Clark, M.

Copley-Merriman, K.

DeMuro, C.

Evans, E.

Fehnel, S.

Gnanasakthy, A.

Mordin, M.

Elsevier - Publisher Connector 

PHP97 PRO LABEL CLAIMS: AN ANALYSIS BASED ON A REVIEW OF PROS AMONG NEW MOLECULAR ENTITIES AND BIOLOGIC LICENSE APPLICATIONS 2006-2010 

negative binomial regression model, controlling for age, gender, race/ethnicity,
income education, general physical andmental health and co-morbidity burden. In
2014 depending on family income and employment status, the uninsured will ei-
ther obtain private or Medicaid insurance coverage, thus the analysis was re-
stricted to individuals less than 65 years with 12 months of continuous private or
Medicaid converge or uninsured for the whole year. Our study sample was nation-
ally representative on behalf of 71.3% of US population.RESULTS: Fivemeasures of
health care utilization were used (emergency room (ER) visits, outpatient visits,
office visit, inpatient visits, prescription use). Uninsured individuals had lower
utilization for all health care services except ER visits. Holding everything else
constant, the uninsuredwill have 1.98 (1.75-2.25) and 1.61 (1.24-2.1) times higher
expected rate of office based visits, 2.39 (1.81-3.15) and 2.62 (1.41-4.86) times
higher expected rate of outpatient visits, 2.17 (1.58-2.97) and 1.70 (1.11-2.62)
times higher expected rate of inpatient visits, 1.70 (1.53-1.89) and 1.92 (1.57-2.34)
times higher expected rate of prescribed medication use after obtaining private
or Medicaid coverage, respectively. CONCLUSIONS: Health care reform will in-
crease the demand for health services and prescribed medications, except ER use
for the uninsured. These results may be used by various stakeholders to estimate
expected changes in health care expenditures.
PHP96
REASONS FOR REJECTION OF PRO LABEL CLAIMS: AN ANALYSIS BASED ON A
REVIEW OF PRO USE AMONG NEW MOLECULAR ENTITIES AND BIOLOGIC
LICENSE APPLICATIONS 2006-2010
DeMuro C1, Clark M2, Mordin M2, Evans E1, Copley-Merriman K2, Fehnel SE1,
Gnanasakthy A3
1RTI Health Solutions, Research Triangle Park, NC, USA, 2RTI Health Solutions, Ann Arbor, MI,
USA, 3Novartis, East Hanover, NJ, USA
OBJECTIVES: Previous analyses of PRO label claims concentrated only on success-
ful label claims. The goal of this research was to explore the reasons why PRO label
claims were either denied or not sought.METHODS: Using the FDA Drug Approval
Report Webpage, all approved new molecular entities (NMEs) and biologic license
applications (BLAs) between February 2006 and December 2010 were identified. For
identified drug products,medical review sections frompublicly available summary
basis of approvals (SBAs) were reviewed to identify PRO endpoint status and any
FDA Study Endpoints and Label Development comments. RESULTS: Out of the 116
NMEs/BLAs identified and accompanying SBAs reviewed, 44.8% of products in-
cluded PROs as part of the pivotal studies; however, only 24.1% received PRO
claims. Primary reasons for denial (where data available) included a lack of dem-
onstration of content validity (inclusive of general measures such as the EQ5D and
SF-36) as well as use of PROs to assess symptoms in an open-label setting, lack of
consensus on clinically meaningful change, interpretation of or missing PRO data,
lack of measurement of full constellation of symptoms, issues of multiplicity and
concerns of “bias” in certain PRO measures. CONCLUSIONS: Nearly half (45%) of
submissions included PROs yet this rate is not reflected by claims granted. Under-
standing the nature of PRO claims granted under the current regulatory guidance is
important. Additionally, a clear understanding of claims denied yields valuable
insight into where sponsorsmay improve implementation of PROs in clinical trials
and the PRO evidence submitted in order to increase the likelihood of obtaining
PRO label claims.
PHP97
PRO LABEL CLAIMS: AN ANALYSIS BASED ON A REVIEW OF PROS AMONG NEW
MOLECULAR ENTITIES AND BIOLOGIC LICENSE APPLICATIONS 2006-2010
Mordin M1, Clark M1, DeMuro C2, Evans E2, Copley-Merriman K1, Fehnel S2,
Gnanasakthy A3
1RTI Health Solutions, Ann Arbor, MI, USA, 2RTI Health Solutions, Research Triangle Park, NC,
USA, 3Novartis Pharmaceuticals, East Hanover, NJ, USA
OBJECTIVES: Wilke and colleagues (2004) previously conducted a review of effec-
tiveness endpoints reported in the labels of new drug products approved in the
United States (US) between 1997-2002 to determine the extent and type of PRO
endpoints utilized. They reported that 30% of product labels reviewed included
PROs. Our study aimed to build upon this work by describing the current state of
PRO label claims granted for new molecular entities (NMEs) and biologic license
applications (BLAs) following release of the draft and final FDA PRO Guidance doc-
uments (i.e., since February 2006). METHODS: Using the FDA Drug Approval Re-
ports webpage, all FDA approved NMEs and BLAs between February 2006 and De-
cember 2010 were identified. Generic products with tentative approvals granted in
this periodwere excluded. For all identified drug products,medical review sections
from publicly available summary basis of approvals (SBAs) were reviewed to iden-
tify PRO endpoint status. Product labels (indication, clinical trials sections) were
reviewed to determine the number and type of PRO claims. RESULTS: Of the 116
NMEs/BLAs identified, 28 (24.1%) were granted PRO claims. The majority (n24)
were for signs and symptoms. Nine of the signs and symptom claims were pain-
related. Of the 28 products with PRO claims, a PRO was a primary endpoint for 20
(71%). All 20 of these primary endpoints were symptom-related and the majority
(12 of 20) were collected via diary. CONCLUSIONS: PRO claims continue to be ap-
proved by FDA, with 24% of NMEs and BLAs granted PRO claims. Successful PRO
label claims over the past five years have been largely in support of treatment
benefit for symptoms specified as primary endpoints. The proportion of NMEswith
PRO label claims during the post-guidance period (24.1%)was lower than that of the
pre-guidance period (30%).
PHP98
A TREND ANALYSIS OF NEW MOLECULAR ENTITIES WITHDRAWN FOR SAFETY
REASONS FROM 1980 TO 2009 IN THE UNITED STATES
Seoane-Vazquez E1, Rodriguez-Monguio R2, Qureshi ZP3, Szeinbach SL4
1Massachusetts College of Pharmacy & Health Sciences, Boston, MA, USA, 2University of
Massachusetts, Amherst,, MA, USA, 3University of South Carolina, Columbia, SC, USA, 4Ohio
State University, Columbus, OH, USA
OBJECTIVES: Besides the influence of economic factors, prescribing patterns, and
market dynamics, decisions to withdraw products from the market are driven by
concerns over safety. This study evaluated new molecular entities (NMEs) ap-
proved by the FDA in the period 1980-2009 that were withdrawn from the market
for safety reasons.METHODS:Datawere obtained from the FDA and theUS Federal
Register. Descriptive analyses were used to classify product discontinuations by
therapeutic category, year, and reason for discontinuation. RESULTS: There were
740NMEs approved by the FDAduring the study period. As of December 1, 2010, the
number of drugs discontinuedwas 118 (15.9%). Safetywas the reason forwithdraw-
ing 27 (3.6%) drugs from the market. Therapeutic categories with the most safety
withdrawals as a percentage of approvals in the 1980s were respiratory (28.6%),
musculo-skeletal (23.1%), and nervous system (7.4%). During the 1990s, classes
with the most safety withdrawals as a percentage of approvals were musculo-
skeletal (18.8%), alimentary tract and metabolism (12.0%), and blood and blood
forming organs (7.7%). Therapeutic categories affected by safety withdrawals as a
percentage of approvals in the 2000s were musculo-skeletal (20.0%), alimentary
tract and metabolism (4.2%), and antineoplastic and immunomodulating agents
(3.2%). Major problems that spurred safety withdrawal were hepatic toxicity, se-
vere cardiovascular effects, and gastrointestinal issues. Average time from ap-
proval to safety withdrawal was 5.9 (SD 5.0) years, with a range of 0.3-18.2 years,
and a 95% CI of 4.0-7.8 years. CONCLUSIONS: Approximately one in seven NMEs
approved in the period 1980-2009 was discontinued from the market. Less than
one-quarter of the discontinuations were attributed to safety reasons. Products
remained in the market for an average of six years before safety withdrawal. An
ongoing evaluation of new drugs through their product life cycle is important to
determine their long-term safety and value to society.
PHP99
WILL BIOPHARMACEUTICAL INNOVATION STILL BE A PICTURE OF HEALTH
AFTER IMPLEMENTATION OF HEALTH CARE REFORM?
Ross KD
University of Washington, Seattle, WA, USA
OBJECTIVES: To determine what impact shortening or lengthening the data exclu-
sivity period (DEP) for biologic drugs has on innovation. As a part of this, the goal is
to determine what effects on innovation the 12-year DEP included in healthcare
reform will have. METHODS: A simulation model is developed to assess the prof-
itability of candidate drugs under varying DEPs. All costs and revenues are dis-
counted. The drugs are then grouped into 10-drug portfolios and the profitability of
each portfolio is determined. The percentage of portfolios that are profitable under
each DEP length is divided by the percentage of portfolios that are profitable under
a indefinite DEP to give a relative level of innovation. RESULTS: A DEP of 0 years
yields a 60% decrease in the level of innovation and there are no increases in
innovation for DEPs above 34 years. For a DEP of 12 years, there is an expected 8.1%
decrease in the level of innovation. CONCLUSIONS: The 12-year DEP implemented
as a part of healthcare reform is likely to decrease innovation in biologic drugs. The
expected 8.1% decrease in innovation may or may not be worth the expected de-
crease in prices once biosimilar competitors enter. The model also indicates that
there would be no returns to innovation by increasing DEP above 34 years, and as
such, it is likely that this would represent a maximum when selecting a DEP.
PHP100
PRICING AND REIMBURSEMENT OF ORPHAN DRUGS IN CANADA
Kumar J, Bachman EM
HERON Evidence Development LLC, Somerville, NJ, USA
OBJECTIVES: The Canadian Organization of Rare Diseases (CORD) defines a rare
disease as one that afflicts less that 1 person in 200 000. Significant market access
and pricing challenges exist for ODs in Canada both at a federal and the provincial
level. The scope of this study is to describe theODs regulations in Canada, evidence
requirements by the national regulatory agency, national and regional funding
criteria, market access challenges associated with ODs, and approaches to obtain
access to ODs in Canada.METHODS: Non-systematic PubMed search, Health Can-
ada, the Canadian Agency for Drug and Technology in Health (CADTH), Common
Drug Review (CDR), Canadian Organization of Rare Diseases (CODR) and different
provinces Ministries of Health websites. RESULTS: Health Canada reviews ODs to
ensure that the drugmeets the criteria of efficacy, safety, andmanufacturing qual-
ity. The CDR conducts the clinical and cost effectiveness review compared to ex-
isting therapies and makes positive or negative recommendations to provinces to
list ODs in their respective formularies. At the federal level, pricing of ODs is reg-
ulated by Patented Medicine Pricing Review Board (PMPRB). At the provincial level,
different provinces can make their own independent reimbursement decision ir-
respective of CDR’s recommendation. Due to the large budget impact of ODs, most
provinces do not provide access. The specialized access mechanism, criteria for
eligibility, extent of coverage, and different data requirement to obtain access in
three important provinces of Canada (Ontario, Alberta, and Quebec) will be dis-
cussed in the poster. CONCLUSIONS: In the absence of a national orphan drug
policy, patients suffering from rare diseases face challenges in obtaining access to
ODs in Canada. However, significant opportunities exist for manufacturers to pro-
vide access to ODs in Canada.
A29V A L U E I N H E A L T H 1 4 ( 2 0 1 1 ) A 1 – A 2 1 4


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PHP97 PRO LABEL CLAIMS: AN ANALYSIS BASED ON A REVIEW OF PROS AMONG NEW MOLECULAR ENTITIES AND BIOLOGIC LICENSE APPLICATIONS 2006-2010

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