If you or your loved one have been given a rare cancer diagnosis, it can be difficult to imagine what the path ahead looks like and what potential treatment options are available. Rare cancer can leave you feeling uncertain and alone. Your cancer may need to be treated differently from other more common cancers, and there may be less information and fewer treatment options available to you. To help you find your way forward when navigating the NHS and beyond, we’ve put together an overview of the different schemes and treatment options available to cancer patients in the UK, so you can navigate your path with confidence.
What is rare cancer?
A cancer is considered rare if it affects fewer than 6 in 100,000 people per year. Due to their rarity, these cancers often have fewer treatment options and less available information compared to more common cancers. This can make diagnosis and treatment particularly challenging for patients and healthcare providers.
Examples of rare cancers include:
- Adrenal Carcinoma: A cancer that starts in the outer layer of the adrenal glands, located above the kidneys.
- CNS Lymphoma: A type of lymphoma that originates in the brain or spinal cord.
- Mesothelioma: A cancer that develops in the lining of the lungs, abdomen, or heart, often linked to asbestos exposure.
- Ocular Melanoma: A rare cancer that forms in the cells that produce pigment in the eye.
- Glioblastoma: A primary brain cancer that starts in the brain or spinal cord. which typically forms from glial cells.
Starting treatment options for rare cancer
Your treatment will be discussed by a team of healthcare professionals, called a multidisciplinary team (MDT). The MDT includes oncologists, radiologists, surgeons and specialist cancer nurses. They will review your medical notes, scans, biopsy results and blood tests to agree on an optimal plan, which they will then share and discuss with you.
Your plan will be based on the latest cancer guidelines and recommendations where possible but is usually limited to what has been approved for use within the NHS. For rare cancers, this can mean that the treatments available aren’t the most up-to-date, or newest available therapies, due to lack of clinical evidence to support the drug approval process.
You do not have to agree to any treatment if you do not want to go ahead. If you have any concerns or questions, it’s best to write these down on a piece of paper and take them to your next appointment.
Getting a second opinion
If you still aren’t sure about starting treatment, that’s okay. Starting cancer treatment is a big deal and you can ask for a second opinion. However, it’s best to ask for this through your specialist, as they can send your scans and notes to a second specialist who can give their opinion and potentially, but not always, take over your care.
You can also request a second copy of your results, notes and letters so you can have them to hand at any given moment, as it can often take time to get these documents from the health service.
Further, it is possible that this second opinion will not change your treatment much or at all, as with rare cancers, your specialist probably already discussed your case with several others during the MDT phase. However, this can still be a reassuring step to take.
Starting your cancer care.
On the NHS, your treatment will usually start within one month from your diagnosis, but in the case of rare cancers, this may be longer as there may be extra tests to help determine what the right course of treatment is.
Possible options if your treatment isn’t effective
It can be very worrying if your initial cancer treatment isn’t working, but there are some other options available. In the UK, the health service is continually striving to improve access to specialist care and experimental cancer treatment for rare cancers.
Below is a list of the potential pathways ahead. However, it’s important to note that the availability of these options may differ between individuals, depending on the type of cancer, stage, and other patient factors, as well as where you live.
Again, It’s also important to note that most of these decisions will need to be made with your specialist. They will need to agree that any cancer treatment options will have some clinical benefit or that it won’t do more harm than good.
Considering different treatment options?
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Early Access to Medicines Scheme (EAMS)
The EAMS is a pathway that can help patients access drugs that have shown some clinical evidence of benefit in research and clinical trials but haven’t been licenced for patients on the NHS yet. Because these drugs are experimental, there is a risk of side effects or a lack of effectiveness.
If you think you would like to apply for the EAMS you can discuss this with your doctor. If your oncologist agrees with the decision and there is a suitable drug available, they can apply to the EAMS on your behalf.
The Cancer Drugs Fund (CDF)
This is an alternative way for patients to access drugs that are not available on the NHS, The National Institute for Health and Care Excellence (NICE) assesses drugs to see if they should be available on the NHS. Sometimes, they will make a drug available through the CDF while more research is carried out before a decision is made to make it available through the NHS.
If an appropriate drug is available, your oncologist can apply to the CDF for you online. if the application is approved, the CDF will pay for the drug, which your oncologist will give to you.
If the drug isn’t covered by the CDF, your doctor may be able to apply for the individual funding request process.
Individual funding request (IFR)
If a drug is not available through EAMS or CDF, then your doctor may be able to make an individual funding request (IFR). Your specialist will apply for you, using information about you and your cancer diagnosis and treatment. If your application is approved by the NHS, a specialist panel of healthcare professionals will discuss your individual case and decide if the treatment options are appropriate and if the NHS will pay for it.
If successful, your doctor can prescribe the drug for you. If your application is unsuccessful, you may be able to appeal the decision.
The IFR process is slightly different, depending on where you live and there are slightly different processes between England, Wales, Scotland and Northern Ireland
Clinical trials
Clinical trials are medical research studies involving people with a certain condition. Usually, they are designed to test if the new treatment options have more clinical benefit. In some cases, they are designed to see if a new treatment is safe or whether there are side effects. Each clinical trial will have specific criteria and rules for patients who can enrol, such as medical condition, age or stage of cancer.
Your cancer doctor may offer you information about a clinical trial if they think you might be suitable. If you agree and are suitable for the trial, they will refer you to the trial for enrolment.
Alternatively, you can ask your doctor if there are any clinical trials, or search on databases such as Be Part of Research which provides information on healthcare research taking place in the UK.
Charities and organisations
There are organisations and charities which may campaign for your type of cancer, the treatment that you are having or patients with similar clinical circumstances. These organisations may be able to provide you with support and information to help you get access to care on the NHS or through clinical trials or payment schemes.
Private medical care
Navigating private healthcare options can seem daunting, but it’s important to know that private treatment can offer additional choices and faster access to certain services.
Doctors and NHS managers should exhaust all reasonable avenues for securing NHS funding before suggesting that you go private, this includes exceptional funding and local funding options available through your primary care trust. Only once these avenues have been explored should your doctor suggest that your only option is to pay privately for treatment.
If you have private health insurance, this may cover the cost of treatment, drugs and any care you receive in and out of hospital. You should check with your insurer to see whether you are fully or partially covered for these costs.
If you don’t have private medical insurance or your insurance doesn’t cover your treatment, you will need to self-fund. It’s good to be aware that prolonged cancer treatments and hospital stays can cost tens of thousands of pounds.
Mixing Private and NHS care
In some cases, you may wish to mix private and NHS care. There are guidelines to ensure that there is some separation of private and NHS treatment. It’s important to note that while you can mix NHS and private care, you cannot have the NHS pay for or subsidize any part of your private treatment. Additionally, you can’t mix different parts of the same treatment plan between NHS and private care
Opting for private treatment for one aspect of your care does not affect your right to NHS treatment for other aspects. If you receive private treatment, you can still return to NHS care at any point, for example if you can no longer afford to receive private care.
Questions you might want to ask your doctor
When facing a rare cancer diagnosis, having open and informative discussions with your healthcare professional is crucial.
Below, we’ve prepared a list of questions you might want to ask your GP or specialist to better understand your diagnosis and treatment options. Take these to your next appointment and discuss them with your oncologist.
About Your Doctor’s Experience
- How many patients with this rare cancer have you previously treated?
- Is it possible to get a second opinion from a specialist in this cancer type?
About Treatment Options and Plans
- What treatment options are available to me?
- What will my treatment plan include?
- What is the likelihood that this treatment will be effective?
- What are the primary goals of my treatment?
About Support and Resources
- Will I be assigned a specialist nurse to support my care?
- Are there any specialist centres that focus on my type of cancer?
- Where can I find reliable information about my specific type of cancer?
- Are there any patient organisations or support groups for this type of cancer?
About Advanced Treatment Options
- Are there any clinical trials for my cancer type that I could participate in?
- Am I eligible for the Early Access to Medicines Scheme (EAMS)?
- Can we consider any drugs available through the Cancer Drugs Fund (CDF)?
- Is it possible to apply for an Individual Funding Request (IFR) for my treatment?
- What are the risks and benefits of using experimental treatments not yet approved by the NHS?
Navigating your way forward
Understanding the treatment options and schemes available within the NHS and beyond can provide a clearer path forward. There are several avenues to ensure you receive the best possible care. By asking the right questions and staying informed, you can make empowered decisions about your treatment and care, bringing confidence and clarity to your journey. Always remember to seek support from healthcare professionals and patient organisations to help guide you through this process.
At Gena, we’re here to help navigate the complexities you might face trying to access personalised therapies. If you do decide to seek treatment beyond the standard of care we can help match and connect you to personalised treatment options. Contact us here to find out more.
Remy is a dedicated physician and medical writer. With over a decade of clinical experience, he has worked globally – from clinics in South Africa and Brazil to research collaborations with universities in London and Cape Town. Remy completed his Medical training at University College London.
A seasoned medical writer and published researcher, Remy’s work has appeared in prestigious journals such as Nature and magazines like Woman & Home. He has collaborated with the Bill and Melinda Gates Foundation the World Organization of Family Doctors (WONCA) and Johnson & Johnson.
Remy’s mission at Gena Health is a personal one. He was inspired to join Gena by his cousin’s battle with neuroblastoma, a rare cancer she fought bravely before passing away at just 16. He is committed to educating people on issues related to rare or hard-to-treat cancers, and making the world a kinder and more hopeful for patients and their families facing similar challenges.
