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Addressing the common challenges of rare cancers

Dr Olivia Ashman
Oncology Medical Director, Pfizer UK

26th June 2019

It seems like a contradiction to describe rare cancers as common. After all, the very definition of a ‘rare’ cancer is if fewer than six in 100,000 people are diagnosed with it each year.1 Individually, it is indisputable that each of the 198 known types of rare cancers are indeed ‘rare’, but in my role as Oncology Medical Director for Pfizer UK, I am aware just how widespread their collective impact actually is. In fact, nearly half (47%) of all cancer diagnoses are for rare or less common forms of cancer and together these cancers are responsible for 54% of cancer deaths in the UK.2 These numbers paint a very different picture; that collectively rare cancers are in fact common.


Despite these startling facts, rare and less common cancers, and the patients impacted by these diseases have been somewhat overlooked. Survival rates for rare cancers are lower than those for common cancers;3 patients are often diagnosed late, or incorrectly, and once a rare cancer is diagnosed there is often a lack of access to appropriate therapies,3 clinical expertise and patient support information.1 For people affected by rare cancers, every stage of the cancer journey can be more difficult.4

This is a challenge we need to address and at Pfizer it’s something we’re focused on. Thankfully, cancer science is progressing at a rapid rate. Our understanding of the epidemiology of cancer is becoming more and more sophisticated and we are continuing to identify sub-types of cancers which can be detected and treated more effectively. We are investing in the development of transformative medicines that offer renewed hope where treatment options have been limited for many years, and the era of personalised precision medicines is providing patients with targeted treatments that can be tailored to their needs.

For those living with rare and less common cancers, and indeed those living with other rare types of genetic disease, the rise of genomic medicine is particularly exciting, and it is encouraging to see a renewed focus on this area of cancer medicine in the NHS Long Term Plan.5 With firm commitments to accelerate diagnosis and harness the power of genomic technologies, the NHS has set itself the ambition of being the world-leading healthcare system in genomic medicine.

The new Genomic Medicine Service has begun working to sequence 500,000 whole genomes by 2023/24, and the NHS will this year begin to offer genome sequencing to children and adults suffering from certain rare diseases and cancers.6 These are significant steps with huge potential to transform patients’ lives by enabling quicker diagnoses for rare diseases and cancers and matching people to the most effective medicines and interventions.

We need to set in place the right framework across the entire health system to ensure patients, particularly those affected by rare cancers, can benefit from the promise of genomic medicine."

But, at a time when our NHS is already stretched and running at capacity, the arrival of new medicines, which may come with complex diagnostics and disrupt the established treatment pathways, will pose challenges. We need to set in place the right framework across the entire health system to ensure patients, particularly those affected by rare cancers, can benefit from the promise of genomic medicine. This means taking a critical but constructive eye to all aspects of our NHS and challenging our thinking on how we can drive efficiencies in clinics, free up expertise and ultimately create headroom for new innovation.

This isn’t something that can be done in isolation by the NHS alone, and nor should it be. Industry has an important role to play, in partnership with the NHS and government, to ensure our health system is best set up to adopt the innovation of tomorrow.

For example, we are still waiting to see how the NHS intends to address the current workforce challenge. It is vital that this plan is developed and implemented with the evolving care pathways of diseases like rare cancers in mind. This is where industry can support – at Pfizer our Oncology Patient Experience team runs projects with NHS trusts across the country which look at patient pathways. We help trusts to understand the pinch points in their service delivery, the areas where perhaps patient experience falls down, and importantly where changes to the workforce need to be made.

In areas like rare cancers, where we know patients are often isolated and have perhaps faced delays to receiving a diagnosis and care plan,1 the scope for these projects is significant and I really believe that meaningful industry/NHS partnership can transform outcomes for patients and clinics alike.

It is also important that the methods and processes for assessing the value of new medicines keeps pace with scientific advances so that breakthrough medicines (that have the potential to change lives) can reach those who need them.

The UK’s current health technology appraisal system can be problematic for medicines for rare and less common cancers. Data collection in small patient populations with rare tumour types is difficult, making it challenging to meet the requirements set by the National Institute for Health and Care Excellence (NICE) to demonstrate the benefit and value of a new medicine.7 The Cancer Drugs Fund has provided a conditional route for access when NICE deems there to be uncertainty around the cost-effectiveness of a medicine,8 but this is not necessarily a fix-all for rare cancers when the data available is simply too limited.

Additionally, it is increasingly common for cancer medicines to be used in combination with one or more other cancer medicines that together target a specific tumour mutation. The concept and potential of combination therapies is hard to dispute, but these medicines present more challenges for a system that expects the combined price of all medicines to meet a single cost-effectiveness threshold. For instance, if a new medicine prolongs survival, and consequently prolongs the use of an existing medicine it is used with, the combination of the two together may not be cost-effective even if one is priced at zero.9

We know that the uptake of new cancer medicines in the UK is lower than the European average and we know that patients in the UK often face delays in accessing the novel treatments that are changing patients' lives.10,7"

We know that the uptake of new cancer medicines in the UK is lower than the European average and we know that patients in the UK often face delays in accessing the novel treatments that are changing patients’ lives.10,7 Without meaningful reform to NICE’s methods and a pragmatic review of how we can improve the access environment in the UK, we risk cancer outcomes slipping further behind our European counterparts.

The upcoming review of NICE’s methods and processes over the course of this year for both its technology appraisal and its highly specialised technologies programme is a welcomed opportunity to get this right and all parties – industry, Government, the NHS and the patient and clinical community - should be involved.

There is no doubt that through pioneering science we are making great progress in the fight against cancer. But we need continued collaboration and investment in rare cancer treatment pathways and innovation so that the NHS is prepared for the new systems and technologies needed and patients can fully benefit.

This starts with partnership. It is promising to see the NHS pledge to build new strategic partnerships with research and industry colleagues and we are passionate about playing our part.11,12 Pfizer has been at the forefront of cutting-edge science and we are committed to working with the NHS and the government to ensure our healthcare system is best-placed to meet the UK’s ambition of leading the world in genomic medicine.

By working together and keeping our focus on the many patients we are all collectively here to help, this bold ambition can be achievable.



  1. Macmillan. What is rare cancer?[Last accessed April 2019]
  2. National Cancer Intelligence Network and Cancer 52. Rare and Less Common Cancers: Incidence and Mortality in England, 2010-2013. Public Health England. June 2015. [Last accessed April 2019]
  3. Rare Cancers Europe. The burden and challenges of rare cancers. [Last accessed April 2019]
  4. Cancer 52.  ‘Living with a rarer or less common cancer. Patient Experiences of treatment and care’ report, p7. [Last accessed April 2019]
  5. NHS England. NHS Long Term Plan. [Last accessed April 2019]
  6. [Last accessed April 2019]
  7. The Institute of Cancer Research. Cancer Drug Manifesto. [Last acccessed April 2019]
  8. NHS England. Appraisal and Funding of Cancer Drugs from July 2016 (including the new Cancer Drugs Fund). [Last accessed April 2019]
  9. Persson U, et al. Multi-indication and Combination Pricing and Reimbursement of Pharmaceuticals: Opportunities for Improved Health Care through Faster Uptake of New Innovations. Appl Health Econ Health Policy. 2018. 16(2): 157-165
  10. Jonsson B, et al. Comparator report on patient access to cancer medicines in Europe revisited – a UK perspective. IHE Report. 2017. [Last accessed April 2019].
  11. NHS England. NHS Genomic Medicine Service. [Last accessed April 2019]
  12. NHS. ‘Improving Outcomes through Personalized Medicines’ report, p8. June 2016. [Last accessed April 2019]


PP-ONC-GBR-0909 / May 2019