Thank you for the opportunity to speak to you today.

Patients Rising is a 501c3 not-for-profit organization with funding by public donations, media companies and the pharmaceutical industry. However, we have received no funding from the company whose drug is being reviewed here today, and therefore no conflict.

We are here because the patient interest is here … in this room. And so the patient voice must be heard … in this room.

Today we’re talking about two liver diseases: PBC and NASH. One disease is being treated effectively by current therapy for many patients. The other has no effective treatment for the most vulnerable cases. Both need medical innovation.

NASH is set to pass Hepatitis C as the leading cause of liver transplants by 2020. By 2025 more than 25 million Americans will be living with the disease, 10% of those being children; mostly Hispanic.

PBC and NASH patients are different, but they are now united in a common concern: Without ready access to the promising and precision medications that could literally save their lives some won’t be able to fight their disease, treat their disease or beat their disease.

Some patients are asking: How could this happen?

It can happen because of the onset of a conversation that is said to be about value … but whose value? Patients?

Because from what I saw in your opening slides you were talking about fire department spending in Massachusetts vs. healthcare.  Does this really have a place here?

Value frameworks like the one you are developing are by any objective measure meant to control access first. Not advance treatment first.

We are more than concerned that the imposition of a complicated math formulary on the only available treatments will affect millions of patients and ultimately medical innovation as a whole. For that reason alone, ICER should pause and reflect and reconsider. Anything short of that will be far less than patients deserve.

My friend Tina Dooley is a PBC patient from Houston. Were you to meet her, she might say, “Nice to meet you. I’m a .83.”

That’s her humorous way of dealing with a QALY system – the same one you are using. It calculates her to be worth less than a healthy person.

Tina has been living with PBC for 18 years.

Tina is a partial responder to UDCA. Partial. That means not enough.

Her ALP levels are not in line with those fully responding to the current standard of care. She is a rare subset of an already rare disease. And she’s not the only one.

The standard of care keeps her going, but for how long? What happens next?

Without the next advancement of medical innovation, she will suffer more. She may lose her liver. She may lose her life. She’s not alone.

Don’t people like Tina deserve better?

Of course they do. They deserve fair access to the best of medical innovation that is right for them. If we as a society focus on the precision capabilities medical innovation is giving us, figure out how to monitor the efficacy of these treatments in real world settings and ultimately create value structures that look at costs in totality across all sectors of healthcare (not fire departments) – now that is a care model I will stand with.

We can do better than the value structure being used in a country with the poorest overall cancer survival rates in Western Europe — We can win the economic battle without sacrificing the access to new therapies patients in this country need.

I can’t tell you how many times I read, “insufficient body of evidence” in your documents” – couldn’t that be because the more targeted our treatments become the more difficult populations will be to gather?

I’m an optimist. We as a nation lead clinical trial development. The pipeline of progress has never been more exciting for patients.

There are many neuances to patient value. I challenge ICER to be as innovative with defining value as researchers are with the science.

I was pleased to see your email yesterday calling on us to offer suggestions to your framework development. I’m an advocate for patients, and I do not claim to know the answer – but consider that my suggestion.

You have ready access to the best and the brightest minds this country has to offer – stakeholders will work with you for the good of patient access and not what can feel like formulary denial or arbitrary benchmarks that have yet to be truly established.

Tina questions ICER’s reasoning behind this hyper-focus on the first new available treatment for PBC patients in more than 20 years.

Her question is the same as so many patients: Is my life worth it?

Of course it is.

As for NASH patients, there is no effective treatment for those that do not show benefit from currently prescribed lifestyle changes. The therapy you are targeting today shows promise. Real promise. It is currently in a phase-three trial for NASH.

With all due respect, why are you Monday morning quarterbacking a game in the middle of the third quarter?

We call on ICER to reconsider and re-set.

I know there must be ways to amend your framework to put patients at the forefront not in the next room.

Try as you or anyone else might to squelch the right treatment for the right patient by creating frameworks that appear focused on cost above care — patients know the score.

That’s why they are rising against it at every turn. Their voices have value.

Patients Rising stands with them. We stand with every PBC patient and every NASH patient because now, they need it more than ever.

We ask you to do the same.

Thank you.