PERSPECTIVE
H E A L T H CARE FOR I M M I G R A N T S
H e a lth C are fo r Im m ig ra n ts — Im p lic a tio n s o f O b a m a ’s Executive A ction Benjamin D. Sommers, M.D., Ph.D., and Wendy E. Parmet, J.D.
n November 20, 2014, Pres ident Barack Obama an nounced his intention to grant millions of undocumented immi grants a reprieve from the threat o f deportation, along with the possibility o f legal employment in the United States. The an nouncement came shortly after midterm elections that saw Re publicans take control o f the Senate and bolster their majority in the House o f Representatives, and it followed more than a year o f congressional gridlock over the comprehensive immigrationreform bill passed by the Senate. The subsequent decision by a federal district judge in Texas to put President Obama’s plan on hold has cast into doubt a policy that — if ultimately upheld — could have substantial effects on the health care system. The cornerstone of the Presi dent’s policy is a plan to allow up to 5 million undocumented immigrants (“covered immi grants”) to live and work in the United States. Modeled on the 2012 Deferred Action for Child hood Arrivals (DACA) program, which applied to young adults who arrived in the United States as children, the new policy will allow many more adults who have been here since 2010 to re ceive deferred-action status. The population affected by the policy is one that currently experiences major disparities in health care coverage and access. Immigrants are far more likely than nativeborn residents to be uninsured:
O
among adults, an estimated 40% o f legal permanent residents and 71% of undocumented immi grants are uninsured, as com pared with 15% of U.S.-born citi zens.1 Not surprisingly, health care utilization is far lower among immigrants than among citizens.2 Although the President’s poli cy does not explicitly address health care, it will most likely in crease access to insurance among covered immigrants and mem bers o f their family through a va riety of direct and indirect means (see table). Many of the coverage gains are likely to occur in the private sector. The President’s plan to issue legal work permits for un documented immigrants could open the doors for many indi viduals and families to gain em ployer-sponsored health insurance for the first time. An estimated 29% o f undocumented immi grants already have private insur ance, and many more immi grants are likely to find jobs that offer benefits once they are legal ly authorized to work — though these gains may be tempered somewhat by the fact that immi grants disproportionately work in jobs that are less likely to in clude health benefits.1 Moreover, under the employer mandate included in the Afford able Care Act (ACA), employers with 100 or more full-time em ployees have to pay a penalty if any of their full-time workers re ceive tax credits to purchase in
N E N G L J M E D 3 7 2 ;1 3
N E J M .O R G
M ARCH 26, 2015
surance through an exchange. To the extent that the mandate en courages employers to cover all employees, covered immigrants — who may find it easier to gain employment in areas from which they have previously been barred — will benefit. On the other hand, because covered immi grants will remain ineligible for premium tax credits, employers will not have to worry about pay ing a penalty for their receiving premium subsidies. Hence, al though the ACA prohibits em ployers from discriminating against workers who purchase insurance through the exchang es, it may create a perverse in centive for employers who don’t offer affordable coverage to hire covered immigrants. In addition to increasing covered im migrants’ access to employer-provided insurance, the President’s plan may enable cov ered immigrants to take better advantage of the many provisions in the ACA that apply to private plans purchased directly from in surers, such as the elimination of exclusions for preexisting con ditions. Notably, however, the Obama administration has indicated that immigrants covered under the new policy will continue to be excluded from the ACA’s ex changes and tax credits. Similar ly, immigrants targeted by the executive action will for the most part remain ineligible for feder ally funded Medicaid, though some states do provide publicly
1187
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HEALTH
CARE FOR I M M I G R A N T S
P o te n tia l E ffe c ts o f D e fe rre d A c tio n f o r U n d o c u m e n te d Im m ig r a n t s o n H e a lth I n s u r a n c e C o v e r a g e .* T y p e o f H e a lt h In s u r a n c e E m p lo y e r-s p o n s o re d h e a lth in s u r a n c e In d iv id u a l p r iv a te in s u r a n c e n o t s o ld t h r o u g h
P o p u la tio n A ffe c te d U n d o c u m e n te d im m ig r a n ts re c e iv in g d e fe rre d a c tio n s ta tu s a n d t h e ir d e p e n d e n ts U n d o c u m e n te d im m ig r a n ts re c e iv in g d e fe rre d a c tio n s ta tu s a n d t h e ir d e p e n d e n ts
an exchange M e d ic a id
C o m m e n ts W o r k p e r m it s m a y e x p a n d e m p lo y m e n t o p p o r t u n it ie s a n d th e lik e lih o o d o f b e in g o ffe r e d h e a lth b e n e fits . W o r k p e r m its m a y in c re a s e in c o m e a n d w illin g n e s s t o a p p ly f o r c o v e ra g e . A C A re g u la tio n s (e.g., n o e x c lu s io n s f o r p r e e x is tin g c o n d itio n s , c o m m u n ity - r a te d p r e m iu m s ) a p p ly to th e s e p la n s .
Legal re s id e n ts a n d c itiz e n s w ith u n d o c u m e n te d f a m ily m e m b e r s
W ith d e p o r t a t io n r is k re m o v e d , e lig ib le f a m ily m e m b e r s m a y be m o r e lik e ly to s ig n u p fo r p u b lic c o v e ra g e . U n d o c u m e n te d im m ig r a n ts w ill g e n e ra lly s till n o t be e lig ib le f o r fe d e ra l M e d ic a id fu n d s .
A C A e x c h a n g e p la n s
Legal re s id e n ts a n d c itiz e n s w ith u n d o c u m e n te d fa m ily m e m b e r s
W ith d e p o r t a tio n ris k re m o v e d , e lig ib le fa m ily m e m b e r s m a y be m o r e lik e ly t o a p p ly f o r c o v e ra g e a n d ta x c re d its . U n d o c u m e n te d im m ig r a n t s w ill s till n o t be e lig ib le f o r e x c h a n g e p la n s o r s u b s id ie s .
M e d ic a r e
U n d o c u m e n te d im m ig r a n ts re c e iv in g d e fe rre d a c tio n s ta tu s
T h e W h ite H o u s e h a s s u g g e s te d it m a y g r a n t M e d ic a r e e lig ib ilit y t o t h o s e m e e tin g p a y r o ll- ta x - c o n tr ib u t io n r e q u ir e m e n ts a n d o t h e r e lig ib ilit y c r ite r ia (re la te d t o age, h e a lth s ta tu s , o r d is a b ilit y ) .
* A C A d e n o te s A ffo r d a b le C a re A c t.
funded health insurance for some classes of undocumented immi grants. To the extent that work permits lead to an increase in taxes paid by undocumented im migrants, ongoing exclusion of these immigrants from the pro grams supported by such taxes would be increasingly inequitable. In contrast, there have been reports that the administration may deem covered immigrants “lawfully present” for the pur poses o f Medicare — which would mean that they could re ceive coverage if they met the other eligibility criteria, which generally include having a family or personal history of contribut ing payroll taxes, in addition to being at least 65 years old or hav ing a disability or end-stage renal disease.3 Although most of the newly covered immigrants are too young to qualify for Medi care now, this policy would a f feet those who develop disabili ties after working the requisite period and for those who age into Medicare in the future. Although the President’s de ferred-action policy is likely to
1 1 8 8
have a positive effect on insur ance coverage of undocumented immigrants, it may, counterintui tively, do more to increase access to insurance for legal immigrants and even citizens than it does for those directly affected by the planned executive order. Previous research has shown that in mixedstatus families (those in which some members are undocument ed and others are legal immi grants or citizens), the perceived threat of deportation is associat ed with lower participation rates in programs such as Medicaid and the Children’s Health Insur ance Program.4’5 Eliminating the threat of deportation may induce undocumented parents to sign their U.S.-born children up for coverage and legal immigrants with undocumented relatives to enroll in Medicaid or subsidized exchange plans. The resulting changes are likely to be greatest in Hispanic and Asian communi ties, including children, and they may narrow some of the coun try’s racial and ethnic disparities in coverage. Similarly, the reduced threat
N EN G LJ
M E D 3 7 2 ;1 3
N E J M .O R G
M ARCH 26, 2015
of deportation may mitigate im migrants’ mistrust as they decide whether to pursue needed medi cal care, regardless of their insur ance status. Ironically, any result ing increase in utilization may exacerbate the financial strain placed on safety-net providers that disproportionately care for immi grants, since many immigrants will remain uninsured if they are ineligible for Medicaid or premi um tax credits. Policymakers will therefore need to continue fund ing streams to support providers who care for uninsured immi grants. Thus, there is reason to ex pect that President Obama’s new immigration policy will increase insurance coverage and health care access among undocument ed immigrants and their relatives. But it remains unclear exactly how these changes will play out, given that any executive policy — especially one as contentious as this one — is temporary by na ture. The extent to which the President’s policy will encourage uninsured people in immigrant communities (undocumented or
P E R S P E C TIV E
4
^ -■
HEALTH
otherwise) to emerge from the shadows and obtain coverage is largely contingent on the per ceived political and legal climate. Earlier this year, Republican Congressional leaders attempted, ultimately unsuccessfully, to tie funding for the Department of Homeland Security to the dis mantlement of the program. Meanwhile, the decision by a federal judge to temporarily halt the policy in response to a law suit filed by 26 states raises the An audio interview possibility that the w ith Dr. Sommers program’s fate will
is a v a ila b le a tN E J M .o rg
£nd
up
bdng
de_
cided by the Supreme Court. These uncertainties may leave many immigrants reluctant to come forward and seek coverage. And any gains may be ephemeral if the policy is ultimately re versed by the courts, legislative action, or a future President.
Overall, the President’s policy — if implemented — is likely to increase insurance coverage in immigrant communities. Never theless, most undocumented immigrants in the United States will remain significantly limited in their ability to obtain health insur ance and to access needed health care.2 Beyond the surrounding le gal and political controversies, this executive action is no substi tute for a comprehensive immi gration-reform law that address es the health needs of immigrants. Given the gridlock in Washington, D.C., however, such a law seems an improbable aspiration. Disclosure forms provided fay the authors are available with the full text of this article at NEJM.org.
CARE FOR I M M I G R A N T S
1. Capps R, Bachm eierJD, Fix M , Van HookJ. A dem ographic, socioe cono m ic, and health coverage p ro file o f un auth orized im m ig ra n ts in the U nited States. W ashington, DC: M igra tio n Policy In stitu te , May 2013. 2. Bustam ante AV, C henJ. H ealth exp endi ture dynam ics and years o f U.S. residence: analyzing spending disparities am ong Latinos by c itiz e n s h ip /n a tiv ity status. H ealth Serv Res 2012;47:794-818. 3. Tum ulty K. Illegal im m ig ran ts cou ld re ceive Social Security, M edicare under Obam a action. W ashington Post. N ovem ber 25, 2014 (h ttp ://w w w .w a s h in g to n p o s t.c o m /p o litic s / illegal-immigrants-could-receive-social-security -m edicare-under-obam a-action/2014/11/25/ 5 7 1 c a e fe -7 4 d 4 -lle 4 -b d lb -0 3 0 0 9 b d 3 e 9 8 4 _ s tory.htm l). 4. Som m ers BD, Tom asi MR, Swartz K, Ep stein A M . Reasons fo r the w ide varia tion in M edicaid p a rtic ip a tio n rates am ong states hold lessons fo r coverage expansion in 2014. Health A ff (M illw ood) 2012;31:909-19. [Errata, H ealth A ff (M illw o o d ) 2012;31:1650, 2831.] 5. W atson T. Inside th e refrigerator: im m i gration e n force m ent and c h illin g effects in M edicaid p a rtic ip a tio n . C am bridge, M A: Na tio n a l Bureau o f E conom ic Research, A ugust
2010
.
From th e D e p a rtm e n t o f H ealth Policy and M anagem ent, Harvard School o f Public
DOI: 10.1056/NEJMpl414949
Health (B.D.S.), and N ortheastern University
C opyright © 2 015 M assachusetts M e d ic a l Society.
School o f Law (W.E.P.) — both in Boston.
Forced V ita l C apacity in Id io p a th ic P u lm o n ary Fibrosis — F D A R eview o f P irfenidone and N in te d a n ib Banu A. Karimi-Shah, M.D., and Badrul A. Chowdhury, M.D., Ph.D.
diopathic pulmonary fibrosis (IPF) is a devastating disease o f unknown cause, and for years the only effective treatment avail able was lung transplantation. In October 2014, two drugs be came available in the United States for patients with IPF when the Food and Drug Administration (FDA) approved pirfenidone (Esbriet, InterMune) and nintedanib (Ofev, Boehringer Ingelheim).1 Both drugs posed a similar regula tory challenge, in that the primary efficacy variable studied in both cases was the change in forced vital capacity (FVC). FVC, a mea sure o f lung function, had not been established as a surrogate
I
for clinically meaningful benefit in IPF. Pirfenidone, a pyridone, is thought to act through anti inflammatory and antifibrotic pathways, although its exact mechanism is unknown. The FDA had declined to approve an ini tial marketing application for pirfenidone in a first review cycle in 2010. At that time, two 72week studies were submitted for review. Their primary efficacy variable was the absolute change in the percentage of the predict ed FVC from baseline to week 72. In one study, pirfenidone had not been shown to have a signifi cantly greater effect than placebo
N ENG LJ
M E D 3 7 2 ;1 3
N E J M .O R G
M ARCH
26, 2015
on that measure. At the time of the agency’s decision not to ap prove pirfenidone, outside experts, including many members of the Pulmonary-Allergy Drugs Advi sory Committee convened in May 2010, supported that decision.1’2 Before resubmitting its mar keting application in 2014, the sponsor conducted a new 52-week study in which the primary effi cacy end point — a decline in FVC from baseline to week 52 that was significantly smaller than that in the placebo group — was met. With two studies demonstrating a statistically sig nificant effect on FVC and pro viding supportive evidence regard-
1189
Copyright © Massachusetts Medical Society 2015.
H E A L T H CARE FOR I M M I G R A N T S
H e a lth C are fo r Im m ig ra n ts — Im p lic a tio n s o f O b a m a ’s Executive A ction Benjamin D. Sommers, M.D., Ph.D., and Wendy E. Parmet, J.D.
n November 20, 2014, Pres ident Barack Obama an nounced his intention to grant millions of undocumented immi grants a reprieve from the threat o f deportation, along with the possibility o f legal employment in the United States. The an nouncement came shortly after midterm elections that saw Re publicans take control o f the Senate and bolster their majority in the House o f Representatives, and it followed more than a year o f congressional gridlock over the comprehensive immigrationreform bill passed by the Senate. The subsequent decision by a federal district judge in Texas to put President Obama’s plan on hold has cast into doubt a policy that — if ultimately upheld — could have substantial effects on the health care system. The cornerstone of the Presi dent’s policy is a plan to allow up to 5 million undocumented immigrants (“covered immi grants”) to live and work in the United States. Modeled on the 2012 Deferred Action for Child hood Arrivals (DACA) program, which applied to young adults who arrived in the United States as children, the new policy will allow many more adults who have been here since 2010 to re ceive deferred-action status. The population affected by the policy is one that currently experiences major disparities in health care coverage and access. Immigrants are far more likely than nativeborn residents to be uninsured:
O
among adults, an estimated 40% o f legal permanent residents and 71% of undocumented immi grants are uninsured, as com pared with 15% of U.S.-born citi zens.1 Not surprisingly, health care utilization is far lower among immigrants than among citizens.2 Although the President’s poli cy does not explicitly address health care, it will most likely in crease access to insurance among covered immigrants and mem bers o f their family through a va riety of direct and indirect means (see table). Many of the coverage gains are likely to occur in the private sector. The President’s plan to issue legal work permits for un documented immigrants could open the doors for many indi viduals and families to gain em ployer-sponsored health insurance for the first time. An estimated 29% o f undocumented immi grants already have private insur ance, and many more immi grants are likely to find jobs that offer benefits once they are legal ly authorized to work — though these gains may be tempered somewhat by the fact that immi grants disproportionately work in jobs that are less likely to in clude health benefits.1 Moreover, under the employer mandate included in the Afford able Care Act (ACA), employers with 100 or more full-time em ployees have to pay a penalty if any of their full-time workers re ceive tax credits to purchase in
N E N G L J M E D 3 7 2 ;1 3
N E J M .O R G
M ARCH 26, 2015
surance through an exchange. To the extent that the mandate en courages employers to cover all employees, covered immigrants — who may find it easier to gain employment in areas from which they have previously been barred — will benefit. On the other hand, because covered immi grants will remain ineligible for premium tax credits, employers will not have to worry about pay ing a penalty for their receiving premium subsidies. Hence, al though the ACA prohibits em ployers from discriminating against workers who purchase insurance through the exchang es, it may create a perverse in centive for employers who don’t offer affordable coverage to hire covered immigrants. In addition to increasing covered im migrants’ access to employer-provided insurance, the President’s plan may enable cov ered immigrants to take better advantage of the many provisions in the ACA that apply to private plans purchased directly from in surers, such as the elimination of exclusions for preexisting con ditions. Notably, however, the Obama administration has indicated that immigrants covered under the new policy will continue to be excluded from the ACA’s ex changes and tax credits. Similar ly, immigrants targeted by the executive action will for the most part remain ineligible for feder ally funded Medicaid, though some states do provide publicly
1187
PERSPECTIVE
HEALTH
CARE FOR I M M I G R A N T S
P o te n tia l E ffe c ts o f D e fe rre d A c tio n f o r U n d o c u m e n te d Im m ig r a n t s o n H e a lth I n s u r a n c e C o v e r a g e .* T y p e o f H e a lt h In s u r a n c e E m p lo y e r-s p o n s o re d h e a lth in s u r a n c e In d iv id u a l p r iv a te in s u r a n c e n o t s o ld t h r o u g h
P o p u la tio n A ffe c te d U n d o c u m e n te d im m ig r a n ts re c e iv in g d e fe rre d a c tio n s ta tu s a n d t h e ir d e p e n d e n ts U n d o c u m e n te d im m ig r a n ts re c e iv in g d e fe rre d a c tio n s ta tu s a n d t h e ir d e p e n d e n ts
an exchange M e d ic a id
C o m m e n ts W o r k p e r m it s m a y e x p a n d e m p lo y m e n t o p p o r t u n it ie s a n d th e lik e lih o o d o f b e in g o ffe r e d h e a lth b e n e fits . W o r k p e r m its m a y in c re a s e in c o m e a n d w illin g n e s s t o a p p ly f o r c o v e ra g e . A C A re g u la tio n s (e.g., n o e x c lu s io n s f o r p r e e x is tin g c o n d itio n s , c o m m u n ity - r a te d p r e m iu m s ) a p p ly to th e s e p la n s .
Legal re s id e n ts a n d c itiz e n s w ith u n d o c u m e n te d f a m ily m e m b e r s
W ith d e p o r t a t io n r is k re m o v e d , e lig ib le f a m ily m e m b e r s m a y be m o r e lik e ly to s ig n u p fo r p u b lic c o v e ra g e . U n d o c u m e n te d im m ig r a n ts w ill g e n e ra lly s till n o t be e lig ib le f o r fe d e ra l M e d ic a id fu n d s .
A C A e x c h a n g e p la n s
Legal re s id e n ts a n d c itiz e n s w ith u n d o c u m e n te d fa m ily m e m b e r s
W ith d e p o r t a tio n ris k re m o v e d , e lig ib le fa m ily m e m b e r s m a y be m o r e lik e ly t o a p p ly f o r c o v e ra g e a n d ta x c re d its . U n d o c u m e n te d im m ig r a n t s w ill s till n o t be e lig ib le f o r e x c h a n g e p la n s o r s u b s id ie s .
M e d ic a r e
U n d o c u m e n te d im m ig r a n ts re c e iv in g d e fe rre d a c tio n s ta tu s
T h e W h ite H o u s e h a s s u g g e s te d it m a y g r a n t M e d ic a r e e lig ib ilit y t o t h o s e m e e tin g p a y r o ll- ta x - c o n tr ib u t io n r e q u ir e m e n ts a n d o t h e r e lig ib ilit y c r ite r ia (re la te d t o age, h e a lth s ta tu s , o r d is a b ilit y ) .
* A C A d e n o te s A ffo r d a b le C a re A c t.
funded health insurance for some classes of undocumented immi grants. To the extent that work permits lead to an increase in taxes paid by undocumented im migrants, ongoing exclusion of these immigrants from the pro grams supported by such taxes would be increasingly inequitable. In contrast, there have been reports that the administration may deem covered immigrants “lawfully present” for the pur poses o f Medicare — which would mean that they could re ceive coverage if they met the other eligibility criteria, which generally include having a family or personal history of contribut ing payroll taxes, in addition to being at least 65 years old or hav ing a disability or end-stage renal disease.3 Although most of the newly covered immigrants are too young to qualify for Medi care now, this policy would a f feet those who develop disabili ties after working the requisite period and for those who age into Medicare in the future. Although the President’s de ferred-action policy is likely to
1 1 8 8
have a positive effect on insur ance coverage of undocumented immigrants, it may, counterintui tively, do more to increase access to insurance for legal immigrants and even citizens than it does for those directly affected by the planned executive order. Previous research has shown that in mixedstatus families (those in which some members are undocument ed and others are legal immi grants or citizens), the perceived threat of deportation is associat ed with lower participation rates in programs such as Medicaid and the Children’s Health Insur ance Program.4’5 Eliminating the threat of deportation may induce undocumented parents to sign their U.S.-born children up for coverage and legal immigrants with undocumented relatives to enroll in Medicaid or subsidized exchange plans. The resulting changes are likely to be greatest in Hispanic and Asian communi ties, including children, and they may narrow some of the coun try’s racial and ethnic disparities in coverage. Similarly, the reduced threat
N EN G LJ
M E D 3 7 2 ;1 3
N E J M .O R G
M ARCH 26, 2015
of deportation may mitigate im migrants’ mistrust as they decide whether to pursue needed medi cal care, regardless of their insur ance status. Ironically, any result ing increase in utilization may exacerbate the financial strain placed on safety-net providers that disproportionately care for immi grants, since many immigrants will remain uninsured if they are ineligible for Medicaid or premi um tax credits. Policymakers will therefore need to continue fund ing streams to support providers who care for uninsured immi grants. Thus, there is reason to ex pect that President Obama’s new immigration policy will increase insurance coverage and health care access among undocument ed immigrants and their relatives. But it remains unclear exactly how these changes will play out, given that any executive policy — especially one as contentious as this one — is temporary by na ture. The extent to which the President’s policy will encourage uninsured people in immigrant communities (undocumented or
P E R S P E C TIV E
4
^ -■
HEALTH
otherwise) to emerge from the shadows and obtain coverage is largely contingent on the per ceived political and legal climate. Earlier this year, Republican Congressional leaders attempted, ultimately unsuccessfully, to tie funding for the Department of Homeland Security to the dis mantlement of the program. Meanwhile, the decision by a federal judge to temporarily halt the policy in response to a law suit filed by 26 states raises the An audio interview possibility that the w ith Dr. Sommers program’s fate will
is a v a ila b le a tN E J M .o rg
£nd
up
bdng
de_
cided by the Supreme Court. These uncertainties may leave many immigrants reluctant to come forward and seek coverage. And any gains may be ephemeral if the policy is ultimately re versed by the courts, legislative action, or a future President.
Overall, the President’s policy — if implemented — is likely to increase insurance coverage in immigrant communities. Never theless, most undocumented immigrants in the United States will remain significantly limited in their ability to obtain health insur ance and to access needed health care.2 Beyond the surrounding le gal and political controversies, this executive action is no substi tute for a comprehensive immi gration-reform law that address es the health needs of immigrants. Given the gridlock in Washington, D.C., however, such a law seems an improbable aspiration. Disclosure forms provided fay the authors are available with the full text of this article at NEJM.org.
CARE FOR I M M I G R A N T S
1. Capps R, Bachm eierJD, Fix M , Van HookJ. A dem ographic, socioe cono m ic, and health coverage p ro file o f un auth orized im m ig ra n ts in the U nited States. W ashington, DC: M igra tio n Policy In stitu te , May 2013. 2. Bustam ante AV, C henJ. H ealth exp endi ture dynam ics and years o f U.S. residence: analyzing spending disparities am ong Latinos by c itiz e n s h ip /n a tiv ity status. H ealth Serv Res 2012;47:794-818. 3. Tum ulty K. Illegal im m ig ran ts cou ld re ceive Social Security, M edicare under Obam a action. W ashington Post. N ovem ber 25, 2014 (h ttp ://w w w .w a s h in g to n p o s t.c o m /p o litic s / illegal-immigrants-could-receive-social-security -m edicare-under-obam a-action/2014/11/25/ 5 7 1 c a e fe -7 4 d 4 -lle 4 -b d lb -0 3 0 0 9 b d 3 e 9 8 4 _ s tory.htm l). 4. Som m ers BD, Tom asi MR, Swartz K, Ep stein A M . Reasons fo r the w ide varia tion in M edicaid p a rtic ip a tio n rates am ong states hold lessons fo r coverage expansion in 2014. Health A ff (M illw ood) 2012;31:909-19. [Errata, H ealth A ff (M illw o o d ) 2012;31:1650, 2831.] 5. W atson T. Inside th e refrigerator: im m i gration e n force m ent and c h illin g effects in M edicaid p a rtic ip a tio n . C am bridge, M A: Na tio n a l Bureau o f E conom ic Research, A ugust
2010
.
From th e D e p a rtm e n t o f H ealth Policy and M anagem ent, Harvard School o f Public
DOI: 10.1056/NEJMpl414949
Health (B.D.S.), and N ortheastern University
C opyright © 2 015 M assachusetts M e d ic a l Society.
School o f Law (W.E.P.) — both in Boston.
Forced V ita l C apacity in Id io p a th ic P u lm o n ary Fibrosis — F D A R eview o f P irfenidone and N in te d a n ib Banu A. Karimi-Shah, M.D., and Badrul A. Chowdhury, M.D., Ph.D.
diopathic pulmonary fibrosis (IPF) is a devastating disease o f unknown cause, and for years the only effective treatment avail able was lung transplantation. In October 2014, two drugs be came available in the United States for patients with IPF when the Food and Drug Administration (FDA) approved pirfenidone (Esbriet, InterMune) and nintedanib (Ofev, Boehringer Ingelheim).1 Both drugs posed a similar regula tory challenge, in that the primary efficacy variable studied in both cases was the change in forced vital capacity (FVC). FVC, a mea sure o f lung function, had not been established as a surrogate
I
for clinically meaningful benefit in IPF. Pirfenidone, a pyridone, is thought to act through anti inflammatory and antifibrotic pathways, although its exact mechanism is unknown. The FDA had declined to approve an ini tial marketing application for pirfenidone in a first review cycle in 2010. At that time, two 72week studies were submitted for review. Their primary efficacy variable was the absolute change in the percentage of the predict ed FVC from baseline to week 72. In one study, pirfenidone had not been shown to have a signifi cantly greater effect than placebo
N ENG LJ
M E D 3 7 2 ;1 3
N E J M .O R G
M ARCH
26, 2015
on that measure. At the time of the agency’s decision not to ap prove pirfenidone, outside experts, including many members of the Pulmonary-Allergy Drugs Advi sory Committee convened in May 2010, supported that decision.1’2 Before resubmitting its mar keting application in 2014, the sponsor conducted a new 52-week study in which the primary effi cacy end point — a decline in FVC from baseline to week 52 that was significantly smaller than that in the placebo group — was met. With two studies demonstrating a statistically sig nificant effect on FVC and pro viding supportive evidence regard-
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