Hello everyone,
At last week’s meeting of the Cross-Party Group (CPG) on Brain Tumours in the Scottish Parliament, clinicians, researchers, patients and charity advocates delivered a unified message: bureaucracy is holding Scotland back from delivering world-class care to brain tumour patients.
Three key priorities were raised:
- Scotland needs equitable access to genomic testing
- Scotland must improve access to clinical trials
- Scotland must eliminate postcode-based disparities in drug access
The meeting highlighted multiple examples where delayed or unavailable genomic sequencing – a critical tool in diagnosing and treating brain tumours – has had serious consequences for patients.
Dr Mark Brougham, a paediatric oncologist at NHS Lothian, described a case where sequencing, outsourced to Great Ormond Street Hospital, revealed a different tumour subtype weeks later – a delay that could have compromised treatment.
“These aren’t academic niceties” he said. “They’re fundamental to getting treatment right.”
Ms Emer Campbell, a neurosurgeon from the Royal Hospital for Children in Glasgow, shared another case in which sequencing results prevented a life-altering surgery by enabling targeted chemotherapy. But she warned: “It took four weeks post-biopsy to get that answer – we could have done this in Scotland in a matter of days.”
Solutions exist – but are blocked.
Scotland’s universities already have validated long-read sequencing capabilities. However, they remain unutilised in clinical settings due to a lack of accreditation from the National Services Division (NSD).
Professor Colin Smith of the University of Edinburgh called this a “false economy”, noting that relying on external labs creates delays and costs more. “We have the tools. The only barrier is bureaucracy” he said. “Scottish patients are being denied timely access to trials and treatments.”
During the meeting, one patient campaigner described his battle to access Vorasidenib, a potentially life-saving drug available in some Scottish regions but not others. “It’s postcode prescribing” he said. “We need leadership to fix this national lottery.”
Dr Sorcha Hume of Cancer Research UK emphasised the importance of Scotland keeping pace with global research, warning that patients risk exclusion from major international trials due to a lack of genomic sequencing infrastructure.
Dr Joanna Cull, our Research Funding Officer, presented 'The Scottish Brain Tumour Research Centre of Excellence – The Story So Far', highlighting the Centre’s progress and its important role in driving translational research. She urged the Scottish Government to recognise brain tumours as a cancer of unmet need, match-fund the Centre, expand research capacity, and provide core funding for essential roles such as clinical research nurses and data analysts.
As the session concluded, we issued a direct call to Scottish Government: Scotland has the brains and the tools – but red tape is currently standing in the way. We’re urging Scottish Government to cut through the bureaucracy, back our experts, and make sure every brain tumour patient in Scotland gets equal access to the tests, treatments, and trials they deserve, no matter their postcode.
We can expect a busy period over the next couple of weeks as the 10-Year Health Plan looks set to be released. The plan will focus on delivering three big shifts in healthcare:
- hospital to community
- analogue to digital
- sickness to prevention
Also expected imminently is the Life Sciences Sector Plan which will include new measures to streamline regulation, introduce low-friction procurement, and support high-potential UK companies to scale, invest, and remain in the UK.
The plan has been developed alongside the 10-Year Health Plan to ensure alignment between health and growth objectives. It sets out a vision to drive growth, innovation and better health outcomes through three interconnected pillars:
- enabling world-class R&D
- making the UK an outstanding place in which to start, grow, scale and invest
- driving health innovation and NHS reform
Later in the year will come the National Cancer Plan and we are pleased to have seen assurances that rarer and less survivable cancers, including brain tumours, are a priority in the upcoming plan and we wait to find out what that will actually mean.
One area that will be under scrutiny is the regulatory world and this week we have had engagement with the National Institute for Health and Care Excellence (NICE) ahead of the committee meeting to “appraise the clinical and cost effectiveness of Vorasidenib within its marketing authorisation for treating astrocytoma or oligodendroglioma with IDH1 or IDH2 mutations after surgery in people 12 years and over.”
This was very useful and we are also encouraged by the Medicines and Healthcare products Regulatory Agency (MHRA) seeking feedback on their draft guideline on the use of external control arms based on real-world data to support regulatory decisions.
Essentially, what they are suggesting is to use external controls, even real-world data, as a comparator in trials, instead of the placebo arm. They are suggesting this design specifically for rare and serious conditions in which giving patients placebos could be viewed as unethical.
This is a hugely complex issue that polarises opinion but shows the willingness of the MHRA to look at doing things differently in the rare and less common space and we applaud this endeavour. Clinical trials are desperately needed for all rare diseases, especially brain cancer. Should you wish to input into the survey, follow the link but note the consultation will close at 11:59pm on Monday, 30th June.
Finally, this week here is a piece from Kelly Sitkin, president and chief executive officer of the American Brain Tumor Association:
The Medicare device coverage gap delays life-saving treatment for seniors.
Whilst we have the NHS, in the US there is Medicare which is a federal health insurance programme for people aged 65 and over, providing health insurance for 65 million individuals.
In the piece Kelly talks specifically about glioblastoma and says that, as it stands, there is “a nearly five-year gap between when a new medical device receives FDA (The Food and Drug Administration) approval and is subsequently covered by Medicare.
“This is time that many patients diagnosed with hard-to-treat cancers do not have.”
Kelly concludes her piece urging Congress to prioritise pivotal legislation “so that patients have unobstructed access to innovative treatment options that the FDA has deemed safe and effective.”
The similarities and differences between the UK and US, when it comes to treatment for diseases such as brain cancer, make this a fascinating read.
There are barriers to breakthroughs on both sides of the Atlantic.
In February 2023, the APPGBT published their report: ‘Pathway to a Cure – breaking down the barriers.’
We will do what we can to keep identifying and breaking down those barriers – our brain tumour community deserves nothing less.
That’s all for this week. We will be back next Friday so wishing you a peaceful time until then.
Karen, Hugh and Thomas