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Ernst R Berndt - One of the best experts on this subject based on the ideXlab platform.
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the generic Drug User fee amendments an economic perspective
Journal of Leukocyte Biology, 2018Co-Authors: Ernst R Berndt, Rena M Conti, Stephen J MurphyAbstract:Since the vast majority of prescription Drugs consumed by Americans are off patent ('generic'), their regulation and supply is of wide interest. We describe events leading up to the US Congress's 2012 passage of the Generic Drug User Fee Amendments (GDUFA I) as part of the Food and Drug Administration Safety and Innovation Act (FDASIA). Under GDUFA I, generic manufacturers agreed to pay approximately $300 million in fees each year of the five-year program. In exchange, the US Food and Drug Administration (FDA) committed to performance goals. We describe GDUFA I's FDA commitments, provisions, goals, and annual fee structure and compare it to that entailed in the authorization and implementation of GDUFA II on October 1, 2017. We explain how User fees required under GDUFA I erected barriers to entry and created scale and scope economies for incumbent manufacturers. Congress changed User fees under GDUFA II in part to lessen these incentives. In order to initiate and sustain User fees under GDUFA legislation, FDA requires the submission of self-reported data on generic manufacturers including domestic and foreign facilities. These data are public and our examination of them provides an unprecedented window into the recent organization of generic Drug manufacturers supplying the US market. Our results suggest that generic Drug manufacturing is increasingly concentrated and foreign. We discuss the implications of this observed market structure for GDUFA II's implementation among other outcomes.
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the generic Drug User fee amendments an economic perspective
National Bureau of Economic Research, 2017Co-Authors: Ernst R Berndt, Rena M Conti, Stephen J MurphyAbstract:Regulation can influence the structure, conduct and performance of consumer product markets and the structure of product markets can influence regulation. Since the vast majority of prescription Drugs consumed by Americans are generic, the structure of the U.S. generic prescription Drug market is of wide interest. The supply of prescription Drugs in the U.S. is also heavily regulated by the U.S. Food and Drug Administration (FDA). We describe events leading up to the passage and implementation of the Generic Drug User Fee Amendments in 2012 (GDUFA I), and compare its FDA commitments, provisions, goals and fee structure to that of the 1992 Prescription Drug User Fee Act (PDUFA) for branded Drugs. Although GDUFA I expires September 30, 2017, reauthorization for GDUFA II is currently underway and is likely to shift the User fee structure away from annual facility fees to annual program fees. We explain how the fee structure of GDUFA I, and that being considered for GDUFA II, erects barriers to entry and creates scale and scope economies for incumbent manufacturers of generic Drugs. Furthermore, in order to implement fees under GDUFA I, FDA required the submission of self-reported data on generic manufacturing practices including domestic and foreign active pharmaceutical ingredient (API) and finished dosage form (FDF) facilities. These data provide an unprecedented window into the recent evolution of generic Drug manufacturing markets. Our analyses of these data suggest that generic Drug manufacturing in 2017 is quite concentrated: a very large portion of ANDA holders have small portfolios consisting of less than five ANDAs, while a small number of very large ANDA holders have portfolios consisting of hundreds or even thousands of ANDAs. The number of API and FDF facilities have each declined by approximately 10-11% between 2013 and 2017. Furthermore, in 2017, generic manufacturing is largely foreign and has become increasingly so since 2013. We discuss the implications of the current structure of the U.S. generic prescription Drug market for GDUFA II ratification and implementation.
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cost benefit analysis of the fda the case of the prescription Drug User fee acts
Journal of Public Economics, 2008Co-Authors: Ernst R Berndt, Tomas Philipson, Adrian H B Gottschalk, Eric C SunAbstract:Abstract The U.S. Food and Drug Administration (FDA) is estimated to regulate markets accounting for about 20% of consumer spending in the U.S. Despite the FDA's strict adherence to evidence-based evaluation of the safety and efficacy of the products it regulates, there exists no generally agreed upon evidence-based methodology to evaluate the agency's own safety and efficacy record. This paper proposes a methodology to evaluate FDA policies in general, and the central speed-safety tradeoff it faces, in particular. We apply this methodology to estimate the welfare effects of a major piece of legislation affecting this tradeoff, the Prescription Drug User Fee Acts (PDUFA). These acts mandated FDA performance goals in reviewing and acting on Drug applications within specified time periods, in return for levying fees on Drug manufacturers' submissions. Our methodology uses data on the U.S. sales of Drugs as well as the FDA review and withdrawal times for those Drugs to estimate measures of the private and social surplus associated with the agency in general, and changes in the speed-safety tradeoff induced by PDUFA, in particular. We find that PDUFA raised the private surplus of producers, and thus innovative returns, by about $7 to $11 billion. Depending on assumptions about the market power of producers during patent protection, we find that PDUFA raised consumer welfare between $7 and $20 billion; thus the combined social surplus was raised by $14 to $31 billion. Converting these economic gains into equivalent health benefits, we find that the more rapid access of Drugs on the market enabled by PDUFA saved the equivalent of 140,000 to 310,000 life years. Additionally, we estimate an upper bound on the adverse effects of PDUFA based on Drugs submitted during PDUFA I/II and subsequently withdrawn for safety reasons, and find that an extreme upper bound of about 56,000 life years were lost. This estimate is an extreme upper bound as it assumes all withdrawals since the inception of PDUFA were due to PDUFA and that there were no patients who benefitted from the withdrawn Drugs. We discuss how our general methodology could be used to perform a quantitative and evidence-based evaluation of the desirability of other FDA policies in the future, particularly those affecting the speed-safety tradeoff of the agency.
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assessing the safety and efficacy of the fda the case of the prescription Drug User fee acts
National Bureau of Economic Research, 2005Co-Authors: Tomas Philipson, Ernst R Berndt, Adrian H B Gottschalk, Matthew W StrobeckAbstract:The US Food and Drug Administration (FDA) is estimated to regulate markets accounting for about 20% of consumer spending in the US. This paper proposes a general methodology to evaluate FDA policies, in general, and the central speed-safety tradeoff it faces, in particular. We apply this methodology to estimate the welfare effects of a major piece of legislation affecting this tradeoff, the Prescription Drug User Fee Acts (PDUFA). We find that PDUFA raised the private surplus of producers, and thus innovative returns, by about $11 to $13 billion. Dependent on the market power assumed of producers while having patent protection, we find that PDUFA raised consumer welfare between $5 to$19 billion; thus the combined social surplus was raised between $18 to $31 billions. Converting these economic gains into equivalent health benefits, we find that the more rapid access of Drugs on the market enabled by PDUFA saved the equivalent of 180 to 310 thousand life-years. Additionally, we estimate an upper bound on the adverse effects of PDUFA based on Drugs submitted during PDUFA I/II and subsequently withdrawn for safety reasons, and find that an extreme upper bound of about 56 thousand life-years were lost. We discuss how our general methodology could be used to perform a quantitative and evidence-based evaluation of the desirability of other FDA policies in the future, particularly those affecting the speed-safety tradeoff.
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assessing the impacts of the prescription Drug User fee acts pdufa on the fda approval process
Forum for Health Economics & Policy, 2005Co-Authors: Ernst R Berndt, Tomas Philipson, Adrian H B Gottschalk, Matthew W StrobeckAbstract:Congress enacted and renewed the Prescription Drug User Fee Acts (PDUFA) in 1992, and renewed it in 1997 and 2002, mandating FDA performance goals in reviewing and acting on Drug applications within specified time periods. In turn, the FDA was permitted to levy User fees on Drug sponsors submitting applications to the FDA. While PDUFA mandated action or review times, its ultimate impacts on actual final Drug approval times are unknown. We model and quantify the impact of PDUFA-I and II on Drug approval times, since these approval dates are the ones most directly related to new medicines becoming available to benefit patients.In assessing the impacts of PDUFA on Drug approval times, it is noteworthy that approval times were trending downwards at 1.7% percent per year prior to implementation of PDUFA. Assuming continuation of that time trend, approval times post-PDUFA would have fallen even in the absence of PDUFA. Our principal finding is that PDUFA accelerated this downward trend so that instead of a counterfactual 6% reduction in approval times from 24.2 to 20.4 months in absence of these acts between 1991 and 2002, there was an observed decline of about 42%, from 24.2 to 14.2 months, following implementation of PDUFA. Thus, of the total observed decline in approval times between 1991 and 2002, approximately two-thirds can be attributed to PDUFA. However, much of this impact occurred in the initial years between 1992 and 1997 (PDUFA-I) rather than during the subsequent 1997-2002 time frame (PDUFA-II). We discuss implications of these findings and how future research might quantify the social value of the observed acceleration in the FDA Drug approvals.
Tomas Philipson - One of the best experts on this subject based on the ideXlab platform.
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cost benefit analysis of the fda the case of the prescription Drug User fee acts
Journal of Public Economics, 2008Co-Authors: Ernst R Berndt, Tomas Philipson, Adrian H B Gottschalk, Eric C SunAbstract:Abstract The U.S. Food and Drug Administration (FDA) is estimated to regulate markets accounting for about 20% of consumer spending in the U.S. Despite the FDA's strict adherence to evidence-based evaluation of the safety and efficacy of the products it regulates, there exists no generally agreed upon evidence-based methodology to evaluate the agency's own safety and efficacy record. This paper proposes a methodology to evaluate FDA policies in general, and the central speed-safety tradeoff it faces, in particular. We apply this methodology to estimate the welfare effects of a major piece of legislation affecting this tradeoff, the Prescription Drug User Fee Acts (PDUFA). These acts mandated FDA performance goals in reviewing and acting on Drug applications within specified time periods, in return for levying fees on Drug manufacturers' submissions. Our methodology uses data on the U.S. sales of Drugs as well as the FDA review and withdrawal times for those Drugs to estimate measures of the private and social surplus associated with the agency in general, and changes in the speed-safety tradeoff induced by PDUFA, in particular. We find that PDUFA raised the private surplus of producers, and thus innovative returns, by about $7 to $11 billion. Depending on assumptions about the market power of producers during patent protection, we find that PDUFA raised consumer welfare between $7 and $20 billion; thus the combined social surplus was raised by $14 to $31 billion. Converting these economic gains into equivalent health benefits, we find that the more rapid access of Drugs on the market enabled by PDUFA saved the equivalent of 140,000 to 310,000 life years. Additionally, we estimate an upper bound on the adverse effects of PDUFA based on Drugs submitted during PDUFA I/II and subsequently withdrawn for safety reasons, and find that an extreme upper bound of about 56,000 life years were lost. This estimate is an extreme upper bound as it assumes all withdrawals since the inception of PDUFA were due to PDUFA and that there were no patients who benefitted from the withdrawn Drugs. We discuss how our general methodology could be used to perform a quantitative and evidence-based evaluation of the desirability of other FDA policies in the future, particularly those affecting the speed-safety tradeoff of the agency.
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assessing the safety and efficacy of the fda the case of the prescription Drug User fee acts
National Bureau of Economic Research, 2005Co-Authors: Tomas Philipson, Ernst R Berndt, Adrian H B Gottschalk, Matthew W StrobeckAbstract:The US Food and Drug Administration (FDA) is estimated to regulate markets accounting for about 20% of consumer spending in the US. This paper proposes a general methodology to evaluate FDA policies, in general, and the central speed-safety tradeoff it faces, in particular. We apply this methodology to estimate the welfare effects of a major piece of legislation affecting this tradeoff, the Prescription Drug User Fee Acts (PDUFA). We find that PDUFA raised the private surplus of producers, and thus innovative returns, by about $11 to $13 billion. Dependent on the market power assumed of producers while having patent protection, we find that PDUFA raised consumer welfare between $5 to$19 billion; thus the combined social surplus was raised between $18 to $31 billions. Converting these economic gains into equivalent health benefits, we find that the more rapid access of Drugs on the market enabled by PDUFA saved the equivalent of 180 to 310 thousand life-years. Additionally, we estimate an upper bound on the adverse effects of PDUFA based on Drugs submitted during PDUFA I/II and subsequently withdrawn for safety reasons, and find that an extreme upper bound of about 56 thousand life-years were lost. We discuss how our general methodology could be used to perform a quantitative and evidence-based evaluation of the desirability of other FDA policies in the future, particularly those affecting the speed-safety tradeoff.
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assessing the impacts of the prescription Drug User fee acts pdufa on the fda approval process
Forum for Health Economics & Policy, 2005Co-Authors: Ernst R Berndt, Tomas Philipson, Adrian H B Gottschalk, Matthew W StrobeckAbstract:Congress enacted and renewed the Prescription Drug User Fee Acts (PDUFA) in 1992, and renewed it in 1997 and 2002, mandating FDA performance goals in reviewing and acting on Drug applications within specified time periods. In turn, the FDA was permitted to levy User fees on Drug sponsors submitting applications to the FDA. While PDUFA mandated action or review times, its ultimate impacts on actual final Drug approval times are unknown. We model and quantify the impact of PDUFA-I and II on Drug approval times, since these approval dates are the ones most directly related to new medicines becoming available to benefit patients.In assessing the impacts of PDUFA on Drug approval times, it is noteworthy that approval times were trending downwards at 1.7% percent per year prior to implementation of PDUFA. Assuming continuation of that time trend, approval times post-PDUFA would have fallen even in the absence of PDUFA. Our principal finding is that PDUFA accelerated this downward trend so that instead of a counterfactual 6% reduction in approval times from 24.2 to 20.4 months in absence of these acts between 1991 and 2002, there was an observed decline of about 42%, from 24.2 to 14.2 months, following implementation of PDUFA. Thus, of the total observed decline in approval times between 1991 and 2002, approximately two-thirds can be attributed to PDUFA. However, much of this impact occurred in the initial years between 1992 and 1997 (PDUFA-I) rather than during the subsequent 1997-2002 time frame (PDUFA-II). We discuss implications of these findings and how future research might quantify the social value of the observed acceleration in the FDA Drug approvals.
Stephen J Murphy - One of the best experts on this subject based on the ideXlab platform.
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the generic Drug User fee amendments an economic perspective
Journal of Leukocyte Biology, 2018Co-Authors: Ernst R Berndt, Rena M Conti, Stephen J MurphyAbstract:Since the vast majority of prescription Drugs consumed by Americans are off patent ('generic'), their regulation and supply is of wide interest. We describe events leading up to the US Congress's 2012 passage of the Generic Drug User Fee Amendments (GDUFA I) as part of the Food and Drug Administration Safety and Innovation Act (FDASIA). Under GDUFA I, generic manufacturers agreed to pay approximately $300 million in fees each year of the five-year program. In exchange, the US Food and Drug Administration (FDA) committed to performance goals. We describe GDUFA I's FDA commitments, provisions, goals, and annual fee structure and compare it to that entailed in the authorization and implementation of GDUFA II on October 1, 2017. We explain how User fees required under GDUFA I erected barriers to entry and created scale and scope economies for incumbent manufacturers. Congress changed User fees under GDUFA II in part to lessen these incentives. In order to initiate and sustain User fees under GDUFA legislation, FDA requires the submission of self-reported data on generic manufacturers including domestic and foreign facilities. These data are public and our examination of them provides an unprecedented window into the recent organization of generic Drug manufacturers supplying the US market. Our results suggest that generic Drug manufacturing is increasingly concentrated and foreign. We discuss the implications of this observed market structure for GDUFA II's implementation among other outcomes.
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the generic Drug User fee amendments an economic perspective
National Bureau of Economic Research, 2017Co-Authors: Ernst R Berndt, Rena M Conti, Stephen J MurphyAbstract:Regulation can influence the structure, conduct and performance of consumer product markets and the structure of product markets can influence regulation. Since the vast majority of prescription Drugs consumed by Americans are generic, the structure of the U.S. generic prescription Drug market is of wide interest. The supply of prescription Drugs in the U.S. is also heavily regulated by the U.S. Food and Drug Administration (FDA). We describe events leading up to the passage and implementation of the Generic Drug User Fee Amendments in 2012 (GDUFA I), and compare its FDA commitments, provisions, goals and fee structure to that of the 1992 Prescription Drug User Fee Act (PDUFA) for branded Drugs. Although GDUFA I expires September 30, 2017, reauthorization for GDUFA II is currently underway and is likely to shift the User fee structure away from annual facility fees to annual program fees. We explain how the fee structure of GDUFA I, and that being considered for GDUFA II, erects barriers to entry and creates scale and scope economies for incumbent manufacturers of generic Drugs. Furthermore, in order to implement fees under GDUFA I, FDA required the submission of self-reported data on generic manufacturing practices including domestic and foreign active pharmaceutical ingredient (API) and finished dosage form (FDF) facilities. These data provide an unprecedented window into the recent evolution of generic Drug manufacturing markets. Our analyses of these data suggest that generic Drug manufacturing in 2017 is quite concentrated: a very large portion of ANDA holders have small portfolios consisting of less than five ANDAs, while a small number of very large ANDA holders have portfolios consisting of hundreds or even thousands of ANDAs. The number of API and FDF facilities have each declined by approximately 10-11% between 2013 and 2017. Furthermore, in 2017, generic manufacturing is largely foreign and has become increasingly so since 2013. We discuss the implications of the current structure of the U.S. generic prescription Drug market for GDUFA II ratification and implementation.
Ke Dong - One of the best experts on this subject based on the ideXlab platform.
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economic impacts of the generic Drug User fee act fee structure
Value in Health, 2017Co-Authors: Ke Dong, Garth Boehm, Qiang ZhengAbstract:Abstract Background A Food and Drug Administration (FDA) Generic Drug User system, Generic Drug User Fee Amendment of 2012 (GDUFA), started October 1, 2012, and has been in place for over 3 years. There is controversy about the GDUFA fee structure but no analysis of GDUFA data that we could find. Objective To look at the economic impact of the GDUFA fee structure. Methods We compared the structure of GDUFA with that of other FDA Human Drug User fees. We then, using FDA-published information, analyzed where GDUFA facility and Drug Master File fees are coming from. We used the Orange Book to identify the sponsors of all approved Abbreviated New Drug Applications (ANDAs) and the S&P Capital IQ database to find the ultimate parent companies of sponsors of approved ANDAs. Results The key differences between the previous structure for Human Drug User fees and the GDUFA are as follows: GDUFA has no approved product fee and no first-time or small business fee exemptions and GDUFA charges facility fees from the time of filing and charges a foreign facility levy. Most GDUFA fees are paid by or on behalf of foreign entities. The top 10 companies hold nearly 50% of all approved ANDAs but pay about 14% of GDUFA facility fees. Conclusions We conclude that the regressive nature of the GDUFA fee structure penalizes small, new, and foreign firms while benefiting the large established firms. A progressive fee structure in line with other human Drug User fees is needed to ensure a healthy generic Drug industry.
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Economic Impacts of the Generic Drug User Fee Act Fee Structure
VALUE IN HEALTH, 2017Co-Authors: Ke Dong, Boehm Garth, Zheng QiangAbstract:Background: A Food and Drug Administration (FDA) Generic Drug User system, Generic Drug User Fee Amendment of 2012 (GDUFA), started October 1, 2012, and has been in place for over 3 years. There is controversy about the GDUFA fee structure but no analysis of GDUFA data that we could find. Objective: To look at the economic impact of the GDUFA fee structure. Methods: We compared the structure of GDUFA with that of other FDA Human Drug User fees. We then, using FDA -published information, analyzed where GDUFA facility and Drug Master File fees are coming from. We used the Orange Book to identify the sponsors of all approved Abbreviated New Drug Applications (ANDAs) and the S&P Capital IQ database to find the ultimate parent companies of sponsors of approved ANDAs. Results: The key differences between the previous structure for Human Drug User fees and the GDUFA are as follows: GDUFA has no approved product fee and no first-time or small business fee exemptions and GDUFA charges facility fees from the time of filing and charges a foreign facility levy. Most GDUFA fees are paid by or on behalf of foreign entities. The top 10 companies hold nearly 50% of all approved ANDAs but pay about 14% of GDUFA facility fees. Conclusions: We conclude that the regressive nature of the GDUFA fee structure penalizes small, new, and foreign firms while benefiting the large established firms. A progressive fee structure in line with other human Drug User fees is needed to ensure a healthy generic Drug industry.SCI(E)SSCIARTICLE6792-7982
Robert Lionberger - One of the best experts on this subject based on the ideXlab platform.
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Innovation for Generic Drugs: Science and Research Under the Generic Drug User Fee Amendments of 2012
Clinical pharmacology and therapeutics, 2019Co-Authors: Robert LionbergerAbstract:Regulatory science is science and research intended to improve decision making in a regulatory framework. Improvements in decision making can be in both accuracy (making better decisions) and in efficiency (making faster decisions). Science and research supported by the Generic Drug User Fee Amendments of 2012 (GDUFA) have focused on two innovative methodologies that work together to enable new approaches to development and review of generic Drugs: quantitative models and advanced in vitro product characterization. Quantitative models faithfully represent current scientific understanding. They are tools pharmaceutical scientists and clinical pharmacologists use for making better and faster product development decisions. Advances in the in vitro product comparisons provide the measurements of product differences that are the critical input into the models. This paper outlines four areas where science and research funded by GDUFA support synergistic use of models and characterization at critical decision points during generic Drug product development and review.
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completeness assessment of type ii active pharmaceutical ingredient Drug master files under generic Drug User fee amendment review metrics and common incomplete items
Aaps Journal, 2014Co-Authors: Huyi Zhang, Robert Lionberger, Wei Song, Diandian Shen, David Skanchy, Kun Shen, Susan RosencranceAbstract:Under the Generic Drug User Fee Amendments (GDUFA) of 2012, Type II active pharmaceutical ingredient (API) Drug master files (DMFs) must pay a User fee and pass a Completeness Assessment (CA) before they can be referenced in an Abbreviated New Drug Application (ANDA), ANDA amendment, or ANDA prior approval supplement (PAS). During the first year of GDUFA implementation, from October 1, 2012 to September 30, 2013, approximately 1,500 Type II API DMFs received at least one cycle of CA review and more than 1,100 Type II DMFs were deemed complete and published on FDA’s “Available for Reference List”. The data from CA reviews were analyzed for factors that influenced the CA review process and metrics, as well as the areas of DMF submissions which most frequently led to an incomplete CA status. The metrics analysis revealed that electronic DMFs appear to improve the completeness of submission and shorten both the review and response times. Utilizing the CA checklist to compile and proactively update the DMFs improves the chance for the DMFs to pass the CA in the first cycle. However, given that the majority of DMFs require at least two cycles of CA before being deemed complete, it is recommended that DMF fees are paid 6 months in advance of the ANDA submissions in order to avoid negatively impacting the filling status of the ANDAs.
