Reimbursement granted to orphan drug in 3 Nordic countries
Orphan drugs are pharmaceuticals developed for rare diseases, but faces the same market access challenges as any pharmaceutical
National recommendation for a ultra-orphan product in Norway
Wickstrøm & Langkilde ApS supported EUSA Pharma by preparing a single technology assessment (STA) for Qarziba® (dinutuximab beta) in Norway. Qarziba was approved in Norway by Decision Forum (Beslutningsforum) to be used in line with the approved indication. Qarziba is indicated for the treatment of high risk (HR) and relapsed/refractory (R/R) neuroblastoma.
High risk neuroblastoma is a rare, pediatric cancer which occurs predominately in infants and young children aged 5 and younger. High risk neuroblastoma is a cancer with a very low survival and in approximately half of high-risk cases, the patient relapse.
Wickstrøm & Langkilde prepared the STA in line with the template specific for Norway and the requirements defined by Norwegian Medicines Agency (NoMA). Furthermore, Wickstrøm & Langkilde adapted a global model to reflect the country specific requirements and submitted the STA and model to NoMA.
In ultra-rare conditions like this, technology assessments may be challenging because clinical evidence is more difficult to establish than is the case for other conditions. In Norway, authorities have set up a set of criteria by which diseases are defined as very rare and severe and by which a new treatment is defined as highly effective. Authorities will accept a higher cost for treatments that meet the criteria for highly effective treatment of a severe, very rare disease than what is normally accepted in Norway.
Hence as a part of the project, it was investigated whether Qarziba would qualify for assessment within the framework for highly effective treatment of severe, very rare disease. Wickstrøm & Langkilde performed the relevant estimations needed, and all requirements on size of patient population, severity of the disease, and effectiveness of treatment was met and in June, 2019 Decision Forum issued a positive recommendation for Qarziba in Norway based on the evaluation by Norwegian Medicines Agency. By this decision, Norwegian patients with high-risk and R/R neuroblastoma were granted access to an effective treatment.
The project was undertaken in close collaboration with the European organization within EUSA Pharma, key input was retrieved from leading Norwegian experts and a close dialogue with Norwegian Medicines Agency was ensured throughout the process.
I worked with Wickstrøm & Langkilde ApS on a numeral submission in Nordic region. I experienced a professionalism with a high level of expertise in health economic, deep insight of the market access landscape and environment, policy requirements and value creation. An excellent collaboration on all project’s requirements. One important aspect is a level of expertise to establish and amend the strategy that fits to the disease specialties and company direction. I was impressed by working with Wickstrøm & Langkilde ApS and I’m confident that in case of any further submission I can rely on a partner that will develop the high-quality submission and lead the communication with the payers in Nordic region
Head of Global Market Access, EUSA Pharma (UK) Limited