Help bring the patient voice to FDA!


#1

On August 29th, FDA is hosting a day long workshop with the diabetes community entitled Diabetes Outcome Measures Beyond Hemoglobin A1c (HbA1c). This is an important meeting and conversation to have with FDA, because as patients we know that despite the many options we have for managing our diabetes, looking at our A1C number doesn’t give us or our healthcare provider the full picture of how we are feeling and how we are doing. Yet this is the measure by which the FDA currently evaluates new drugs and devices. I have signed up for the event and will be attending in person.

You have the opportunity to help FDA understand what is important to patients when they think about new therapies. The diaTribe Foundation is creating a video to show during the meeting, and they want to include as many people with diabetes as possible – type 1 and type 2.

Here is what you need to do – it is so easy!

  • Take a video on your phone (under 10 seconds) and answer these two questions:

  • What I struggle with most in managing my diabetes is________.

  • Beyond helping me with A1C, I wish a new diabetes drug would_______.

  • Send it to videos@hcmstrategists.com

  • You can share it on social media with #DOCasksFDA

You will also be able to watch the event live – all you have to do is register through this link.

Many thanks to Ronnie Tepp of HCM Strategists and Kelly Close and her team at Diatribe.


Going Beyond A1c - A Deeper Dive by diaTribe
#2

Thanks Brian–I’m glad we’ll have such an articulate and exceedingly well-informed representative at this meeting.

I have to say, when I read “Diabetes Outcome Measures Beyond Hemoglobin A1c (HbA1c)” my first thought was “Yes! Finally!” followed rapidly by, “Oh no, sorry, wait a sec. I bet they don’t mean the same thing by ‘outcome’ as I do.”

A while back, my endo was saying there was some debate in the treatment community about the value of basal-bolus MDI in light of studies showing that the “outcomes” were not significantly better than those for patients on R/NPH. I didn’t quite fly into a rage, but I did point out with some vehemence that for me, having been on R/NPH for 20 years before switching to MDI, the “outcome” was immediate and life altering. It was like being let out of PRISON.

A lot of people feel that way about pump technology. A lot of people feel that way about CGM technology. This disease has a lot of prison cells and it’s not only the people who have it that are in ‘em. I think of the especially nasty one parents of T1 kids are in, and how much it helps to have a CGM with share capability so they can actually get a decent sleep at night, and not be on tenterhooks at work worrying about how their kid is managing throughout the day at school. Not to mention caregivers of older people with the disease–a close friend at work can’t get her elderly dad on a pump, even though he uses insulin, because he has T2 and pumps aren’t covered. She’s worried sick about his increasing dementia and his own or his caregivers’ ability to manage his disease safely, which a pump would help with enormously. One of the “outcomes” a lot of us are now looking at as we approach retirement age is the LOSS of options that have helped us live more “normal” lives. I’ve had been diagnosed T1 almost my entire adult life, but I’m told that in order to “qualify” for a pump on Medicare I’ll have to submit to peptide and antibody tests I’ve never had, and the results of which I’m told are uncertain so long after diagnosis. Not to mention the great number of us approaching retirement with trepidation because we have come to rely on CGMs for everything from hypo unawareness to controlling post-prandial spikes and exercise-induced hypos and just the general comfort of being able to track what the hell’s going on with our sugars throughout the day and night–and as things stand we’re all going to lose access to that technology and the freedom from uncertainty it provides because Medicare doesn’t cover it and the cost for individuals is exorbitant.

These are “outcomes” that matter to us as people living with this disease every minute, not just the question of whether we’ve managed to avoid blindness or having a limb amputated.


#3

Thanks @DrBB. I’ll be posting in the next week or so to get everyone’s ideas about this. There will be people who will talk about “time in range” but I also believe that there are many factors which affect quality of life that also matter. If a treatment negatively affects our quality of life it makes a difference in how much we adhere to the treatment and the eventual “hard outcomes” which are how long we live and the the complications we have to deal with.


#4

Thank you so much for posting this, Brian! And thanks for planning to go to the workshop and speak! I’m sure you will be an articulate and respected representative for us!

Glad to know that the FDA is at least asking the question, also!

And thank you DrBB for your post, also. There are a lot of issues to consider.

Best wishes to all,

marty1492


#5

There are a ton of heavy hitters from industry, pharma, healthcare, academia signed up to attend.

It is IMPERATIVE that those of us who actually live with diabetes have a voice in the conversation. The measures that come out of this (and other) events are important. They will be used to decide patient success, as well as the treatments (medicine and devices) that we will be using to live longer, healthier and happier lives.

I invite you to look over information about the workshop. It isn’t often that the folks who make the rules - the Food and Drug Administration - take the time to listen and learn from the people who are impacted the most.

Take time to share your thoughts with the FDA. Think outside the box. Do you want your diabetes ‘performance’ to be graded against some arbitrary laboratory value, or against how well you are meeting a goal established by you and your doctor? Should quality of life be included in measuing how well you are dealing with diabetes? How about factoring what it does to your mental health, social life, etc? Those are all part of living with diabetes - do you think they should be part of measuring success, and if so, how?

I’d also invite you to share information about this event with your healthcare team, other people with diabetes and anyone else who is impacted by this godawful disease.

-Tom


#6

@Tom_Goffe, you are right, this is an important meeting. More than 500 people have now signed up. I have no idea whether I will have an opportunity to make a statement, but I will be prepared. This workshop is pivotal as the A1c has been the entire focus of diabetes outcomes. There are “hard” outcomes such as mortality or complication rates, but drugs are not evaluated against these measures in terms of effectiveness (except the FDA does focus on negative CVD/mortality with drugs). Instead the FDA depends on the “surrogate” measure of HbA1c. But life is not summarized by the HbA1c. There will be others who argue that other measures such as “time-in-range” matter. But I think from a patient perspective the “quality-of-life” measures have been woefully neglected. And if drugs cause terrible side effects and patients can’t keep taking them it is silly to suggest they are “effective.” These are the types or point I hope we can bring up.


#7

I’ve been thinking about this issue since you posted this a few days back. I’m happy to see some formal recognition that the A1c number contains some serious flaws. First is the nature of any average number, if you don’t see the range of the data then you have no idea as to blood glucose variability.

I have four outcomes that I use my CGM to monitor that help me gauge how well my regimen works. Note that A1c is not one of them! In ranked order of importance to me and my quality of life:

  1. Time in range, 65 mg/dl - 140 mg/dl, >= 80%.

  2. Time hypo, under 65 mg/dl, <= 5%.

  3. Blood glucose variability as measured by standard deviation <= 30 mg/dl.

  4. Blood glucose average < = 105 mg/dl.

Now I know that the medical and regulatory community may not agree with my use of the term “outcome” in this context. I have long experience, over 280,000 hours living with type I diabetes, and I know that meeting these goals produces more motivation than I exert, makes me feel physically better, and convinces me with each successful day that I have the power to deal effectively with diabetes. This is a potent reinforcement to my emotional well-being. Perhaps a question and answer survey could measure this sense of well-being. It is obvious to me when I have it and when I don’t.

Do I understand the nature of the FDA patient video request? If it only seeks 10 seconds of my thoughts then I’m not sure about the quality of the feedback.

It seems that Pharma is more concerned with economic success than they are with patient outcomes. Why do most drug studies screen out, during phase zero, patients that may show susceptibility to adverse side effects? Adverse side effects of any proposed drug need to show their full quantity to regulators, clinicians, and patients, not some “doctored number.”

In summary, I propose outcomes be measured regarding quality of life and the emotional impact of any tactic or drug used.


#8

I also went through the list of attendees. One glaring absence are patients. I know that the time and expense is an insurmountable hurdle for most of us. I’m going to make the trek, but before doing so I’m reaching out to see if there is a way to either get on the program (ideally, but probably a farfetched hope) or to provide comments directly to the ‘right people,’ i.e.: decision makers.

Before the event, some substantial interest from the patient community is essential. There might be some who have brilliant suggestions that are ideally suited for this particular forum. Those voices need to be heard. Just like questions, the only dumb ideas are those that don’t get a hearing.


#9

Here’s the video I am submitting:


#10

Could anyone who was able to attend fill us in on how it went?


#11

diaTribe posted a good summary last week. I also watched about 3/4 of it remotely on my computer. Time in range was strongly supported by diaTribe’s Kelly Close and a few others who spoke at the meeting.


#12

I attended but haven’t had the time to write anything up on the meeting. There are video recordings that have been put up on the FDA site and there will be a transcript sometime at the end of September. Below is a picture from the meeting of me waiting to ask a question behind my friend @StephenS (of Happy-Medium) (kudos to @BadShoe (Bennett Dunlap) for the picture).

https://scontent-iad3-1.xx.fbcdn.net/v/t1.0-9/14089178_10209684369403514_9118068516536956832_n.jpg?oh=c1e0aa18875e98113055e1955ea8fc6e&oe=5841CC25

Here are some of my initial notes from the meeting:

I was very pleased to see the FDA embrace inputs, particularly calling out patient participation. There were limited windows for input from the workshop participants as a large proportion of the time was taken up with scheduled speakers. Fortunately key patient advocates including Kelly Close, Anna McCollister-Slipp and Christel Aprigliano got on the agenda and did a wonderful job of representing.

Overall, there seemed to be broad support for using time-in-range as an additional outcome. There was a lot of talk about Patient Reported Outcomes (PRO) which includes quality of life, but very little on how that should factor into FDA decisions or lead to changes in labelling. Other outcomes such as fasting blood sugars or insulin sparing (medications that reduce insulin requirements) got little attention.

I had hoped for more attention to outcomes that could improve the precision in which medications are prescribed. There seemed to be little interest in that. By my count there are nearly 60 type 2 medications and knowing which ones will work on which patients would seem to be kind of important. I know in my case, I was basically a Nonresponder to type 2 medications. I had a chronic elevated fasting blood sugar and the best I can tell only the SGLT-2 drugs work to reduce a fasting blood sugar like mine. Patients can end up rotating through useless medications with serious side effects and elevated blood sugars without this knowledge. Too bad these outcomes aren’t a priority and some panel members felt there isn’t sufficient understanding in order to include the medication effects (like reduced fasting blood sugars) as an outcome.

The big takeaway for me seems to be that CGMs are now considered standard in studies and pretty much the entire group felt that they would be a routine part of study protocols moving forward… This means that time-in-range will be broadly available. The other takeaway was that the FDA is listening to patients and sees social media as an important part of their direction moving forward.


#13

Hopefully this also moves things in the direction of getting them covered under Medicare.


#14

I really like this quote from Kelly Close:

Close mentioned the enormous time that many patients spend either in hyperglycemia (high blood sugar) or hypoglycemia (low blood sugar) or recovering from it. As she put it: “A1c is one thing; the quality of A1c is another.” (Emphasis mine)


#15

My read was that CGM should be part of studies to track time in range, hypos, hypers etc.

Happy to see the photo used.

I was lucky enough to use one of the kids’ CGM’s for a week and found that the timing of when I took metformin made a huge difference in my day. I didn’t really expect that. It was a one week deal, I would love to put on Libre and see if that experience holds true.

I think it would also help identify early when it is time to find new T2 meds to stay successful. I like your definition of precision being success. I too would like some clinical information on the likelihood of which next steps will maintain success with T2 meds. I am unsure of how that path will be defined other the formulary choices.


#16

@badshoe, I’d be interested in hearing your discoveries about timing the Metformin. Of course, YDMV :sunglasses:


#17

OK, nobody should take any of this as any advice other than play with your reactions and find how Your Diabetes May Vary.

The short version: I was taking 1500 mg 1000 at the evening meal, my biggest of the day, 500 in the am. had ok fairly consistent fasting BG when I checked. So all good right?

The CGM showed that I was going way up 10am-noon (even with little or no food in the am), was going low 8-10, in the evening. I didn’t know I could go low on metformin, but I was. It caused serious munchies. That doesn’t help anything. Did 5 days of pattern tracking then changed.

So I swapped to 1000 in the AM and treated the evening 500 as a bolus as a function of carbs eaten for dinner. flattened out the curve.


#18

Was this metformin or metformin ER?


#19

regular


#20

DiabetesMine published good coverage today of the FDA “beyond A1c outcomes” meeting on August 29.