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Q: Average clotting factor consumption in the U.S. ( Answered 4 out of 5 stars,   0 Comments )
Subject: Average clotting factor consumption in the U.S.
Category: Health > Conditions and Diseases
Asked by: shmidt-ga
List Price: $125.00
Posted: 08 Dec 2004 14:31 PST
Expires: 07 Jan 2005 14:31 PST
Question ID: 439997
I work in marketing and need some "normative" data to compare internal
research results to... Specifically ,I need to know the average amount
of clotting factor medication used per year by a person with
hemophilia in the United States.

This could be reported according to the type of hemophilia (hemophilia
A and B collectively or both separately), disease severity (severe,
moderate, mild), age group (pediatric and adult patients collectively
or separately), or any other grouping, so long as the answer can be

Note, I need to know the average number of units (IUs) of clotting
factor used per year, not the average cost per year. Data I've found
over the years show the average cost to be anywere between $40,000 per
year, per patient to $130,000 per year, per patient, depending on
disease severity, age, inhibitor status, etc. One unit of clotting
factor costs ~$1.00, so I would expect the final answer to be
somewhere in the range of 40K to 130K units per year.

Clarification of Question by shmidt-ga on 21 Dec 2004 12:20 PST
Some useful websites...

Request for Question Clarification by pafalafa-ga on 21 Dec 2004 13:24 PST
Hello shmidt-ga,

I've found some very authoritative CDC data on doses that puts the
average dose for a person with hemophilia in the US at a bit less that
80,000 units per year, so your estimate certainly seems to have been
right on the mark.

There are also a fair number of medical reports that provide dose
information for individual users -- I've found doses ranging from
6,000 units per year to (believe it or not) 500,00 unit per year.

There's information, as well, about the total number of units sold per
year in the US, and the numbers of companies that manufacture them.

There's also some good demographic information on the number of
hemophiliacs in the US, and breakouts between A and B, and between
mild, moderate and severe cases (But not both!  That is, I can tell
you the total number of people with severe cases of hemophilia, but I
can't tell you the number with hemophilia A that are severe).

The reason I haven't posted an answer yet is because of your desire
for information that "can be extrapolated".  I don't know what sort of
extrapolation you have in mind, but there are so many variables
associated with the doses provided to people with hemophilia, that I
would be hesitant, myself, to attempt to make any sort of

However, I'd be more than happy to post the straightforward
information I've found as an answer to your question, if you think
that would meet your needs.

Let me know.


Clarification of Question by shmidt-ga on 22 Dec 2004 07:23 PST
Thanks Pafalafa. To clarify, I would accept anything from an
authoritative source that either...

A) ...states the average number of units used annually by a patient
with hemophilia. This number would bundle all patients together --
mild, moderate, severe and types A and B. (It sounds like you might
have this with your CDC data.)

B) ...breaks out the average number of units used by patients in
certain groupings (which I could then extrapolate to find the overall

A couple examples of what these groupings in Option B might be:
     i. Grouped by Hemophilia type A and B (e.g. "the average
hemophilia A patient uses 90,000 units per year, while the average
hemophilia B patients uses 60,000 per year)
     ii. Grouped by hemophilia severity (e.g. "the average factor
utilization of a severe patient is 120,000 units per year, while the
average utilization of a moderate and mild patient is 40,000 units and
2,000 units respectively")

Based on known demographic mix, I would then be able to "extrapolate"
the final combined average for the entire hemophilia population
because we know that approximately 80% of hemophilia patients are type
A and 20% type B. Or, likewise, we also know that overall, among both
types of hemophilia, approximately 60% are severe, 25% moderate, 15%
mild. So, in the case of Option B, I would just need an authoritative
source for the original utilization numbers that I could use to
calculate the final overall average.

Feel free to post another request for clarification if you're unsure
what you have. FYI, due to the holidays, I might not be able to get
back to you until next week.

Request for Question Clarification by pafalafa-ga on 30 Dec 2004 09:06 PST
Hello again shmidt-ga,

Thanks for being patient while I sorted through the information I have
(and also recovered from the holidays!).

As I indicated earlier, I have found a lot of information on typical
doses, and I believe I have the information you need.  However, it
isn't exactly in the form that you stated in your clarification, so I
wanted to check back in with you.

Essentially, I have an authoritative report that analyzed data from
the CDC to come up with average dose information.  However, the report
calculates the average annual use of clotting factor ONLY for
hemophilia A patients, and puts this dosage at 78,000 units per year.

The same report also: 

--confirms the predominance of hemophilia A (79% of all patients), and 

--allows a calculation of average dose for ALL patients (A+B) at
72,150 units per year.

It seems to me that this information would meet your needs, especially
given the predominance of hemophilia A over B in the patient
population.  However, I just wanted to confirm that this was the case
before posting a formal answer to your question.

Let me know what you think.


Clarification of Question by shmidt-ga on 03 Jan 2005 08:44 PST
Thanks. Yes, this sounds like what I'm looking for.

Subject: Re: Average clotting factor consumption in the U.S.
Answered By: pafalafa-ga on 03 Jan 2005 15:07 PST
Rated:4 out of 5 stars
Hello again Shmidt, and a very happy 2005 to you.

I think you'll be pleased with the information I've uncovered, as it
offers some interesting detail on the use of clotting factor by the
population of hemophilia patients in the US.

Although I came across quite a number of sources of information, one
stood out, so let's get right to it.  The US government's General
Accounting Office conducted a comprehensive study of clotting factor
use in an attempt to determine whether the Medicare program was being
overcharged.  The report can be seen at:

January 2003

Payment for Blood Clotting Factor Exceeds Providers? Acquisition Cost

I would recommend a careful reading of this report, as it contains a
great deal of information on clotting factor use, as well as
references to other key sources of information.

Among the most salient pieces of information in the report are these:

--hemophilia A accounts for 79% of the total population of hemophilia
patients, hemophilia B is 21%

--18,000 Americans, nearly all male, have hemophilia

--Total clotting factor use is approximately 1 billion units per year

--the average annual use of clotting factor VIII for a person with
hemophilia A is 78,000 units,

--approximately 23 percent of individuals with  hemophilia use no
clotting factor at all

So, based on the above numbers, we can compute the following:

1 billion units per year / 18,000 patients = 55,556 units per year as
average dose per patient (including patients that use no clotting
factor at all).


1 billion units per year / 13,860 patients = 72,150 units per year as
average dose per patient (accounting for the 23% of patients NOT using
any clotting factor)

Thus, the use by hemophilia A patients is 78,000 units per year, on
average, while that for all patients that use clotting factor is
72,150 units per year.


There are some other items of interest in the same GAO report, including:

--There are 13 unique clotting factor products used to treat the two
most common types of hemophilia. These products vary by manufacturer,
protein composition, and manufacturing process.

--In any given year, approximately 23 percent of individuals with
hemophilia use no clotting factor at all, while a very small
percentage of individuals may use more than 500,000 units.

--An individual?s prescription varies according to weight and whether
the individual is infusing on demand or for prophylactic purposes.
Physicians use their own discretion in calculating the exact quantity
to prescribe in any given situation. According to a physician...a 150
lb. individual with a moderate injury should be prescribed
approximately 1,500 to 2,000 units of factor VIII. The same individual
should be prescribed 3,000 to 3,500 units for a severe injury, such as
a head injury.

--While patients infuse once or twice in response to a bleeding
episode, those under preventive treatment infuse three times per week
to maintain their baseline amount of clotting factor... a total of
5,700 to 6,500 units of factor VIII infused over the course of each
week would be a suitable preventive strategy for a 150 lb. individual.

--the distribution of the severity of hemophilia is classified as: 
Mild 32%,  Moderate 24%,  Severe 41%,  Unknown 3%.


Although the GAO report is your best single source of information,
there is one other report I'd like you to be aware of, as it provides
similar sorts of numbers for the global population:
World Federation of Hemophilia

Among the data presented in the report are these:

--worldwide usage of clotting factor is estimated at 3.7 billion units in 2000

--the global population of hemophiliacs is at least 400,000, though
the actual number may be considerably higher depending on which
factors are used for the estimate.


Beyond these two, I came across quite a number of reports that
provided details on the doses used by individual patients, or small
groups of patients.  Let me know if these are of interest, and I'll be
glad to post information about them as well.

I trust this information provides you the data you were looking for.  

Before rating this answer, however, please let me know if you need
anything else.  Just post a Request for Clarification, and I'll be
happy to assist you further.

All the best,


search strategy -- Google search on [ hemophilia "units * year" ]

Request for Answer Clarification by shmidt-ga on 04 Jan 2005 13:51 PST
The GAO report is close to what I'm looking for. While I understand
how you arrived at 72,150 units using this report, I really need some
verification of the 1 billion number that's used to support it. The
GAO report states that "Total clotting factor use is about 1 billion
units per year." The word "about" raises a significant question here
because, with the small population of hemophilia patients, a few
hundred million units here or there makes a significant impact on the
authoritative per-patient average we're trying to arrive at.

In addition, the GAO report doesn't cite the source of the 1 billion
number, so I wonder if it's even from the same year as the CDC
utilization data. The GAO report was done in 2003, so when/where did
they pull that number (is it in the CDC report)? That could make a
significant difference since demand for factor has been consistently
on the rise nationwide while the population estimate by the CDC has
remained constant at 18,000 since as far back as 1997.

The following link contains a quote from a manufacturer representative
attending the 2001 Advisory Committee on Blood Safety and Availability
( He reported
that U.S. demand for factor VIII alone was up to 1.2 billion. (While
that source might not be as authoritative as GAO, it could be about
right considering the increase in prophylactic use.) Moreover, since
only 79% of hemophilia patients use factor VIII (hemophilia A),
there's another 21% of factor users unrepresented in that 1.2 billion
who use factor IX, making total U.S. consumption closer to 1.5 billion
in 2001. A 500 million unit difference for 13,860 patients would add
another 36,075 units to the 72,150 per patient average we're
estimating based on total U.S. consumer demand of 1 billion. Even a
200 million unit difference in total demand would add more than 14,000
units on average per patient.

One thought that occurs to me is that the GAO mistook factor VIII
demand for total U.S. factor demand, forgetting the other 21% with
factor IX. For instance, I noticed on the front page of the report it
states unequivocally, "Hemophilia patients use an average of 78,000
units of clotting factor annually," but then later on in page 6 it
clarifies that "annual use of clotting factor VIII for a person with
hemophilia A is 78,000."

Finally, the last thing that concerns me is that the 78,000 unit
number for hemophilia A is pretty old data (sourced from 1998 CDC
reports). Since then, the National Hemophilia Foundation has
recommended prophylactic use for all severe pediatric patients. This
recommendation, and medical practice in general, has increased factor
utilization dramatically over the past 5 years (the National
Hemophilia Foundation estimates 150,000 units per year for a 65 pound
kid on prophylaxis). With drugs in particular, a few years can be a
pretty steep increase. Something no more than 2 to 3 years old would
be ideal.

Since I didn't originally give you any time frame requirements for the
data, if you can verify that the 1 billion number is valid (ie, it can
be authoritatively sourced, it includes factor VIII and IX, AND it's
from 1998), then I think my question is sufficiently answered.
Otherwise, if you are able to dig up more recent data (within the past
2 to 3 years or so) within the same research parameters, I'll be sure
to compensate you for the added work.

Let me know what you think. Thanks.

Clarification of Answer by pafalafa-ga on 05 Jan 2005 07:43 PST
Hello Shmidt,

I can see you've done your homework on this topic!

I've done some additional homework myslef, and from what I can tell
(1) there isn't any more in-depth presentation of the methods to
arrive at the GAO numbers, other than the (fairly limited) discussion
in the GAO report itself, and (2) there aren't any published updated
numbers beyond what is presented in the GAO report.

Since there's nothing in the published literature that would appear to
help you, my recourse at this point is to contact the CDC directly to
see if they can confirm and/or update the numbers that GAO presented. 
I will do this, but I suspect it may take a few days, at least, to
pinpoint the right people, and have the necessary conversations.

So....sit tight, and stay tuned.  I'll let you know, probably next
week, what I come up with.


Request for Answer Clarification by shmidt-ga on 05 Jan 2005 08:01 PST
Understood...Maybe a good place to start is with Laura Dummit who
authored the GAO report. Her contact information is on page 2 of the


Clarification of Answer by pafalafa-ga on 05 Jan 2005 16:56 PST
Ms. Dummit owes me a phone call, and I'm hoping to hear back soon from
the CDC as well.

In the mean time, you might want to have a look at this:

as it cites a "mean factor VIII concentrate use was 128,517 units per
patient per year" in hemophilia A patients.

Clarification of Answer by pafalafa-ga on 09 Jan 2005 13:22 PST
A bit of an update.

I spoke with researchers from both the GAO and the CDC.  The GAO said
"we got those clotting factor numbers straight from CDC" and the CDC
says "we never gave them those numbers!"

This isn't a case of getting the run around -- both research staffs
were very helpful, and willing to dig a bit on my (actually, your)
behalf.  But they've lost some institutional memory on this topic, so
the digging may yet take a few days more.

I'll let you know as soon as I hear back from them.  Stay tuned....


Clarification of Answer by pafalafa-ga on 11 Jan 2005 16:59 PST
Hi Shmidt,

I haven't heard back from CDC yet, though I'm confident that I will. 
But I'm getting ready to head out of town for a while, so I wanted to
at least let you know what I've found thus far.

I did come up with one study that puts average clotting factor usage
in the same ballpark as the GAO numbers, and gives some individual
detail as well.  The time period is 1994 -- so it's still not anything
current -- but they're good numbers just the same.

The study is:  

American Journal of Hematology 59:36?41 (1998)
Viral Infections Among Patients With Hemophilia in the State of Georgia
Holly A. Hill and Sidney F. Stein

and the relevant data is:

TABLE II. Number of Concurrent Viral Infections (HIV, HBV
and/or HCV) and Amount of Clotting Factor Used in 1994

0 (33) 27,248  =  899,184
1 (18) 75,499 =  1,358,982
2 (44) 96,391 =  4,241,204
3 (38) 118,184 = 4,490,992


The first line is for patients with 0 viral infections (n=33), who
averaged 27,248 units/year, compared with (say) the last line of
patients with 3 viral infections, who averaged 118,184 units/year.  I
summed up the total unit usage, and arrived at a weighted average:

133 patients used 10,990,362 units = 82,634 per patient

The sample was for all hemophilia patients in the state, so it
includes both A and B.


This is probably all I will post for a while.  But when I'm back in
town towards the end of January, I'll be sure to let you know the
status of information from the CDC.


Clarification of Answer by pafalafa-ga on 24 Jan 2005 16:15 PST
Just a quick update.  I'm back from a great, very cold, vacation.  The
CDC let me know that they're still looking into this, and I expect to
hear from them in the next few days.

I'll let you know what they come up with.


Request for Answer Clarification by shmidt-ga on 24 Jan 2005 19:03 PST
Thanks for your diligence.

Clarification of Answer by pafalafa-ga on 29 Jan 2005 08:07 PST

I finally heard back from the CDC, and I must say that after numerous
phone calls and emails with the CDC and GAO staff, it's still not very
clear where/how -- exactly -- the numbers arose for clotting factor
use.  One problem may be the retirement of one of the key researchers
that worked on the hemophilia numbers, but whatever the issue -- the
researchers involved do not seem clear as to how the nubmers were
arrived at.

However, we can certainly put a time-frame on the data.  The response
I received from the CDC was:

I have been reviewing our contributions to this report. We provided
data in March 2002 that was primarily focused on differences in the
use of recombinant factor products based on Medicare status. We used
data collected in the Hemophilia Surveillance System as the basis.
This was a cooperative agreement with the State health departments in
6 states that was designed to identify all persons with hemophilia in
those states and to abstract demographic and clinical information
about these patients from medical records. We have published several
papers using these data including a paper that estimates the total US
hemophilia population size based on our findings in these six states
(Soucie, J.M., Jackson, D., Evatt, B. The occurrence of hemophilia in
the United States. Am J Hematol 1998; 59:288-294).

If you would like more details about the surveillance project or have
further questions, please feel free to contact me.

Best Regards,
Mike Soucie, PhD
Acting Associate Director for Science
Division of Hereditary Blood Disorders
National Center on Birth Defects and Developmental Disabilities
Centers for Disease Control and Prevention
1600 Clifton Road, MS E 64
Atlanta, GA 30333
Phone: 404-498-2753;  Fax: 404-498-2750

I do not have ready access to the "occurrence of hemophilia" paper
that Soucie cites, but from the abstract, it seems more oriented at
overall demographics than at getting at total clotting factor use:

Still, you might want to have a look at the full paper, or contact Dr.
Soucie for additional information.

This has been a long process, and I thank you for your patience. 
However, the process isn't finished until you say it is!  If there's
anything more I can do for you to fill in the blanks here, just let me
know and I'm at your service.


Request for Answer Clarification by shmidt-ga on 04 Feb 2005 13:59 PST
Pafalafa -
My wife gave birth last week and I will be back in the office on
Monday. Please give me a few more days to look at this. Thanks.

Clarification of Answer by pafalafa-ga on 04 Feb 2005 14:20 PST
Oh, that's wonderful to hear.  Hope that wife and baby (and you!) are all thriving.

Look forward to hearing back from you when the moment is right...


Request for Answer Clarification by shmidt-ga on 16 Feb 2005 12:31 PST
Pafalaf -
Thanks for all your research. It's been helpful. I've sent an email to
Mr. Soucie for additional information.


Clarification of Answer by pafalafa-ga on 16 Feb 2005 12:47 PST
Thanks...and best of luck with your work.

shmidt-ga rated this answer:4 out of 5 stars

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