The National Institute for Health and Clinical Excellence (NICE) has reversed its initial draft guidance and approved the use of Mepact® for high grade, non-metastatic osteosarcoma in children, adolescents and young adults.
NICE has implemented a clarification to its ‘methods guide’, relating to the appraisal of treatments (such as Mepact®) which can restore the health of a patient for a sustained period of time, normally at least 30 years.
The clarification considers the cost effectiveness of a treatment when both discounting of costs and health effects is applied.
This clarification in appraisal methodology, together with an enhanced patient access scheme (PAS) proposed by Takeda has resulted in a reduction of the cost per QALY of Mepact® to £36,000 which NICE has now considered to be cost-effective.
“This decision by NICE to recommend Mepact® is encouraging news for children and young adults with osteosarcoma,” said Dr Bruce Morland, Consultant Paediatric Oncologist at Birmingham Children’s Hospital.
“I’m really pleased NICE reversed its previous decision and has recognised the exceptional nature of its appraisal of ultra-orphan treatments for rare conditions such as osteosarcoma. This is a real triumph for clinicians and patients alike.”
Roger Paul, Chairman of the Bone Cancer Research Trust, added: “This result is thoroughly deserved and reflects the culmination of a long journey and tireless campaigning by many of our members who have contributed to the appraisal process with their personal experiences.
“This will give further hope to the patients and their parents as they battle with this terrible disease”.