Neuroendocrine cancer and radioligand therapy: how patient involvement can shape the regulatory process

9 November 2021

My name is Nikie Jervis and I have been supporting people with neuroendocrine cancer for almost 30 years, working as a Patient Support and Information Nurse Specialist at Neuroendocrine Cancer UK (NCUK) since 2015.

The cancer treatment landscape is changing; knowledge about how cancer develops is improving and new ways of treating the condition are being discovered. A key part of system readiness preparation is being able to assimilate new developments into practice.

Radioligand therapy is an innovative method for targeting malignant cells that improves survival for people diagnosed with cancer. According to Josh Mailman, President of NorCal CarciNET Community, radioligand therapy isn’t that new, but its expansion across different types of cancer and its incorporation into routine cancer treatments is.

How can we increase the use of radioligand therapy?

During my career, I have seen a number of significant changes in cancer care, not least the exponential growth in incidence of neuroendocrine cancer.

Neuroendocrine cancer and radioligand therapy

I have also seen the progression of radioligand therapy and its incorporation into expert consensus guidelines for neuroendocrine cancer care. However, there are limitations and barriers to expanding the use of radioligand therapy and delivering it to people who will benefit from it.

The therapy’s successful integration depends on addressing factors outlined in the Radioligand Therapy Readiness Assessment Framework, which includes regulatory recommendations for use. In the UK, the National Institute for Health and Care Excellence (NICE) is responsible for providing guidelines with recommendations for improving health and social care. For me, a vital component of NICE’s appraisal and recommendation process is the involvement of patients.

Radioligand therapy and the UK appraisal process

The Cancer Drugs Fund (CDF) is a source of funding for cancer drugs in England, which provides people with access to promising new cancer treatments in certain situations before full approval by NICE. In 2015, the CDF stopped funding for lutetium-177 dotatate radioligand therapy for neuroendocrine cancer. At the time, the treatment was at an important point in the approval processes for both the US Food and Drug Administration and European Medicines Agency.

Lutetium-177 dotatate, however, had not been put forward for NICE review, and could only be appraised if referred to the body by the Secretary of State for Health and Social Care through a process called topic selection. Patients, NCUK and clinicians joined forces to advocate for the selection of the therapy as a topic for consideration. This ran concurrent to pharma’s submission of data and trial information.

In 2016, NICE announced that lutetium-177 dotatate would be included in an appraisal of treatments for people with neuroendocrine cancer.

Mark’s story

In 1998, Mark started to develop symptoms, but he was not diagnosed with neuroendocrine cancer until 2007. He had surgery in 2008 and went on to manage his cancer with somatostatin analogues until 2011, when surveillance imaging revealed progressive, inoperable disease.

Mark was halfway through radioligand therapy when CDF funding was stopped – he was stunned: ‘this just couldn’t be right.’ He was first in line to apply to be a patient expert on the NICE panel for appraisal, and joined the panel in 2016. Mark was galvanised and, with support from NCUK and clinicians, set about giving a number of presentations to the neuroendocrine cancer community to encourage patient involvement in regulatory processes. He highlighted that ‘there were only two people in the appraisal room who could make points to anyone – one was the head of the panel and the other was the patient . . . we have that power’.

The first round with NICE, however, resulted in a negative ruling on lutetium-177 dotatate. This ‘appraisal looked like we had missed our opportunity … it felt like we had missed a “2-foot putt”. As a team and a NET [neuroendocrine tumour] community we were devastated.’

But all was not lost: NICE proposed a re-evaluation of lutetium-177 dotatate in 2017 which included a submission from Mark, as well as trial data. In addition to demonstrating his understanding of the cost-effectiveness of the therapy, he shared what he called a ‘quality-of-life profile’, discussed the radioligand therapy he had received and explained what he thought was possible for his future. Mark was also involved a NICE initiative to improve patient involvement, sharing what he had learnt with fellow patients.

In 2018, lutetium-177 dotatate radioligand therapy for neuroendocrine cancer was recommended by NICE. Mark said that his involvement in this ‘was one of the greatest experiences’ in his life.

Many issues remain, however. Based on his experience and discussions with others in the neuroendocrine cancer community, Mark made several recommendations. For effective patient involvement, regulators must:

  • include patients ‘from the get go’
  • use up-to-date and expert-led scoping documents to inform decision-making
  • include patient and clinician engagement meetings (for example, those held by the Scottish Medicines Consortium)
  • clearly communicate the cost/benefit of a treatment
  • use disease appropriate quality of life assessments, in order to show how the treatment can impact quality of life
  • use real-world data, as clinical trials do not show the whole picture of how a treatment can be used
  • give patients an equal stakeholder standing.

After Mark passed away in 2019, a fellow patient thanked him ‘for the incredible work he did to help persuade NICE about reinstating funding for PRRT [peptide receptor radionuclide therapy].’

Neuroendocrine cancer and radioligand therapy


Nikie Jervis, Senior Patient Support, Information Nurse Specialist and Education Lead, Neuroendocrine Cancer UK