RE: Bone Morphogenetic Protein (BMP)
Dear Sirs:
As the Vice-President of Clinical and Regulatory
Affairs for Stryker Biotech, the manufacturer of
Bone Morphogenetic Protein-7, the following
relevant information is shared to help CMS
appreciate that BMP-7, also known as OP-1 Putty,
need not proceed to a national coverage
determination.
The U. S. Food and Drug Administration (FDA)
granted market clearance to OP-1 Putty as a
Humanitarian Use Device on April 7, 2004 (#
H020008). As defined in the Federal Food, Drug,
and Cosmetic Act (21 CFR 814.124), the definition
of a Humanitarian Use Device (HUD) is a device
that is intended to benefit patients in the
treatment and diagnosis of diseases or conditions
that affect or is manifested in fewer than 4,000
individuals in the United States per year.
Therefore, as a Humanitarian Use Device, the
number of patients who have access to OP-1 Putty
in any given year is very limited, especially
eligible patients who are sixty-five years of age
or older. In addition, under the HUD exemption,
there are significant controls on how and where
in the body OP-1 Putty can be used.
The medical necessity of and patient selection
criteria for OP-1 Putty is best expressed by its
intended use - as an alternative to autograft in
compromised patients requiring revision of a
failed posterolateral (intertransverse) lumbar
spinal fusion, for whom autologous bone and bone
marrow harvest are not feasible or are not
expected to promote fusion. Examples of
compromising factors include osteoporosis,
smoking and diabetes. Spine surgeons implant OP-
1 Putty in a very select group of patients who
have exhausted conventional treatment options for
their lumbar pain. Conventional practices and
procedures have either failed or are not
feasible. Patients who have undergone prior
autograft harvest are often not candidates for
further autograft procedures. In addition,
patients who are obese, advanced in age, have
poor bone quality (osteoporosis) and/or
concurrent medical co-morbidities will likely
suffer unacceptable donor site morbidity and risk
of future anatomic disruption. OP-1 Putty is an
important biologic advancement that fills an
unmet clinical need in select patients. CMS
recognized this need when it granted OP-1 Putty
new technology add-on payment in FY2005. In
receiving new technology add-on payment, CMS
determined that OP-1 Putty represented an advance
in medical technology that substantially improved
patient care relative to technologies previously
available.
In conclusion, OP-1 Putty, as a humanitarian use
device, is a major advancement in spine surgery
for a small patient population that has been
unable to attain acceptable health outcomes with
conventional interventions. FDA developed and
published the Safe Medical Devices Act on June
26, 1996 to provide an incentive that would
encourage innovative manufacturers, such as
Stryker Biotech, to pursue discovery and
development of devices that would benefit only a
small patient population each year. Because OP-1
Putty is an HUD, Stryker Biotech and U.S.
hospitals that purchase it must comply with
certain FDA requirements. Specifically, as an
Humanitarian Use Device, OP-1 Putty may only be
used after a hospital’s Institutional Review
Board (IRB) has approved its use for
appropriately selected patients consistent with
its labeling intended use. Stryker Biotech’s
sales team works very closely with its hospital
customers to ensure that OP-1 Putty is only used
in patients requiring revision of a failed
posterolateral (intertransverse) lumbar spinal
fusion, for whom autologous bone and bone marrow
harvest are not feasible or are not expected to
promote fusion. In addition to this brief
comment, I would be happy to speak with
interested medical professionals at CMS, as well
as facilitate introductions to key opinion
leaders and clinical investigators for peer-to-
peer discussions. At your convenience, I can be
reached at Stryker Biotech, 35 South Street,
Hopkinton, Mass. 01748. My office phone number
is (508) 416-5326 In the meantime, the following
bibliography of peer-reviewed publications is
shared for your review. In advance, thank-you
for your time and consideration.
Bishop GB, Einhorn TA. Current and future
clinical applications of bone morphogenetic
proteins in orthopaedic trauma surgery. Int
Orthop. 2007 Dec;31(6):721-7. Epub 2007 Aug 1.
Carlisle E, Fischgrund JS. Bone morphogenetic
proteins for spinal fusion. Spine Journal.
2005:5:240S-249S.
Govender PV, et al. Use of osteogenic protein-1
in spinal fusion: literature review and
preliminary results in a prospective series of
high-risk cases. Neurosurg. Focus. 2002 Dec;13
(6): article 4.
Johnsson R, et al. Randomized radiostereometric
study comparing osteogenic protein-1 (BMP-7) an
autograft bone in human noninstrumented
posterolateral lumbar fusion. Spine. 2002;27
(23):2654-2661.
Vaccaro AR, et al. The safety and efficacy of OP-
1 (rhBMP-7) as a replacement for iliac crest
autograft for posterolateral lumbar arthrodesis:
minimum 4-year follow-up of a pilot study. Spine
J. 2008 May-Jun;8(3):457-65.
Vaccaro AR, et al. Comparison of OP-1 Putty
(rhBMP-7) to iliac crest autograft for
posterolateral lumbar arthrodesis: a minimum 2-
year follow-up pilot study. Spine. 2005 Dec 15;30
(24):2709-16.
Vaccaro AR, et al. A 2-year follow-up pilot study
evaluating the safety and efficacy of op-1 putty
(rhbmp-7) as an adjunct to iliac crest autograft
in posterolateral lumbar fusions. Eur Spine J.
2005 Sep;14(7):623-9.
Vaccaro AR, et al. A pilot study evaluating the
safety and efficacy of OP-1 Putty (rhBMP-7) as a
replacement for iliac crest autograft in
posterolateral lumbar arthrodesis for
degenerative spondylolisthesis. Spine. 2004 Sep
1;29(17):1885-92.
Vaccaro AR, et al. A pilot safety and efficacy
study of OP-1 putty (rhBMP-7) as an adjunct to
iliac crest autograft in posterolateral lumbar
fusions. Eur Spine J. 2003 Oct;12(5):495-500
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