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How can pharma innovate in child and TYA cancer?

by Dr Tim Ringrose, M3 (EU)
Digital Handbook

Child and TYA (teenage-young-adult) cancer is rare: only 1:600 kids get a malignancy and they fall into 20 unequally prevalent diseases.

Consequently it can be difficult to get commercial backing from pharma for drug development and testing, and there are challenges in many areas including supportive and relapse care studies, new agents and front-line treatments.

We asked member and Consultant in Paediatric, Teenage and Young Adult Oncology, Dr Bob Phillips, to tell us more about the challenges in this specialty and what role he would like to see pharma play in tackling them.

Dr Phillips explained that in supportive care, drugs to tackle problems, such as sickness, infection, mucositis, constipation and lethargy, seem to be based only on adult drugs and do not follow as quickly into paediatrics as other cancer medicines.

In relapse care, there are not enough relapse therapies for children and TYAs, and not many trials that compare relapse strategies. There are also problems with trials awareness, access and applicability, although considerable work is being done to address this within the medical profession.

Dr Phillips would like to see continued engagement with pharma to make new agents available and to expand programmes to investigate which drugs might be useful for children and TYAs for front-line treatments.

He said: 'Essentially, I want to see pharma taking an approach to children's cancer that says we want to help, and we'll use the profits from other drugs to test kid cancer drugs – rather than just using adult drugs.'

Persuading children and young people to participate in studies can be difficult compared to older people, owing to school and college commitments. Also, in the late stage of the disease when time is particularly precious, a young patient may really resent having to go into hospital regularly for tests.

While the paediatric oncology network is good about putting children and young people forward for trials, Dr Phillips says trials tend to be very restrictive, particularly in terms of the TYA age, comorbidity and when a patient needs to attend for tests within a demanding treatment cycle.

One of the big problems, he believes, is that pharma is 'designing trials for the drug rather than the patient' and not reflecting the challenges of children and young people who are going through cancer and are already receiving a lot of care.

He also wants transparency from pharma when a trial has been completed so that the data is in the public domain and full access to the data is provided to reputable researchers - an example of which is the Yale School of Medicine's Open Data Access (YODA).

In conclusion, Dr Phillips would like to see pharma making 'sensible adaptations of its adult drugs and clinical trials' and developing new drugs for specific groups of child and TYA cancers. He says that since paediatric and TYA cancer is rare, it's not about making money for pharma – 'it's about helping people' and this could reap great rewards for the industry in terms of improving its reputation.

For more information on M3, the global provider of technology services in healthcare, and its new European Division which includes,, and, please call Tim Ringrose on +44 (0)1235 828400, or email

Article by
Tim Ringrose

15th December 2014


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