Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
The Kilimanjaro Climb to Fight Cancer was a marvelous experience. In every way.
Weeks later, I’m still absorbing what it means to scientists at Fred Hutch, and to the biotech community.
To start, consider the numbers:
Equally important, if not moreso, were the things you can’t quantify.
The relationships formed on this climb were meaningful. They will last a lifetime.
As you can see in the KOMO-TV report below, and hear in the satellite-phone audio reports below, this team was imbued with a spirit of kindness and generosity. There was joy and good humor. The Tanzanian crew that supported this trip touched us all deeply.
As Bob More of Alta Partners, one of the climbers, said on summit day: “I often tell my children that life is about three things. It’s about relationships, experiences, and being part of something bigger than yourself. This trip has all three.”
Now at home, many of the climbers are trying to apply some of the spirit from this trip to their everyday lives. They are organizing mini-reunions on their own. I don’t know how you measure the impact of such a thing, but I’m excited to see where this leads.
Count on me to continue climbing mountains to support the cancer research cause. A big part of my task will be mobilizing the biotech community to give back.
To all who supported this Climb to Fight Cancer campaign — thank you from the bottom of my heart.
— Luke Timmerman
KOMO News, Aug. 16, 2019
Eric Murphy, lead guide: Ladies and Gentlemen. Friends. Family and loved ones. It’s your favorite team — Climb to Fight Cancer!
We’re giving a shout from our first camp at Machame on Kilimanjaro. It’s July 20th.
We were fortunate enough to make our way from Arusha, at the hotel, to the mountain. It’s our first day of walking. We went from 6,300 feet, and walked our way up to 9,900 feet at the Machame camp, at the edge of the heather and forested moorland zone. We had a bit of late start, rolling in right about dark with a little song and dance.
We are a team of 29 individuals. A good-sized group. We have approximately 115 Tanzanian staff.
We are all doing fantastic. Very happy to be here.
Eric Murphy (left), Kilimanjaro lead guide, Alpine Ascents International. Photographed with me at Machame camp, Kilimanjaro, 9,900 feet. July 20, 2019
Luke Timmerman: Hi, this is Luke Timmerman, team captain of the Kilimanjaro Climb to Fight Cancer. Here at our first camp, 9,900 feet of elevation on Kilimanjaro.
I want to say Thank You to all the donors out there who made this campaign a huge success. We’ve raised $1.5 million for cancer research at the Fred Hutchinson Cancer Research Center.
And we’re not done.
I have a little tidbit of breaking news. One of our climbers, Dan Bradbury, tells me he just got a donation from Goldman Sachs for $5,000. He found out about it via text message when he landed here in Tanzania.
So, for those of you who have heard about this expedition, and still would like to contribute, all of our climber pages – all 27 of us – still have active fundraising pages.
Go to fredhutch.org/climb.
Now, as for this expedition itself — we are having a wonderful time. The conversations people are having — on the trail, in the hotel, around camp – it’s something special. We are enjoying the Tanzanian culture. The food, the hospitality, the service. It’s exceptional. The people are wonderful.
It was a little bit emotional for me to see all of this come together. We’re making something special happen. I really appreciate all of you out there who have helped make this possible.
Thank you very much.
Eric Murphy, lead guide: Hey everybody, it’s your favorite team: Climb to Fight!
[Cheers]
It’s the 21st of July. We are now located at 12,500 feet. On the broad expanse of the Shira Plateau. We are doing fantastic. We had about 5.5 to 6 hours of climbing today. Everybody did wonderful. We had great weather. Lots of sunshine. Good food.
We just celebrated Luke’s birthday with a cake.
[Cheers]
Everyone is doing fantastic. We thank all of you for following along.
Nina Kjellson: Hi, this is Nina Kjellson, one of the climbers on the team.
Nina Kjellson, general partner, Canaan Partners
Today we had some minor rock scrambling. It was a beautiful, beautiful day.
Tomorrow gets a little more real. It will be 8-9 hours, going from our current 12,500 feet, up to over 15,000 feet, then back down to 13,000 and change. So we prepped today for altitude sickness. What to look for, and what we’ll do about it. There were definitely a lot of questions, and some butterflies.
Climb high, sleep low – and slow, slow – are our mantras.
We have each others’ backs.
As Eric said, we had a marvelous celebration of Luke’s birthday, including a great hot meal. And fabulous cake.
My reflections on this trip so far are that it has exceeded every expectation. Luke and a couple of us had a strong commitment to making this a gender balanced team, which we did. We are 13 women and 14 men. Beyond that, we also span in age from the early 30s to the early 60s. We represent a broad span of backgrounds, geographies, and current biopharma gigs. We are definitely common in purpose and curiosity. And definitely common in our nerdiness.
Nina Kjellson (left), taking a break on Kilimanjaro with Katrine Bosley and Zoe Barry.
Our conversations have spanned from T-cell biology and the microbiome to business strategy and culture and leadership, as you might expect. But we’ve also discussed music, hobbies, travel, literature, family life, practical jokes, and of course, the potty humor that comes with bodily changes at altitude and eating different foods.
We’re having a lot of fun, and truly connecting.
For all of you out there sponsoring us, supporting us, and wishing us well — Asante Sana!
That’s Swahili for Thank You Very Much.
Eric Murphy, lead guide: Friends, family, loved ones, sponsors. It’s Team Climb to Fight!
[Cheers]
It’s the 22nd of July.
We’re coming to you live at 8:15 pm Tanzania time. From 12,900 feet here at Barranco Camp. At the due Southwest corner of the mountain. Beneath, the mighty, beautiful Breach Wall.
We are tired.
But we’re happy. We had a wonderful day. From Shira, we went up to Lava Tower, our high point at just over 15,000 feet. Some of the team opted to do the scramble to the top of Lava Tower, where we got some additional fun photos and views.
Then we made our way down into the beautiful Barranco Valley.
We are located at THE most beautiful camp on the mountain.
It was a long day. 9-10 hours for us. High altitude. We are tired. We are looking forward to some horizontal time, having been fed well and treated lovely by our amazing Tanzanian crew.
[Cheers]
Katrine Bosley: Thanks, Eric. This is Katrine Bosley.
I want to try to share with you a little bit of what it’s like being up here.
Katrine Bosley, biotech entrepreneur
You think about a climb like this, and you tend to first think of what you see.
The amazing views.
The red flame of the gladiolus flowers by the trail.
The pink and the gold rays of the sunset as they touch the peak of Kilimanjaro, just before it dips below the horizon.
But you sense this mountain with all five senses.
There are smells of the wild mint. It looks like thyme. The leaves are really full. But the smell is a little minty, a little tangy. It’s different.
Today we were hiking in much dustier conditions. Taste for me – we’ve had wonderful food, but on the trail, I was tasting the dust all day long [laughs].
Then there’s what you hear. You hear the cawing of the ravens. They’re swirling above us. On Lava Tower they were swirling below us, which was amazing.
You also hear the gentle reminders from our guides, to go “Pole, Pole.” It means “slowly, slowly.”
And there’s touch.
At lunch today, we were in a beautiful, sunny, rocky place. And the warmth of the sun had made the rocks warm. You could lay down and feel it soothing your aching muscles.
Then there’s touch in the morning when you put on your backpack and it settles onto your shoulders, and you clip the hip straps and feel it settle onto your hips.
Then when you’re climbing and you feel your toes in your boots finding their right grip.
And I’ll tell you right now, there’s a particular feeling I’m looking forward to. It’s lying in the sleeping bag, when you feel your body warming up the space and make a toasty little cocoon. Think of us all in our toasty little cocoons, lined up on the shoulder of Kilimanjaro with the stars of the Southern Hemisphere above us. That arc of the Milky Way is splayed out above us.
That’s where we’re heading in about 32 seconds.
So,
Usiku mwema, Lala salama
That’s Swahili for “Good night, and sleep well.”
Eric Murphy, lead guide: Hi friends, it’s Climb to Fight!
[Cheers]
It’s the 23rd of July. We’re coming to you live from Karanga Camp. On the southern slopes of Kilimanjaro. 13,300 feet above sea level.
It’s about 8:30 pm Tanzania time.
We are well fed and getting ready for bed.
A couple of our teammates, Ken and Spencer, would like to tell you guys about camp life.
Ken Brunt: Thanks, Eric. I want to give you a little overview of camp life, more specifically, tent life.
What happens in tents, stays in tents. Until now.
Ken Brunt, vice president of customer operations, Veracyte
Our rules are: No spilling pee bottles. No farting. And by all means, do not bring in collapsible pee bottles and misplace the lid after filling it. Spencer…
Another tactic we employ in our tent is tent setup and breakdown. It’s really easy when you follow Spencer’s strategy.
Act like you’re struggling until a porter sees you and offers to help [laughs]. Or just patiently wait it out.
You’ll get the help, eventually.
Anything you’d like to add about camp life?
Spencer Guthrie: Yeah, I’d like to add that tent placement is especially important. We like to have our tent close to the bathroom, because the smell puts us to sleep quickly.
Spencer Guthrie, biotech entrepreneur
It’s also important to have a good tentmate, that you let get in there to organize all of his things – and maybe some of your things – before you even get in there. Being lazy pays off a little.
It’s really nice being here with so many smart people. And then me and Ken.
We’re been nominated for potty humor for that very reason.
I’d also like to say we are extremely grateful to the Tanzanian crew. They are amazing.
We came here for the mountain and the charity, which are amazing, but it’s been great to spend time with the Tanzanian people.
Now I want to hand it back to Ken.
Ken Brunt: I want to give a quick overview of what the day entailed.
It was an early start. About an hour earlier than our normal days. We were trying to get ahead of the pack. We had a steep climb out of camp, up the Barranco Wall. It was short, but there was lots of rock scrambling. The scrambling was fun, and the group did awesome. We have a few rock stars around here. It was a lot of fun. A great day. A short day. We got lots of naps in. A little bit of card play for some of us.
I want to close with a shout out to friends and family, the Veracyte family. Your support means the world. We have about 300 employees, and we have about 75 percent participation in support of this climb. It’s pretty awesome.
[Cheers]
Eric Murphy: Thanks to all. Tune in tomorrow as we head up to High Camp, and get ready to head to the top.
Eric Murphy, lead guide: Friends, family, loved ones, sponsors. It’s Climb to Fight!
[Cheers]
We are at high camp on Kilimanjaro. 15,800 feet above sea level. It’s July 24th. Just before 4 pm.
We are all here, and we are all doing well. We are all in our final preparations of getting ready to go up to the summit of Kilimanjaro. Starting at midnight tonight.
Kristin Anderson: In preparation for our summit, we thought it would be a nice opportunity to highlight some parallels between a cancer journey and the journey of summiting a mountain.
Kristin Anderson, postdoctoral fellow, Greenberg Lab, Fred Hutch
My name is Kristin.
My story, very briefly, is that I’m a cancer survivor. I was diagnosed with aggressive, triple-negative breast cancer when I was 28. I went through a really intense chemotherapy and surgery and reconstruction. It took about a year. I know that every cancer journey is incredibly unique.
I also know that because it is unique for every individual, it can be incredibly isolating.
It’s physically, mentally, and emotionally draining. [sniffs]
In my experience, at my low points, from faith, to an unexpected card or email, or a meal prepared by a friend – all these things reminded me that I was not alone.
On this mountain, when I’ve had low points, or struggles, a shared snack, a word of support, or reaching camp to a chorus of guides and porters singing really enthusiastically – it’s been a real morale booster. It’s been impossible to convey just how valuable the small gestures really are.
No. 2. On my cancer journey, there were many long, exhausting stretches that were punctuated by really short breaks. Really short reprieves.
Kristin Anderson on the summit of Kilimanjaro, July 25, 2019.
A visit from a friend.
A day where I finally felt like myself again.
Or good news from a doctor. They were all short breaks that made a huge difference in my mental health on that journey.
On the mountain, we push hard. Consistently, and slowly. Each push is punctuated by a short break. A delicious snack. Some information about a 100-year-old plant that only flowers on this mountain, or in this part of the world.
Or maybe you see a beautiful view that you only get from the ceiling of Africa.
These are the brief breaks we get on this trip that help keep us going.
Finally, parallel number 3 is the fear of the unknown. Every cancer journey, because it is unique, is also unpredictable. Our journey up the mountain is similar. It’s unpredictable. We have incredible guides who are experienced. They can give us lots of information about what may happen.
Just like how a doctor can set expectations for a patient. But nothing is ever certain.
It’s moments like this that I feel the impact of our mission. Raising money to research cures to provide hope, and lessen the fear of the unknown, for all the patients out there.
So thank you, friends, family, loved ones, and cancer haters everywhere for all of your support.
We’ll see you on the other side.
Eric Murphy, lead guide: Friends, family, loved ones, sponsors, the world.
It’s your favorite team here on Kilimanjaro. Climb to Fight!
[Cheers]
Bob More: Hello folks, this is Bob More of Alta Partners. Reporting from Millennium Camp at 12,200 feet, with just incredible news.
We went 27 out of 27 on people summiting today on Mt. Kilimanjaro.
[Cheers]
It’s probably honestly something that none of us really expected.
It’s a tribute to everybody on the team. It’s a tribute to Luke. And it’s a tribute especially to Alpine Ascents International for helping us get to the top of the mountain.
We had an incredible day. We had perfect weather.
We woke up at 11 pm, which is a bit early for a wake-up call. We began the ascent and arrived at around sunrise. So it was timed perfectly.
From the top of Kilimanjaro, you could see the curvature of the Earth.
You could see the shadow cast from Kilimanjaro over Mt. Meru.
It was just an incredible scene. I’m so proud of everybody, and so proud to be part of this. Again, this is for the Fred Hutchinson Cancer Research Center. We’re all part of a team. One team, one dream. We’re all really excited to have made it to the top today.
I often tell my children that life is about three things.
Alec More (left) and Bob More.
It’s about relationships, experiences, and being part of something bigger than yourself. This trip has all three of those.
Now I’ll turn it over to my son, Alec.
Alec More: I’m the son and unfortunate tentmate of Bob More.
It was a great day. We went 27 for 27. We owe that to our wonderful team leaders.
As far as the trip, it’s kind of coming to a close.
We head back to Arusha tomorrow. Reflecting back, the team was amazing. Amazingly positive, amazingly supportive. Whenever anyone needed something, the rest of the team rallied around.
Getting to the summit a little after 6 am today, and seeing the sunrise, proved that Eric knew what he was doing. He got us there right around the right time. It’s been an amazing week. I’m so thankful to be a part of it. I’m thankful to Luke for bringing us all out here.
Bob More: Making hard things look easy is very difficult. It’s a credit to everybody here. Everyone on the team pulled their weight and more. We’re very excited for a successful climb.
I hope this is one of many in the future.
Thanks everybody for listening.
Eric Murphy: We’ll try to give you guys one more shout from Arusha after we’re showered and cleaned and fed. We’ll also be getting some pictures out on the Cybercast. Stay tuned.
Eric Murphy, lead guide: Hey everyone, it’s Climb to Fight!
[Cheers]
We’re giving one last shout to our friends, and our families, and donors, from Arusha, Tanzania.
We are here celebrating in the city, after making our way off Kilimanjaro back to the hotel for showers and here for our celebration dinner.
[Cheers]
Nancy Miller-Rich: Hello everyone, my name is Nancy Miller-Rich. I wanted to give you some facts about what it was like to summit.
We had a great day. All 27 of us summited.
Nancy Miller-Rich, chief executive, Miller-Rich Associates
But I’ll say, there was some of us who had some – I’d just say — up-and-down experiences. I was one of those people. My husband made me promise not to make any headlines.
I think I was pretty close to making some headlines. [laughs]
It was really an amazing day, and an amazing family of people we have met here.
My issues came when we started hitting 15,000 feet, and I realized I was really having altitude sickness. After we hit 15,000 feet, we went down, and slept, and then as we got up at 11 pm the next evening, and started our trek up Mt. Kilimanjaro, at about 3 am, many of us on the 4th or 5th push started getting sick.
But everyone else picked themselves up. I didn’t seem to have the ability to do that.
What was amazing to me, was a young Tanzanian fellow came by and asked, “Do you want to go up, or go down? Let me carry your bag.” This young person just took me by the side and was my guardian.
He took me up the trail. It was amazing. When we reached the top, about 6:30 or 7 am, I was looking over the views of Kilimanjaro – which are absolutely breathtaking – I also looked at this young man who helped us, and helped me, and Alpine Ascents. I knew I could never have done it without him. He said ‘Congratulations, you summited.’ I said ‘No, no, we summited.’ That’s the way it felt. We were a team and we were in it together.
Nancy Miller-Rich (left) on Kilimanjaro.
Interestingly enough, it wasn’t just a difficult summit, but as you went down, it was difficult going down. But everyone was there for each other.
It was an amazing experience.
My closing insight is that not only is Kilimanjaro amazing, but the people of Tanzania are incredible in their caring, their compassion, and their strength.
I know, I, for one, met some incredible people in this industry that I didn’t know before.
I am so thankful to Alpine Ascents. I’m thankful to you, Luke, for setting this up. Our guides were unbelievable.
Eric Murphy: Thanks Nancy. From the bottom of our hearts, we thank you all for following along. That’s all.
Sandy Zweifach, president and CEO, Nuvelution Pharma
By Sandy Zweifach
Would you climb Kili one day
Not at all is what I did say
Not with Peter, Simba or Uciane
I would not climb Kili on any given day
What about with Zoe and her Instashots?
Or Praveen with his boom box on the way to the top?
No. And not with Nancy Rich or Leslie or Bill
Even though no matter how sick — these ladies will crush the hill
With about with Bob and his smallest pack?
And his son Alec on his back?
Or Dan the man (who I hear did throw up)
Not with Spencer, who will never grow up
How about with Katrine and Julia — two women CEOs?
Katrine shakes her booty like no one would know
Not with Adam, Ken or Oren each six feet long
Nor with little engine, Nancy Hong
Not with Kirsten, or Kristen — come that’s too much!
Or even with Kelly, leader from the Hutch
Please stop. Don’t suggest I would go with Heather
I know at the top is freezing weather
Senthil or Heidi, to them I would say
Let’s go to St Bart’s and have a warm day!
I will not climb Kili is what I did say
But I didn’t know Luke would lead us this way
To Eric and Lakpa whose wisdom and skills would blow me away
So finally I did say Yes to the climb
With my amazing wife Nina whom I was dragging behind
I sure am glad I made it this way
And together we will fight cancer every day
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Today’s guest on The Long Run is Janelle Anderson.
Janelle is the chief strategy officer of Century Therapeutics. This is a company using induced pluripotent stem cells to make off-the-shelf cell therapies for cancer. This is the new dream of cell therapy, as scientists imagine ways to lower the cost and expand access for patients following the first wave of technical success with personalized cell therapies. Last month, the company announced it got started with a $250 million financing commitment from Bayer, Versant Ventures, and Fujifilm.
Janelle Anderson, chief strategy officer, Century Therapeutics
Janelle came to this company through her role at Versant, where her job was to scope out some of the latest science and think about building companies around it. She was born and raised in Canada, got her PhD in chemistry at Harvard University, and surprisingly found herself gravitating to venture capital and startups while at Merck. Somewhere along the way, she combined her interest in science and business with a cool extracurricular activity – a podcast called Human Proof of Concept. That ran for a couple years before she came to Versant Ventures and got immersed in the creation and building of Century Therapeutics.
See Human Proof of Concept archives here.
Now, please join me and Janelle Anderson on The Long Run.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Please subscribe and tell your friends why it’s worthwhile. Quality journalism costs money. When you subscribe to Timmerman Report at $169 per year, you reward quality independent biotech reporting, and encourage more.
Leora Schiff, principal, Altius Strategy Consulting
There is growing discontent among many healthcare stakeholders with the Institute for Clinical and Evidence Review known as ICER, something which may not surprise many TR readers.
Recently, I had the opportunity to moderate a panel on value-based agreements for the CBI/EBD Group Rare Disease Innovation & Partnering Summit in Boston. Among the topics that the panel discussed was the increasing role of health technology assessments (HTAs) in US reimbursement.
Much of the discussion focused on the growing impact of ICER. It was clear that ICER had no fans in this audience of industry professionals and patient advocates. Several patient advocates, in particular, were extremely critical of ICER’s recent report on drugs for Duchenne Muscular Dystrophy (DMD). Their main complaint was that the model was based on inaccurate data regarding the DMD population. They also thought that ICER does not listen to patients, failing to adequately act on patient comments and feedback. A health economist in the audience questioned whether or not any of the ICER reports could pass muster for publication in a peer-reviewed journal. No one came to ICER’s defense.
The biotech community has been critical of ICER for years. Last year, following ICER’s publication of its report on cystic fibrosis drugs, Vertex sent a scathing letter to Steve Pearson, ICER’s president, accusing ICER of using “arbitrary modeling choices” and that its process of evaluation was a “sham” since ICER “presented the final cost-effectiveness recommendations . . . three weeks before the public hearing at which patients, physicians and Vertex were scheduled to present their evidence.”
BIO (the Biotechnology Innovation Organization) has publicly disagreed with ICER’s approach to the Value Assessment Framework that it uses in making its health technology assessments.
Sarepta Therapeutics, the maker of a treatment for Duchenne Muscular Dystrophy, issued a statement in May that blasted ICER’s assessment of DMD therapeutics. Sarepta called ICER’s approach “fatally flawed as it relates to rare and genetic disease”. Sarepta followed up the harsh language with action, saying it has “chosen not to participate in reviews by ICER until it adapts its model to address the inherent limitations and biases that compromise its evaluations of therapies intended to treat patients with serious, rare diseases.”
A few years ago, I wrote an article for TR called “Watching the Watchdog: How Does ICER Determine ‘Fair’ Prices for Drugs?”, in which I was quite critical of ICER. My main criticisms at the time were ICER’s lack of transparency regarding its models and inputs which made it impossible for other organizations to reproduce its results. I also thought there were questionable assumptions used in its models.
More recently, health economists Peter Neumann and Joshua Cohen of the Center for the Evaluation of Value and Risk in Health at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, have also taken ICER to task for its lack of transparency. They also raise concerns about ICER as a private entity making decisions about which thresholds should be used in cost-effectiveness and public and private budget determinations, rather than having the people who are actually responsible for healthcare plans make such decisions.
Why we should care about ICER
Like it or not, ICER has become the go-to source for HTAs for commercial payers.
The Academy of Managed Care Pharmacy (AMCP) conducted a webinar in February on “ICER: Payer perspectives on the use and usage of ICER reports”. During the webinar, the speakers cited survey data on a sample of 614 US payers, integrated healthcare delivery systems and hospitals. 87% of the respondents indicated that they use some form of value framework during their pharmacy and therapeutics (P&T) committee formulary decisions. 76% of the respondents that use value frameworks (predominantly managed care organizations and PBMs) indicated they use ICER reports. These organizations use ICER reports predominantly as a secondary source of evidence, in determining product affordability and in determining tier placement for drugs. Some respondents also use ICER reports to support pricing negotiations.
In addition to commercial plans, ICER reports are also being used by government agencies. Since 2017, ICER has been working with the Veterans Administration to use its HTAs in determining drug coverage and price negotiations.
Why are these organizations turning to ICER for their HTAs?
Historically, payers have used a variety of HTA resources, including reports from the Agency for Healthcare Research and Quality (AHRQ) and Hayes, Inc. The Blue Cross and Blue Shield Association conducts its own HTAs through its Evidence StreetTM platform. However, these reports provide comparative clinical effectiveness analyses of new treatments. ICER appears to be filling a vacuum, addressing a growing need/desire for cost-effectiveness analyses.
From my own conversations with payers, I have learned that many payers, particularly the smaller regional ones, lack staff trained in health economics and the resources to conduct appropriate health technology assessments. Having a source of free HTA reports would certainly be attractive for them.
Is ICER “NICE”?
There have been many comparisons of ICER to England’s National Institute for Health and Care Excellence (NICE). Neumann and Cohen point out several similarities between the two organizations (as well as a number of arguments about important differences). The similarities include:
However, ICER is not NICE.
NICE is a special health authority that reports to England’s National Health System (NHS). NICE’s operations are explicitly defined in a formal legal framework that establishes how it will work with England’s Department of Health & Social Care “to serve patients, the public and the taxpayer.”
This contrasts with ICER’s private funding through large grants from the Laura and John Arnold Foundation totaling over $19 million, as well as contributions from health plans, providers and drug manufacturers. While ICER solicits public feedback, and its reports clearly have influence over public and private health spending decisions, there is no formal mechanism for it to be held accountable by public or private organizations, patients and the public.
There are also critical differences in terms of how NICE and ICER function. Let’s start with NICE. In order to make a cost-effectiveness determination for a medical product, NICE will commission an Evidence Review Group (ERG), usually an academic group out of a university such as York or Liverpool, to conduct an evidence review. The ERG submits a report to NICE’s Appraisal Committee (AC); the product’s manufacturer submits its own dossier to the Appraisal Committee. Both reports provide assessments of the clinical evidence, cost-effectiveness and NHS budget impact models. The Appraisal Committee then holds public hearings. Based on the ERG and manufacturer’s submission and the public feedback, the Appraisal Committee then makes a determination of whether or not the NHS should invest in covering the new product as an NHS benefit, which is issued as formal NICE guidance for the NHS. Topics for evaluation are determined either by NICE or by referral from the Department of Health.
Now let’s look at the ICER process. ICER chooses its topics based on its own scan of drugs in the pipeline, suggestions from advisory boards (for non-drug technologies) and public suggestions. ICER will develop a scope for its evidence report, soliciting public comments during the scoping process. ICER also will speak to manufacturers to discuss appropriate comparisons and the current state of the published evidence. ICER will then draft a report which it makes available for public comment. In developing the report, ICER is interested in inputs from manufacturers for ICER’s models which it develops internally or with external collaborators. ICER often requests manufacturers’ unpublished “data on file”, which they are not required to provide. There is a risk here to manufacturers. If the data is provided, ICER decides whether or not to use it and how. ICER is NOT interested in looking at the manufacturer’s own economic models. Following the public comment period, ICER revises its report, which it posts online, followed by a public meeting to solicit feedback. ICER then publishes the final version of the evidence report on its website and publishes a press release summarizing the results.
In effect, ICER is acting like an Evidence Review Group, producing a report that commercial payer organizations will then compare with analyses by product manufacturers. There is no independent arbiter reviewing the two submissions, no transparency and no public accountability of final decisions.
It is important to note that payers are not commissioning ICER for technology assessments; ICER is setting its own agenda. It has also established its own criteria for determining cost-effectiveness and methodology for making assessments.
Does the US need its own NICE?
If ICER is becoming the de facto source of HTAs in the US but healthcare stakeholders such as patients, manufacturers and health economists question the validity of these reports and the accountability of the ICER organization, is it time to consider an alternative – should the US establish its own version of NICE?
The following would argue in favor of the establishment of a US NICE:
Assuming that there is agreement that a US NICE should be established, the question is how to do that in order to ensure objectivity and accountability to the American public. Should this be an organization that, like NICE, is accountable to a government agency such as the Department of Health and Human Services?
PCORI as NICE?
A potential candidate for such an organization is the Patient-Centered Outcomes Research Institute (PCORI).
PCORI was established through the Patient Protection and Affordable Care Act (PPACA) otherwise known as Obamacare. The purpose of PCORI is to support comparative clinical effectiveness research which it does through grant-funding. [Disclosure: I have served as merit reviewer in PCORI’s granting process. I’m a fan – call me biased.]
PPACA also set up the Patient-Centered Outcomes Research Trust Fund (PCOR Trust Fund). The trust fund receives monies from the US Treasury, the Centers for Medicare and Medicaid trust funds, and PCOR fees assessed on commercial health insurance plans.
PCORI’s research agenda is set by a Board of Governors which includes representatives of all healthcare stakeholder groups: patients, physicians and providers, health insurers, pharmaceutical, device and diagnostic manufacturers and the federal and state government.
So far, so good. However, there are major hurdles in converting PCORI into a US NICE. These hurdles are imbedded in PPACA itself, which prohibits PCORI from using incremental cost-effectiveness ratios (ICERs) “as a threshold to establish what type of health care is cost effective or recommended” and prohibits the federal government from using QALY-based cost-effectiveness analyses “to determine coverage, reimbursement, or incentive programs” under Medicare. As a result, PCORI policy states that “Our founding legislation prohibits us from doing cost-effectiveness analysis.”
If the US government were ever to establish PCORI as its version of NICE, the Affordable Care Act would have to be changed legislatively in order to permit PCORI to conduct cost-effectiveness analysis. Let me rephrase that – Obamacare would have to be revised. Think about that. Given the current political climate, it’s safe to say that this is highly unlikely. However, what the situation will be after the 2020 Presidential election is anyone’s guess.
How I learned to stop worrying and love cost-effectiveness*
Right now, the healthcare industry is in a period of crisis and flux. The system is clearly not working and many patients are being hurt either by a lack of access to care and/or crushing financial burdens. Feelings about drug prices are intensely negative toward manufacturers. Political discussions on what to do are often heated and not always rational.
Unfortunately, there are no easy solutions – ours is a cobbled-together healthcare system based on decades-old practices and reimbursement processes trying to cope with 21st century technologies (and their associated costs).
The movement from volume to value was an important first step in trying to rationalize the system, but it is only part of a bigger process of moving towards optimized healthcare. We need to get to the point where patients are treated with the best option for them – whether it is a drug or a procedure or a device or maybe nothing at all. Ineffective treatments need to get weeded out of the system. Incentives built into the reimbursement process need to be aligned with choices that make sense for patients, rather than what drives profit for healthcare stakeholders – whether they be payers, providers or product manufacturers.
Going forward, cost-effectiveness analysis is not only going to play a role in determining where health plans allocate limited resources – it could be an essential tool to help us balance the system. We need to understand what works well for a given indication on a population basis and how much it costs the system compared to other, lesser clinical choices.
However, current cost-effectiveness approaches using QALYs fail to truly measure the full extent of benefit that new treatments can bring to healthcare stakeholders. We need cost-effectiveness methodologies that are able to capture a broader range of benefits delivered to the full range of healthcare stakeholders, such as productivity gains and a more expansive definition of quality of life improvements.
Members of the International Society for Pharmacoeconomic and Outcomes Research (ISPOR) have been working on developing such new methodologies that go beyond the QALY. Lou Garrison, Sachin Kamal-Bahl and Adrian Towse have proposed an expanded value framework for cost-effectiveness with a “broader range of elements of value”. Such elements could include the real option value for patients being able to survive long enough to wait for future treatments that can extend their lives or even provide a cure and the potential scientific spill-over effects of a new treatment that provides insights that advance the development of future therapies.
Also, at the patient level, the healthcare system needs to be able to flex so that patients can access what works best for them without being bankrupted in the process. We’re far from that point.
I know that the idea of cost-effectiveness in reimbursement is a scary prospect for many in the biopharma industry, particularly given the many examples of ICER calling out drug prices as being 70%, 80% or 90% too high for the drugs to be cost-effective. For some, wide-spread adoption of cost-effectiveness analyses in reimbursement is seen as a death-knell for the biopharma industry, crushing drug prices, chasing away investors and killing innovation.
However, for some therapies, such analyses have been powerful arguments in favor of their reimbursement and adoption, even given the currently limited QALY methodology. As an example, last year ICER published a report on Genentech’s emicizumab (Hemlibra), a bispecific antibody for the treatment of hemophilia A. ICER found the drug not only improves patient outcomes, it also significantly reduces the need for costly blood and plasma transfusions, making it cost-saving. To quote ICER’s CMO, David Rind, “Emicizumab appears to be a very rare win-win-win in treating a small population of patients who have hemophilia and cannot be treated with factor VIII.”
For drugs to be fairly valued in cost-effectiveness analyses, we need both better methodologies to capture the full spectrum of benefits that innovative therapies provide and a fair, objective and independent arbiter to perform the assessments.
The biopharma industry can continue trying to put a finger in the dike, objecting to the use of cost-effectiveness to determine reimbursement policy. But the water is already leaking pretty fast, with a flood looming on the near horizon.
Alternatively, the biopharma industry can roll up its sleeves and get involved in trying to find a better solution. This means engaging in discussions of how to improve cost-effectiveness methodologies to better reflect a drug’s overall value and how best to develop robust models that are convincing for all stakeholders.
This also might mean calling up your congressman and talking about the virtues of cost-effectiveness.
Leora Schiff is the Principal of Altius Strategy Consulting. She can be contacted at lschiff@altiusstrategy.com.
*For our millennial readers, if you didn’t catch this film allusion, I highly recommend the classic “Dr. Strangelove or: How I Learned to Stop Worrying and Love the Bomb”.
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