Appendix G – patient/carer organisation submissiontemplate

NATIONAL INSTITUTE FOR HEALTH AND CAREEXCELLENCE

Patient/carer organisation submission (STA)

Nanoliposomal irinotecan for treating pancreatic cancer after prior treatment with gemcitabine [ID778]

Thank you for agreeing to give us your views on this treatment that is being appraised by NICE and how it could be used in the NHS. Patients, carers and patient organisations can provide a unique perspective on conditions and their treatment that is not typically available from other sources. We are interested in hearing about:

  • the experience of having the condition or caring for someone with the condition
  • the experience of receiving NHS care for the condition
  • the experience of having specific treatments for the condition
  • the outcomes of treatment that are important to patients or carers (which might differ from those measured in clinical studies, and including health-related quality of life)
  • the acceptability of different treatments and how they are given
  • expectations about the risks and benefits of the treatment.

To help you give your views, we have provided a questionnaire. You do not have to answer every question — the questions are there as prompts to guide you. The length of your response should not normally exceed 10 pages.

  1. About you and your organisation

This is a joint response from Pancreatic Cancer UK and Pancreatic Cancer Action

Your name:Leah Miller
Name of your organisation:Pancreatic Cancer UK
Your position in the organisation:Campaigns and Policy Officer

Your name: Ali Stunt
Name of your organisation: Pancreatic Cancer Action
Your position in the organisation: Founder and CEO

Brief description of the organisation:(For example: who funds the organisation? How many members does the organisation have?)

We are asking for your collective view as an organisation and will be asking patient experts for their individual input separately. If you have the condition, or care for someone with the condition, you may wish to complete a patient expert questionnaire to give your individual views as well.

Pancreatic Cancer UK is fighting to make a difference. We’re taking on pancreatic cancer together: by supporting those affected by the disease, investing in research, lobbying for greater recognition of pancreatic cancer, and being there for everyone involved in the fight.

We provide a UK-wide, expert and personalised support and information service, staffed by pancreatic cancer specialist nurses. This provides easy access to the best and most up-to-date information on pancreatic cancer to patients, their carers and families. We also run online discussion forums for pancreatic cancer patients, their families and carers to enable them to share experiences, information, inspiration and hope. We fund innovative research that makes the most impact with limited resources and leverages additional investment. Working closely with patients and their families and carers, clinicians and other healthcare professionals, researchers, politicians and policy makers, we seek to increase awareness of the disease and campaign to bring about improved outcomes in care and treatment.

Our funding comes from a variety of sources, although mostly from small donations and fundraisers. In 2015/16, 0.89% of our income came from pharmaceutical companies in the form of grants supporting our education work such as Nurse Study days etc. Full details of pharmaceutical contributions are available on request. Our policy is that pharmaceutical funding must not exceed 5% of our total budgeted income of the financial year and that any monies received cannot be used for campaigning.

Pancreatic Cancer Action is a national charity focussed on giving every pancreatic cancer patient the best chance of survival by improving earlier diagnosis and treatment.

Set up by a pancreatic cancer survivor, we raise awareness among the public and medical communities, fund research to improve early diagnosis, provide information for patients and develop educational courses for clinicians.

The majority of our funding comes from individual donors and supporters, most with a very personal connection to pancreatic cancer. While we do receive funding from pharmaceutical companies, the total amount we received equated to a mere 0.4% of our total revenue in 2014. In 2015, while campaigning to keep the drug Abraxane® on the Cancer Drugs Fund list, Pancreatic Cancer Action made a conscious decision to refuse a grant from that drug manufacturer, Celgene even though the grant was not linked to any campaigning activity.

Links with, or funding from the tobacco industry - please declare any direct or indirect links to, and receipt of funding from the tobacco industry:

Neither Pancreatic Cancer UK nor Pancreatic Cancer Action receive any funding – be it direct or indirect – from the tobacco industry.

  1. Living with the condition

What is it like to live with the conditionor what do carers experience when caring for someone with the condition?

Receiving a diagnosis of pancreatic cancer can be a devastating and bewildering time for patients and their family members. Pancreatic cancer patients often have complex supportive care needs, including support dealing with pain management, weight loss, nutritional issues, depression and other emotional and psychological needs.

A diagnosis of pancreatic cancer for many is seen as a death sentence with an average life expectancy among metastatic patients of two to six months. Patients often report feeling helpless and without hope due to the lack of effective treatment options available.

Being diagnosed with a disease that has such a poor prognosis is extremely difficult for both patients and their loved ones to deal with. In a 2014 survey (n=130) run by Pancreatic Cancer UK and Pancreatic Cancer Action asking how patients and their family members felt on diagnosis, respondents most commonly reported feeling “devastated”, “alone”, “helpless”, “scared”, “shocked” and “completely without hope”.

As such, the psychological impact of a diagnosis of pancreatic cancer can be significant. We know from conversations with patients and carers, through calls made to the Pancreatic Cancer UK Support Line, and from participation in both organisations’ patient and carer forums, that a diagnosis of pancreatic cancer can lead to depression.[1] Simply increasing the treatment options available to patients can also help relieve some of the psychological impact of diagnosis by giving patients a new hope.

There are also many physical symptoms and side-effects associated with pancreatic cancer and treatment.For example, patients may experience symptoms related to diet (including Pancreatic Enzyme Insufficiency and diabetes); nausea and vomiting; changes to bowel habits; chronic fatigue; neuropathy; alopecia and pain.

These symptoms and side-effects can have a significant impact on quality of life for both patients and carers. Patients and families often report that they find themselves unable to carry out simple day-to-day activities, with many patients and carers forced to give up work:

“I had to give up work to care for her, we all felt like a time bomb waiting to go off. I think we all felt like we were given a death sentence.”(Carer quote from 2014 survey)

  1. Current practice in treating the condition

Which treatment outcomes are important to patients or carers? (That is, what would patients or carers like treatment to achieve?) Which of these are most important? If possible, please explain why.

Pancreatic cancer is the fifth leading cause of cancer death in the UK[i] and has the worst survival outcomes of any of the 20 most common cancers, with a UK 5-year survival rate of less than 5%[ii] (5.4% in England in 2014[iii]) and a ten year survival of less than 1%[iv]. Metastatic pancreatic cancer patients have a median survival of between just 2 – 6 months.[v]

Pancreatic cancer is not a rare cancer – around 9,400 cases were diagnosed in 2013[vi] - and yet there are very few treatment options available. Surgery provides the only hope of a cure, and the best survival outcomes, and yet only around 10% of patients are eligible for surgery in the UK[vii], largely because of late diagnosis of the disease.

This means that non-surgical treatments are of huge importance to the vast majority of pancreatic cancer patients. However, at the current time there are very few treatment options available.

Given those statistics, it is perhaps unsurprising that both Pancreatic Cancer UK and Pancreatic Cancer Action find from patient surveys, our forums and conversations with patients and carers,that extending overall survival is usually the number one, most desired treatment outcome.

Also of great importance is how a treatment can help manage or control side-effects of the disease itself.

A separate issue is how manageable the potential symptoms and side-effects from a treatment will be, and the impact these will have on quality of life,and this is also of significant consideration for patients.

It is important to note that individual patients weigh these considerations slightly differently. That is why as patient representative organisations we constantly stress the need for patient choice and as wide a variety of treatment options for clinicians and patients as possible.

From our work with hundreds of patients and carers each year, we know that patients want to be the ones to make that choice of having a life-extending treatment, even if it often means that treatment has significant side-effects.

Individual patients will have different levels of tolerability, both from a physical and psychological perspective but also based on their personal or family circumstances.

Both Pancreatic Cancer Action and Pancreatic Cancer UK firmly believe treatment decisions for metastatic pancreatic cancer should be about providing an informed choice for patients who, knowing the possible side effects of any given treatment, will then decide if they wish to undergo the treatment concerned.

In view of the limited number of treatments currently available for pancreatic cancer patients, we believe that it is vital that all treatment options should be made available to patients on the NHS no matter where they live.

The previously mentioned joint Pancreatic Cancer Action/Pancreatic Cancer UK survey of 2014 found that over 80 per cent of patients, carers and family members would want to see a new drug that offered an extra two months’ survival gainapproved for use on the NHS, without reservation (the background to the survey at that time was nab-paclitaxel being appraised by NICE).Only 1% of respondents said they would not want the treatment to be made available, based on reported side-effects. The remaining 19% said they were unsure, with the reason for their uncertainty being that they would want to assess the likely side effects with their families and doctors. However, even in those circumstances, respondents made it clear they felt that patients should have a choice and that the treatment should be made available on the NHS.

Given the potential life-extending benefit of nanoliposomal irinotecan, as displayed in the NAPOLI-1[2] trial, has also been shown to be around two months vs the control of 5FU and Folinic Acid, we felt it appropriate to reproduce some of the comments from that 2014 survey here:

“Time is precious and having more time with family means more than anything”

“I would give anything for two extra months with my wife and daughter”

“It would give me two more months to support my children at a critical stage in their lives”

“Two more months to any person with a terminal illness – is a long time, a bit of hope, precious”

In our experience, we know that the majority of patients will, even when faced with potentially severe side effects, try the treatment if they are eligible. And should the side effects become intolerable, they will cease treatment or look for an alternative.The lack of treatment options currently available to pancreatic cancer patients means many are left feeling there is no choice for them.

“To have had another option which could potentially extend [my husband’s] life would have given us hope. The utter despair when told there is nothing really on offer cannot be put into words.”(Carer quote from 2014 survey)

In particular there is currently no recognised standard of care for second line treatment of metastatic pancreatic cancer in the UK, let alone a licensed option. A NICE approval of nanoliposomal irinotecan would therefore be of particular importance, providing an extra option – above and beyond the limited off-label treatments - for patients who had had prior treatment with gemcitabine.

What is your organisation’s experience of currently available NHS care and of specific treatments for the condition? How acceptable are these treatments and which are preferred and why?

Surgery followed by chemotherapy remains the only option for a cure. However, although it is estimated that whilst about 20% of patients diagnosed with the disease may be eligible for surgery, less than 10% go on to have it[3].

For those patients with metastatic pancreatic cancer, there are more limited options. Currently, the only standard treatment is with single agent gemcitabine, which is the only treatment approved by NICE, for patients with a Karnofsky performance score of >50. It is not approved by NICE as a second line therapy. Gemcitabine has proven to offer a modest survival benefit (median 7 months) as well as symptom control. However, currently only approximately 10% of patients will respond to gemcitabine chemotherapy[4].

An alternative is gemcitabine combined with capecitabine. This involves taking capecitabine in tablet form at home in addition to the administration of gemcitabine. Studies have shown that gemcitabine used in combination with capecitaine offers modest improved survival of 0.9 months compared to gemcitabine alone.

Currently, oncologists’ preferred first line treatment for pancreatic cancer is FOLFIRINOX, which is used off label. Evidence suggests that it provides the best overall survival outcome, around four extra months compared to gemcitabine alone, and a total of around 11 months on average[5]. However, this treatment is extremely toxic and only patients with a very high performance status are eligible for this treatment.

Importantly, and of most relevance to this technology appraisal, there is no recognised second line treatment option for metatstatic pancreatic cancer patients who have previously received gemcitabine-based therapy, let alone a licensed option,although Oxaliplatin in combination with flurouracil and folinic acid (FOLFOX) is often used as an off-label, second-line treatment where patients are fit enough. However, clinical trial data on the survival benefit of FOLFOX as a second line for metastatic cancer is ambiguous. Whilst the CONKO[6] trial shows an overall survival benefit similar to that shown by nanolipsomal irinotecan in the NAPOLI-1 trial, a separate trial, PANCREOX, concluded that there was no benefit to FOLFOX vs 5FU and folinic acid alone[7].

A NICE approval of nanoliposomal irinotecan would therefore be of particular importance, providing an extra option – above and beyond the limited off-label treatments - for patients who had had prior treatment with gemcitabine. This might be as a second line treatment for patients who have been treated with mono-gemcitabine therapy and who have responded well; for those who have been treated with gemcitabine-based combination therapy; or perhaps those who have previously been treated with gemcitabine as an adjuvant or neo-adjuvant therapy following surgery.

As such we welcome the development of nanoliposomal irinotecan, which robust trial data has shown offers a significant survival benefit, as well as a manageable safety profile[8]. We hope that the NICE Technology Appraisal will result in a positive recommendation for this new treatment.

Yet again, as patient representative organisations we would also stress the importance of choice, for both patients and their clinicians, in ensuring that treatment is best suited to their needs.

  1. What do patients or carers consider to be the advantages of the treatment being appraised?

Benefits of a treatment might include its effect on:

  • the course and/or outcome of the condition
  • physical symptoms
  • pain
  • level of disability
  • mental health
  • quality of life (such as lifestyle and work)
  • other people (for example, family, friends and employers)
  • ease of use (for example, tablets rather than injection)
  • where the treatment has to be used (for example, at home rather than in hospital)
  • any other issues not listed above

Please list the benefits that patients or carers expect to gain from using the treatment being appraised.

There is little widespread patient or carer knowledge of nanoliposomal irinotecan at this time, as it currently has no marketing authorisation in the UK. However, as patient representative organisations, both Pancreatic Cancer Action and Pancreatic Cancer UK have worked to inform patients and carers of the drug, its benefits and side effects as reported in trials. Some patients and carers are also aware of it having been already approved for use in the US and welcome the treatment’s reported life extending qualities. The following areas highlight the main benefits patients have told us they expect from the new treatment.