How can pharma innovate in childhood and TYA cancer?

As cancers in children and teenagers are rare, there is little commercial incentive for pharma to invest in researching drugs in this sector. Dr Tim Ringrose speaks to specialist in the field, consultant oncologist Dr Bob Phillips, about the challenges and opportunities.

Childhood and TYA (teenage-young-adult) cancers are rare. 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.

Dr Bob Phillips, Paediatric, Teenage and Young Adult Oncology consultant at Leeds General Infirmary, explains the difficulties: “Only one in 600 kids gets a malignancy and they fall into 20 unequally prevalent diseases.

Dr Bob Phillips

“This makes commercial involvements with pharma difficult, particularly when it is not a ‘lucky by-product’ of adult cancer (e.g. ALK1 mutations), but a fundamental element of an ‘exclusively’ paediatric cancer, such as antiGda2 antibody for neuroblastoma.”

In supportive care, Dr Phillips says 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, he believes 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 in the medical profession.

He 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.

“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.

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

“One of the big problems is that pharma is ‘designing trials for the drug rather than the patient'”

He believes that one of the big problems 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.

“Pharma needs to think about whether it needs to do so many tests and evaluations or whether it could get the data with half those investigations. For example, visits for study observations can really interfere with the life of a teenager or young person and their family and therefore they must be really, really essential for pharma to justify them. If the trial is less of a burden, they’ll get more people involved and obtain the answers faster – for themselves and everyone else.”

Another problem that needs to be addressed is that the people running trials are often used to dealing with adults and not children, which can be difficult for young patients. Also, some teenage cancers, such as leukaemia, are subtly biologically different to adult and childhood ones.

When considering setting up a trial, Dr Phillips urges pharma companies to be clear with doctors about how likely it is to go ahead, since sometimes doctors spend a lot of time answering preliminary questions only to find out that a trial is no longer happening.

“He also wants transparency from pharma when a trial has been completed so that the data is in the public domain”

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

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 childhood 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.

About the interviewer:

Dr Tim Ringrose is CEO, M3 Europe. He trained in nephrology and intensive care in Oxford before joining in 2000. Tim has led the development of services provided to doctors and has had considerable experience working with a wide variety of healthcare clients to deliver market research, targeted online communications and educational programmes to doctors.

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

Have your say: Will pharma rise to the challenge and research drugs specifically for children and teenagers?

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