Supporting the

Neuroendocrine Cancer Community

A new route for NET Ambassadorship recently launched

Feb 13, 2019

“A fabulous account of Mark’s venture into the new patient involvement panel for NICE. Really worth a read and I am totally behind talking about the Chair’s role in an appraisal meeting. It is a daunting experience without the Chair dismissing your comments, and I will never forget at the Lutetium meeting when I presented some factual data and the chair said ‘ well I do not think you have that quite right do you”, in a very derogatory way – To coin one of Mark’s phrases; I got ‘my assed kicked’ too! A change is needed and brilliant that we have such a valuable NET ambassador to speak on the behalf of our community.
Thank you Mark for your great work”. – Cathy, NPF CEO

2018 was a good year for NET patients, as key treatments received authorisation in the USA, Europe, and the UK. Lanreotide, Sunitib, Everolimus, and Lutathera (Lu-177) PRRT.

A major effort with Lutathera received partial positive guidance in August 2018, but there are still patient groups on the edge of approval. Specifically; higher proliferation index patients and those needing retreats. We’ve still got work to do. This may be difficult, as the data wasn’t really part of a major clinical trial like NETTER-1.This data deals with lessons learned since.

The good news is that we are very much wiser with the lessons learned since the PRRT was removed from NHS in 2015. We have momentum again, after three years of dealing with NICE appraisal process.

You may have noticed I used a new title “ambassador” above. It’s a little different from advocate, although the goals are the same. At the end of the day, both try get the right treatment to every patient. Advocates do this by raising awareness, gathering support, and getting into the weeds of the processes to clear pathways to specialists and access to drugs.

An “ambassador” is looking to build diplomatic relationships in the cancer community, and look for ways to pick up and pass on lessons learned.

As an ambassador, it allows for building connections between NHS patients, UK private patients, and foreign patients. The model of NET worldwide is better than any other cancer network I’ve seen.

For example, building a relationship with Breast Cancer Now to learn about their appraisals, and Prostrate Cancer UK to pass on information for their new clinical trials of Lutetium PSMA.

Some of the other relationships built during the NICE appraisal process were with NICE, AAA, advocates, patient inputs, private healthcare, with NET healthcare professionals as part of the same team, and our own patient foundation charity.

It was very complicated, but we know a lot more about process (and these processes are not just with NICE and the NHS, they are with international and across public and private schemes). We have not mastered these yet, but we are in very good shape. With probably the best lesson learned being we can work together to get things done.The Lutathera appraisal proved it.

One of the connections made with NICE during the appraisal was with their “public involvement” team. Who have launched an initiative to bring more meaningful patient participation into NHS decisions. Not just appraisals, but across the board to include guidelines for diseases like diabetes, arthritis, waiting times, access, and within budget constraints.

As a result, we have a NET patient as part of a small steering group to lay the foundation.

The small steering group is part of the working group of about 30 stakeholders, and includes cancer charities (NET, Breast Cancer Now, Cancer52, Prostate Cancer UK), Genetic Disorders UK, Alzheimer’s, Diabetes, and NICE appraisal “lay members”.

The first meeting was a fantastic day! It was very professional, well planned, and Karen Facey (PhD) did a super job as facilitator.

I’ve been reading her book, and the day pretty much went right down the same path of her theory of public involvement in the HTA process.

After wrestling with the “Top 2” objectives we identified during the “outside the box” exercise. Here are my inputs to towards the event report. I hope it gives you an idea of how I view the power and role of a patient ambassador.

Number 1 – Better defined QoL. (How can we accurately reflect the importance of QoL)

I absolutely agree this should be our focus. QoL is part of the formula the committees use to decide on guidance. Currently, QoL is an index between 0 and 1. A tenth of a point can reduce the ICER by thousands. I believe the role of the patient expert is to justify the QoL index being used. In my opinion, this is really what a submission is for, but there is a disconnect between that submission and the actual ICER calculations. As a result, no one I have talked to knows what a “.7” means. The result is that both drugs get a .7 score even though everyone knows they aren’t the same. This is something we can fix. Karen’s book gives examples of different QoL indexes for different diseases. Patient support charities, HCP’s and patients need to own this data.

HINT & TIP: This may not be as hard as it seems. I’ve looked at EQ-5D-5L, and it is almost insulting at how simple it is. (It only takes 2 mins, click here, I spent weeks trying to get a license to demo it, and finally my requests for access were pushed to the receptionist who told me “Mark, the demo is the questionnaire”).

Here is a very basic explanation of the ICER (incremental cost effectiveness ratio) used in the NICE appraisal process. The rough ICER limit of approval is £30K per additional Quality of Life year (called a “QALY”). The formula is ICER=((QoL against existing treatment) x (progression free survival))/cost.

This is basic! It quickly gets complicated with what is included in the cost, (this is a discussion that needs to happen, but for now let us leave this for another day).

Example of QoL & Patient power. To illustrate my point (by doing math) if you raised the QoL index “Targeted Radiation Therapy” being appraised up by .2 points (compared to chemo for example and an extra 14 months of PFS) this would drop the ICER from £40K to close enough to £30K. This would take the discussion from a “not likely” to a something the committee can consider.

I used this example to get respect from the healthcare professionals and drug company. It is simple, but I think it would be quite an accomplishment to see a scientist in the lab come up with the same kind of results.

The power of the patient is that we are the only people in the room that can tell the difference between radiation sickness, cancer, blood sugar, diet, supplements, cholesterol statins, gout, exercise, and emotional & cognitive impacts. In my opinion, this is our core strength.

Requirement: Accurate QoL data is required to be able to make the right decision on the appraisal (HTA). Specifically, the QoL Index in the ICER needs to be accurate and current.

Recommendation: Patients own the QoL piece of the formula. A possible collateral benefit may be savings for the NHS on paying for assessment groups and/or legal fees.

Number 2 – Appraisal/Committee Chairs/Heads should be “facilitators” not HCP.

I’m not 100% behind the way it was portrayed, but I’m glad this was a “top 2”, because this was the elephant in the room. I saw this as a combination of issues dealing with personality, disrespect, dictatorial behaviour, been in the role too long, or didn’t know how to facilitate. I just feel we may be painting this role with the same “dehumanising” brush as we feel we are receiving.

What impact would this have if NICE “public involvement” took a picture of this statement written on the flip chart as written and forwarded as our recommendation? To me, this just seems incongruent. Another caution I’d like to make; this wasn’t written as an issue, it was written as a fix.

What is the requirement we are really trying to identify? I believe it is the effectiveness (worthiness) of committee members needs to be right for making the best decision on an appraisal. This includes all the players: The Chair, lay- members, patients, charity representatives, medical experts, and committee members (including health economists).

If I empathise with the Chairs, I’d see a professional who has dedicated their life to healthcare. They are the gatekeepers of the NHS, and charged with taking care of patients and the budgets. This responsibility is huge. I’d probably be just as big a “dictator” as what I saw on the chair of our committee. Especially, if I was faced with deciding the fate of thousands of patients and families when the data present to me included ICERS of £52K from an assessment group, and £27K from the drug company. If one of my military commanders had been put in the same spot, they would have exploded. But, I saw this “dictator” have the backbone to bend the rules just enough to get the right decision made. If this was a “professional facilitator”, I’m not so sure you’d have that flexibility. We don’t have to look very hard to see what happens when non-HCPs get involved. The legal wrangling in the news is costing the NHS a lot of resources going back to define the requirements.

When I empathise with the patient “experts”, we have to overcome a big hurdle in that arena. For me, this was “worthiness”. I hadn’t been to medical school (my step daughter has just completed by the way, and it looks pretty damn hard). Who did I think I was? I didn’t know what my team was, or what a win looked like. I felt quite alone. All I had was a deep belief that the treatment that helped me needed to be made available to everybody.

What helped me with this issue was a lot of hard work on dealing with my own worthiness issues, and to separate the appraisal from my ego. After a lot of work, it became clear to me that there were only two people in that room that were free to point to the “elephants” without a conflict of interest. The Chair and me.

The Chair may not have realised it, but I was actually a lot of help to the committee as an ambassador (this is the term Karen Facey uses in her book as well). The medical experts, the charity organisation, and even the drug company could talk to me in a way they could not talk to the committee. I could in turn use diplomacy to question and inform. It wasn’t a deciding factor, but I’m convinced it was crucial to getting the right decision made, which, has only been partially made for us so far as my appraisal still has work to do.

I was fortunate enough to be given a second chance. The first meeting I attended I was not aware of this power, and (please excuse my language) I got my ass kicked in the first appraisal.

A large portion of Karen Facey’s book deals with this issue. From the cultural divide of HCPs and patients to the “tokenism” and “worthiness” of patients in HTAs. I’m still reading her book, and so far, she doesn’t claim to have the answer, but she has got the core issue in print.

Dr. Brene Brown’s Ted Talks and books “Rising Strong” and “Dare to Lead” do a really good job of explaining what I’m trying to apply. If you aren’t familiar with her stuff, here is a short 3 min introduction. 

The “man in the arena” is not just the patient, but the Chair as well.

Requirement: The patient and the Chair need an effective connection. The scope for the appraisal needs to be clear with “buy in” from all parties.

Recommendation: Pick models of how this has been done effectively and ineffectively. Identify core issues. Use the patient submission as the core document between the Chair, patient, and committee with the same importance level as the economic levels of the drug company & assessment groups and the HR & survivability data presented by the HCPs.

For example: The patient submission is about QoL.

[ICER = (QoL Index x PFS)/cost] or put in lay terms [decision made = (patients x HCPs)/drug companies]

I’m very optimistic about what we have initiated, and I’m looking forward to our next meeting. I’ll do my best to pass on the progress. If you have any ideas, please pass them.

Thank you,

Mark Zwanziger
NET Patient Ambassador