the Reimbursement Law, all drugs are reimbursed for the indications listed in the current summary of product characteristics (SmPC). With regard to rheumatic diseases of inflammatory and autoimmune etiology, for most drugs, the registration indications do not cover all indications for which the drugs are used in practice. For some drugs, reimbursement indications are expanded in accordance with current medical knowledge and recommendations.

Currently in Poland, not all classical drugs are reimbursed in the full range of indications specified in the SmPC (leflunomide) and not all in off-label indications resulting from current clinical practice, knowledge and recommendations. There are also some basic drugs that have not been reimbursed in Poland so far (hydroxychloroquine).

Of the listed classics, patients have broad access, covering "all registered indications" and "autoimmune diseases other than those specified in the SmPC" for all of the above-mentioned drugs. drugs except: leflunomide (reimbursement covers only RA and to a limited extent MIZS), mycofenolate mofetil (reimbursement covers vasculitis, systemic lupus erythematosus, lupus nephropathy and systemic scleroderma) and oral methotrexate (reimbursement covers only registered indications, reimbursement in other autoimmune diseases applies only to subcutaneous methotrexate).

The notation "autoimmune diseases other than those specified in the SmPC" is interpreted differently, as it does not exhaust all clinical indications, especially arthritis with an etiology other than autoimmunity. This is particularly relevant in the case of sulfasalazine and antimalarials, which are often used in practice, for example, in cases of reactive arthritis or undifferentiated arthritis.

Improving access to classic drugs in mainstream pharmacies should primarily include the following active substances to the following extent (proposal to expand on the drugs or reimbursement criteria in relation to current availability):

• Hydroxychloroquine - for all registered indications and autoimmune diseases other than those specified in the SmPC, as well as arthritis of other etiologies,
• Chloroquine - arthritis of other etiologies,
• Methotrexate- oral form - autoimmune diseases other than those specified in the SmPC,
• leflunomide - for all registered indications and autoimmune diseases other than those specified in the SmPC,
• Sulfasalazine - arthritis of other etiologies,
• mycofenolate mofetil - interstitial lung disease in the course of systemic connective tissue diseases.

Among biologic drugs, reimbursement of rituximab and tocilizumab should also be considered off-label under drug programs for the indications:

Rituximab

• systemic lupus erythematosus (TRU, SLE) (B.150)
• Interstitial lung disease in the course of systemic scleroderma (SSc-ILD) and in the course of other systemic connective tissue diseases (PF-ILD in UChT³) (B.135)

Tocilizumab

• Interstitial lung disease in the course of systemic scleroderma (SSc-ILD) and in the course of some other systemic connective tissue diseases (including mixed connective tissue disease, MCTD-ILD).

In the case of the two aforementioned biological drugs, biosimilars are already covered by reimbursement, which should result in a broadening of indications for their use in Poland.

On 9.04.2025, during the "Medical Rationale of State" think tank debate on rheumatology, which was attended by PTR experts and representatives of the Ministry of Health, the National Health Fund and AOTMiT, various needs of rheumatology patients were addressed, including reimbursement of off-label drugs. The Polish Rheumatological Society, in cooperation with the national consultant in rheumatology, will continue to take active measures resulting in improved access to treatment in rheumatic diseases in Poland.

dr. med. Zbigniew Żuber, UAFM prof.

President of the Polish Society of Rheumatology

dr. n. med. Marcin Stajszczyk

Chairman of the Committee on Drug Policy of the Polish Society of Rheumatology

Source: PTR