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Orphan Drug Award


Osteosarcoma – the children's cancer

Takeda UK in association with Liberation Communications

Summary of work

Each year in the UK an estimated 150 children and young adults are affected by osteosarcoma, a very rare bone cancer for which there has been no treatment developments for the last 20 years. Currently about one-third of all children and young adults who are diagnosed with osteosarcoma will not survive. Mepact could save the lives of an additional eight children per 100 treated each year in England who could go on to live ‘normal’ lives.
With just one leading osteosarcoma clinician in the UK with experience of Mepact and queries over the study design, Takeda needed to address awareness issues and allay any concerns.
Takeda UK focused on consultation and relationship building. To allay concerns relating to the study design, the lead investigator of the US studies (where the product was developed) visited the UK to address key clinicians face-to-face. He was also linked live to a major UK meeting where he was able to address a larger audience. As a result, one attending clinician made an individual funding request to a PCT very quickly following the meeting. Others were to follow.
In July 2010, NICE issued its interim guidance to recommend that Mepact should not be made available for the treatment of osteosarcoma in children and young adults. Following this, NICE received more third-party submissions than usual supporting Mepact when looking at more common areas of cancer. After 18 months of deliberation, a clarification to NICE’s ‘methods guide’ and an unprecedented six appraisal meetings, Mepact has now been recommended by NICE for use on the NHS for the treatment of osteosarcoma in children and young adults.  

Judges comments

“This entry shows recognition of a very specific healthcare environment and gives an honest appraisal of issues faced with a NICE STA. The market access activity was effective, bringing in prescribing clinicians, external stakeholders including patient groups, policy makers and MPs. The marketing approach centred on advocacy and relationships and was executed well.”

Stakeholder comments

“Takeda UK approached us as soon as it started working on the launch of Mepact. The company had a refreshing attitude of wanting to learn from us rather than trying to sell to us. Takeda knew that it needed to convince some people of the validity of their data and brought the lead investigator to our clinicians so that he could answer their questions directly. That went down well.”
Dr Bruce Morland, Consultant, Paediatric Oncologist, Birmingham Children’s Hospital
“Takeda has consulted with Sarcoma UK from the outset. The company is very open and honest about what it is doing – you know where you are with Takeda. Sarcoma UK is delighted with NICE’s positive decision and believe Takeda approached the STA with tenacity and a genuine commitment to see this treatment made available for the benefit of patients. Mepact is a significant step forward in the fight against osteosarcoma and will give further hope to patients and their parents as they battle the disease.”
Roger Wilson, Hon President (and former Chief Executive) Sarcoma UK

Key dates

Entry deadline 26 August 2022
Extended entry deadline 2 September 2022*
*additional fee applies
Judging Day 6 October 2022
Company/Team of the Year Interviews 21 October 2022
PMEA Event 23 November 2022