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Equal access to care: - the challenges of cost and proximity

10 March 2025
In this blog RCPCH Wales Officer, Dr Nick Wilkinson, access to medicines.
Dr Nick Wilkinson

Children and young people often experience inequalities in access to care. We know this, but are we asking why and can we fix this?  

A 鈥渜uick鈥 win 

The recent (CP299) by NHS Wales took just eight months to be published from the time I raised an equity issue with the Joint Commissioning Committee (JCC). Young people aged from puberty (roughly 12 years and older) now have 鈥渆qual access鈥 to specialist, high cost medication for the same condition approved for adults (more than 18 years old).

For patients, this looks like great news. In rheumatology this doubles the access to new medication for adolescents. That鈥檚 a win! 

Alas, it鈥檚 not so simple. I still have to jump through hoops. The commissioning requires discussions about suitability with additional colleagues, documenting a discussion in a multidisciplinary meeting and completing additional paperwork. It does not, however, mandate my additional discussions with children, young people (CYP) and their families about why a drug is off-label and why there is no CYP specific patient material that would support joint decision making. This differs greatly from the flow of information and decisions in adult services.

From my experience, the hoops add delays and extra work and for some colleagues and patients, significant barriers. My frustrations aside, the situation is better than it was before, but it ultimately made me sit back and question why these variations exist.

Could it be that paediatricians are less trusted? Surely not. An IPSOS poll tells us paediatricians are the most trusted profession for secondary school children, across all ethnicities and backgrounds,1 but does this equally apply to our colleagues? 

I didn鈥檛 put the 鈥渢rust question鈥 to the JCC, and it was explained the drugs in question are off-label - although there is known dosing and safety in CYP and use in adults of similar maturity with the same condition. Of prescriptions across all ages, 21% are 鈥渙ff-label鈥.2 This is higher in children,3 and astonishingly, although pragmatically up to 94% in neonates4 . There is RCPCH and other guidance on unlicensed and off-label medication to guide clinicians.5

I am not advocating a game-for-all situation and I鈥檓 aware of cost implications. The reason for off-label medication in paediatrics is that clinical trials are too expensive despite demonstrable need.6 Not that efficacy trials, on which licensing is based, are safety studies - post marketing surveillance is relied on for this.

Is there data?  

It would be good to be shown the data informing the updated , which is the basis of new Welsh guidance I鈥檝e already discussed (CP299), but I can't even find the proportion of the 拢19 billion NHS drug expenditure spent on CYP. My data, however, from a 2013 audit of biologic use in 661 adults and 121 CYP with inflammatory arthritis showed just 2% of the total biologic cost was spent on juvenile arthritis (adjusting to population coverage), 63% of which was off-label, with no difference in efficacy or adverse event rates.  

Perhaps there are other heuristics at play, I鈥檇 argue paediatric practice is at times unnecessarily restricted by risk aversion - except obviously when it comes to neonates! Enhanced risk aversion reduces learning, adding to uncertainty and more work,7  while also grossly affecting patient choice as well as access8  and is in part why risk aversion is discouraged.9

Positive risk taking is supported by the Social Services and Well-being (Wales) Act 2014, the Mental Capacity Act 2005, the Human Rights Act 1998 and the Equality Act 2010. However, awareness of and confidence in this is limited within social care and among health professionals.

Imogen Blood and Shani Wardle, from Positive Risk and Shared Decision-making, Social Care Wales

How do we change this?  

CP299 feels to me to be representative of a broader issue of unchallenged perceptions and cognitive biases which recommends greater engagement with commissioners and colleagues in adult services. This is to better manage regulation which can have an effect opposite to that intended. There also needs to be a population health view beyond transactional care and simple risk aversion that carry significant costs. Trusting clinical responsibility and professional guidance will help and there has to be better data collection to monitor the impact of regulation, safety and other unintended consequences.  

RCPCH, at a recent Council meeting, actively promoted a system to enhance research opportunities and resilience, to address the lack of paediatric trials that leads to off label use, while also promoting systems thinking and leadership to address inherent biases and behaviours.10

[Models of care; The future] reminds us of the need to be brave and to face up to doing things differently.  

For now, document CP299 has, at heart, delivered improved access to care for adolescents via an alternative commissioning route and I鈥檓 grateful for greater transparency and fewer laborious individual patient funding requests to complete. I鈥檇 welcome your thoughts and drive to address inequality in access to care and how we can work together to improve the care offered to our patients.

A case history

I鈥檝e spent three appointments discussing medication for a 4-year old boy with arthritis and tenosynovitis but no loss of function - 鈥渉e joins in activities the same as his friends鈥. He has mild knee swelling with slight restriction, which his parents can see, and ankle and elbow swelling without restriction, which his parents can鈥檛. Ultrasound has confirmed the findings. He also has covert uveitis picked up on ophthalmology screening and quickly responsive to steroid eye drops. In usual circumstances methotrexate would be commenced, but for understandable reasons his parents would like to try other 鈥渕ore natural鈥 remedies before medication.

Treatment does not have to be started straight away. At the first visit we as a team carefully introduced information, had a more detailed discussion about risk-benefit during the second, and on the third found the discussion was at risk of becoming polarised and we spent this time discussing the parent鈥檚 perceived risks. My experience is that this was time well spent. Building rapport and trust (themes we discussed openly) was key to future adherence, adapting management and long-term outcome.

I can only provide information to inform parental decision in a way that allows them to feel heard and safe and gain confidence to experiment with ideas and actions. Sometimes I can鈥檛 do this, but our nurse, youth worker or other (peer specialist) parents can. And sometimes they can鈥檛, and we wait. Interestingly our discussions with similar parents about new high-cost biologic medications are completely different.

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