HUMIRA (adalimumab) - Rectocolite hémorragique pédiatrique

Key points

Favourable opinion for reimbursement in the paediatric indication extension, i.e., the treatment of moderately to severely active ulcerative colitis in paediatric patients (from 6 years of age) who have had an inadequate response to conventional therapy including corticosteroids and/or 6-mercaptopurine (6-MP) or azathioprine (AZA), or who are intolerant to or have medical contraindications for such therapies.

What therapeutic improvement?

No clinical added value in the therapeutic strategy.

Role in the care pathway? 

The objectives of the treatment of ulcerative colitis (UC) in children and adolescents are to induce and maintain clinical remission and improve quality of life (in particular to maintain schooling). As in adults, there is no curative treatment for ulcerative colitis (UC).

In paediatric patients from 6 years of age with severely active forms who have had an inadequate response to conventional therapy including corticosteroids and/or 6-mercaptopurine (6-MP) or azathioprine (AZA), or who are intolerant to or have medical contraindications for such therapies, infliximab (REMICADE and its biosimilars) is the only TNF with an MA. It is administered by intravenous infusion. The alternatives in these patient are ciclosporin (off-label) and surgery for severe corticosteroid-resistant and corticosteroid-dependent forms. Ciclosporin is rapidly effective in corticosteroid-resistant patients, but its long-term safety is poor (nephrotoxicity, induced cancer risk), meaning that it cannot be considered as a long-term treatment. In practice, it is used for a short period (3 months) to induce remission pending the efficacy of another long-term treatment introduced simultaneously. Surgery is required in around 25 to 45% of patients due to an absence of improvement of symptoms or disease complications. The choice of surgery depends on age, how long the UC has been present, the degree of extension of the disease in the bowel, pregnancy plans, rectal status and risk factors for bowel cancer. In fact, total proctocolectomy and ileoanal anastomosis (IAA), with an ileal pouch, is a major surgical procedure that needs to be performed in 2 or 3 stages. Mortality is low (less than or equal to 1%) and morbidity high (30-40%: occlusions, pelvic sepsis, etc.). In addition, it significantly reduces female fertility.

Role of the medicinal product in the care pathway

HUMIRA (adalimumab) is a second-line option in the treatment of moderately to severely active ulcerative colitis in paediatric patients (from 6 years of age) who have had an inadequate response to conventional therapy including corticosteroids and/or 6-MP or AZA, or who are intolerant to or have medical contraindications for such therapies. It is an alternative to infliximab administered by the IV route, the other TNF inhibitor with an MA in this indication in paediatric patients. However, there is no comparative data available relative to the efficacy and safety of these two medicinal products.

The Committee highlights that treatment with HUMIRA (adalimumab) should be initiated and supervised by a qualified physician experienced in the diagnosis and treatment of UC. It reiterates that it is essential to ensure good treatment compliance.

Special recommendations

Given the risk of hypersensitivity reactions with adalimumab administered subcutaneously (see paragraph 4.4 of the SPC), but also with other biologic disease-modifying drugs, the Transparency Committee recommends that the first subcutaneous injection of this drug be given in an appropriate care structure.