The
impact of new (orphan) drug approvals on premature mortality from rare diseases
in
the U.S. and France, 1999-2007
Frank
R. Lichtenberg
10 January 2011
This research was supported by the American Enterprise Institute,
Novartis, and Pfizer
Inc. The sponsors placed no restrictions or limitations on data,
methods, or conclusions,
and had no right of review or control over the outcome of the
research. I am grateful to
Segolene Ayme, Marie Georget, and Ana Rath of Orphanet for
providing their data to
me.
The
impact of new (orphan) drug approvals on premature mortality from rare diseases
in
the U.S. and France, 1999-2007
Abstract
This paper investigates the impact of the introduction of new
orphan drugs on
premature mortality from rare diseases using longitudinal,
disease-level data obtained
from a number of major databases. The analysis is performed using
data from two
countries: the U.S. (during the period 1999-2006) and France
(during the period 2000-
2007). For both countries, we estimate models using two
alternative definitions of
premature mortality, several alternative criteria for inclusion in
the set of rare diseases,
and several values of the potential lag between new drug approvals
and premature
mortality reduction.
Both the U.S. and French estimates indicate that, overall,
premature mortality
from rare diseases is unrelated to the cumulative number of drugs
approved 0-2 years
earlier, but is significantly inversely related to the cumulative
number of drugs approved
3-4 years earlier. This delay is not surprising, since most
patients probably don’t have
access to a drug until several years after it has been launched.
Although the estimates for
the two countries are qualitatively similar, the estimated
magnitudes of the U.S.
coefficients are about four times as large as the magnitudes of
the French coefficients.
This may be partly due to greater errors in measuring dates of
drug introduction in
France.
Our estimates indicate that, in the U.S., potential years of life
lost to rare diseases
before age 65 (PYLL65) declined at an average annual rate of 3.3%,
and that, in the
absence of lagged new drug approvals, PYLL65 would have increased
at a rate of 0.9%.
Since the U.S. population age 0-64 was increasing at the rate of
1.0% per year, this means
that PYLL65 per person under 65 would have remained approximately
constant. The
reduction in the U.S. growth rate of PYLL65 attributable to lagged
new drug approvals
was 4.2%.
In France, PYLL65 declined at an average annual rate of 1.8%. The
estimates
imply that, in the absence of lagged new drug approvals, it would
have declined at a rate
of 0.6%. The reduction in the French growth rate of PYLL65
attributable to lagged new
drug approvals was 1.1%.
Frank
R. Lichtenberg
