1、Patient Access Report2 2Table of ContentsExecutive Summary.4Introduction and Briefing Document Objective.7Measuring Biopharmaceutical Market Access Barriers.10Fewer Innovative Medicines and Longer Wait Times.16Conclusion.24Appendix.253The U.S.Chamber of Commerces Global Innovation Policy Center()is

2、working around the world to champion intellectual property rights as vital to creating jobs,saving lives,advancing global economic growth,and generating breakthrough solutions to global challenges.The U.S.Chamber of Commerce is the worlds largest business federation representing the interests of mor

3、e than 3 million businesses of all sizes,sectors,and regions,as well as state and local chambers and industry associations.This report was conducted by Pugatch Consilum,(www.pugatch-)a boutique consultancy that provides evidence-based research,analysis,and intelligence on the fastest growing sectors

4、 of the knowledge economy.Authors of this report are Meir Pugatch and David Torstensson.Professor Meir Pugatch,Managing Director and Founder|Prof.Pugatch is the Managing Director of Pugatch Consilium a boutique consultancy that provides evidence-based research,analysis and intelligence on the fastes

5、t growing sectors of the knowledge economy.He is an IPKM Professor of Valorisation,Entrepreneurship and Management at the University of Maastricht in the Netherlands;as well as a Professor at the School of Public Health,University of Haifa in Israel,in which he acts as the Chair of the Health Manage

6、ment Division since 2019.Prof.Pugatch specializes in innovation strategies,organizational entrepreneurship,intellectual property management,pharmacoeconomics,pricing and reimbursement,and the management of public health systems.He is the author and editor of an extensive number of publications and s

7、erves as a referee and editorial board member of numerous peer review journals.David Torstensson,Partner|Dr.Torstensson specializes in innovation,tax and intellectual property policy,with a particular focus on the health care,information and communication technology and content industries.He has wid

8、e experience in policy and economic analysis,as well as data sampling and creation of strategic operational and advocacy plans.He is the author of a number of academic and commissioned reports and publications and is the co-author of all ten editions of the U.S.Chamber International IP Index.4Execut

9、ive SummaryCountries with the right public policy frameworks in place can create an ecosystem where innovation can thrive.Free market frameworks,underpinned by effective intellectual property protection,have fostered life sciences innovation in key global markets.For that innovation to continue,coun

10、tries must continue to allow marketplace competition to support the development of lifesaving treatments and cures.The imposition of price controls creates a fundamental market access barrier that deters future innovation.Research published by the Chamber in 2019 highlighted how a countrys legal and

11、 regulatory framework can hinder the creation of and access to innovative goods and services in key global markets.Legal and regulatory frameworks have changed in many countries since the onset of the COVID-19 pandemic.The 2023 Patient Access Report(Report)examines the national biopharmaceutical mar

12、ket for nine Organisation for Economic Co-operation and Development(OECD)economies and assesses the impact of their policies on access to innovative medicines.The updated report illustrates how countries that impose price controls have less access to lifesaving treatments and cures.The overall score

13、s for the 2023 Report are include in Figure 1 below.U.S.UKGermanyFranceItalyCanadaAustraliaJapanSouth Korea80%100%60%40%20%0%94.95%63.94%63.94%63.94%57.69%51.59%47.95%35.00%34.55%Figure 1:Patient Access to the Latest Medicines:(Pricing,Reimbursement,and Access Regulation to a National Biopharmaceuti

14、cal Market,2022)5Key FindingsStrict price controls are widely used in most OECD economies:Before the passage of the Inflation Reduction Act,the U.S.was the sole economy included in the Report that did not impose direct national price controls or other policies adopted in the name of biopharmaceutica

15、l cost containment.Consequently,the U.S.achieves an overall score of 94.95%,as indicated by Figure 1 above.However,following the implementation of the IRA,the U.S.score will fall dramatically and be more in line with OECD economies where the government intervenes to set the price of medicines.Countr

16、ies that use price controls see the following:Fewer overall biopharmaceutical product launches:Canada,Japan,South Korea,Australia,and European Union(EU)member states have seen significantly fewer overall biopharmaceutical product launches than the United States has over the past 20 years.Fewer biolo

17、gics:Countries with the most severe price controls in place,including South Korea and Australia,have seen fewer than half of new biologics launched in the same period.Only 49%of new biologics launched in the U.S.over the last 20 years were available in South Korea,while only 38%of those biologics we

18、re launched in Australia.Fewer oncology products:Out of 104 new oncology products launched globally since 2017,80%were launched in the U.S.,but only 56%were launched in Europe.Delayed access to treatments:The long lag time between market authorization and inclusion for government reimbursement delay

19、s access to the newest innovative medicines.In Germany,patients wait an average of 133 days to access new treatments;in Spain,the delay is as long as 500 days.56The price controls included in the Inflation Reduction Act will have a detrimental impact on biopharmaceutical innovation.Government interv

20、ention in price setting undermines the innovation ecosystem that empowered the U.S.to become one of the most innovative countries in the world.The price control provisions could lead to fewer new products and medicines developed and introduced in the United States.Government officials must carefully

21、 consider the implications of price controls for patients,as well as litigation risk and other practical considerations,before proceeding with the implementation of a legally problematic framework that would jeopardize U.S.leadership on biopharmaceutical innovation and patient access to treatments.7

22、Introduction and Briefing Document ObjectiveFrom a Free Market to Command and Control?Introduction of Biopharmaceutical Price Controls in the United StatesHistorically,the provision of health care in the United States has been based on a health insurance model principally managed via private funding

23、 and private delivery.Health care facilitiesincluding hospitals and clinicsare mainly privately owned and operated.The elderly,those with low income,military personnel,and public sector employees are eligible for public coverage of health care(either full or subsidized)via Medicare,Medicaid,and othe

24、r programs that are run by the federal and state governments.Similarly,the biopharmaceutical market has been predominantly market based.Private payers,including insurers,managed care organizations,and pharmaceutical benefit managers,aggregate various health plans and purchase biopharmaceuticals on b

25、ehalf of their members.Private payers often employ formularies,differential cost sharing(including tiered copayments),and other methods to influence prescribing practices.In doing so,they can negotiate discounted prices from biopharmaceutical manufacturers and pharmacies.Individual hospitals and oth

26、er health care institutions are also increasingly using formularies to manage costs.However,unlike many other high-income OECD economies,the U.S.federal government has not imposed national price controls or other restrictions and market access barriers on health technologies,including biopharmaceuti

27、cals and medical devices.This may now be changing.The Trump administration introduced several reform initiatives aimed at lowering the cost of prescription medicines.In February 2018 the Council of Economic Advisers released Reforming Biopharmaceutical Pricing at Home and Abroad,an analysis of the g

28、lobal biopharmaceutical market.A few months later the Department of Health and Human Services also announced a set of reforms in the blueprint document American Patients First.In late 2018 the administration announced a plan to build an International Pricing Index and to develop a most-favored-natio

29、n(MFN)model to be used by Medicare Part B.This plan was formalized in late 2020 by the Department of Health and Human Services and Centers for Medicare and Medicaid Services(CMS).The MFN model would benchmark the price of a basket of 50 biopharmaceutical products with 8the price for the same product

30、s in a sample of OECD economies.1 The comparator economies were chosen based on OECD membership and per capita gross domestic product(GDP),at purchasing power parity(PPP),of 60%or above that of the United States.After several court rulings in late 2021,the CMS formally rescinded the proposed MFN mod

31、el.Similar proposals for controlling expenditure on prescription drugs through price controls have been introduced in Congress.In 2019 and 2021,members of Congress introduced H.R.3Elijah E.Cummings Lower Drug Costs Now Act.Similar to the CMS MFN model,the bill introduced an international reference p

32、ricing framework whereby the price of medicines in the U.S.would be determined based on the average price of a basket of products in a selection of high-income OECD economies.In 2022,Congress passed the Inflation Reduction Act of 2022.2 The legislation included a series of fundamental changes to the

33、 pricing framework for medicines covered under Medicare Part B and Part D in the United States.Among other things,the legislation(1)empowers the Secretary of Health and Human Services(HHS)to use what the law refers to as a“negotiation”process to set the price of an expanding number of medicines that

34、 are covered under Medicare;and(2)requires manufacturers to pay the federal government a“rebate”if they raise their prices for a large number of medicines covered by Medicare at a rate that is higher than the rate of inflation.9Briefing Document ObjectiveThe imposition of price controlsincluding int

35、ernational reference pricingconstitutes a fundamental market access barrier and deters future innovation.Such policies have historically had a direct and negative impact on the availability of innovative drugs and medical technologies for patients and consumers in the affected market.Simply put,econ

36、omies that impose price controls see fewer innovative products on the market,and patients are less likely to be able to access the latest drugs and medical technologies.This fact is not sufficiently recognized or considered in any of the previously described reform proposals in the United States.Bio

37、pharmaceutical innovation does not take place in a vacuum.Like all high-tech industries,the life sciences sector relies on an ecosystem and set of enabling conditions that encourage research and development(R&D)and the development of new products.Human capital,adequate R&D infrastructure,strong and

38、targeted intellectual property(IP)protection,transparent and effective regulations and administration,a technology transfer framework that encourages innovation and the translation of R&D into actual products and full commercialization,a predictable legal environment,and a stable commercial environm

39、ent are all key factors and enablers of biopharmaceutical innovation.The purpose of this briefing document is twofold:1.Empirically measure and quantify the biopharmaceutical market access environment as currently constituted in the United States compared with a selection of high-income OECD economi

40、es.2.Show the real-world negative consequences of imposing price controls on patient access to biopharmaceutical innovation and technologies.10Measuring Biopharmaceutical Market Access BarriersFirst launched in 2019,the Patient Access Report(Report)is a unique social scientific tool dedicated to mea

41、suring the extent to which products,innovations,and creative works are allowed to gain access to their intended markets.The Report seeks to translate information tracked by international institutions(such as the World Trade Organization WTO and OECD)and large trading partners into a quantifiable eco

42、nomy-to-economy comparison and index measuring real-world market access environment for knowledge-intensive and intellectual propertybased products and services.3 The Report consists of 16 individual indicators across four separate categories.The 2019 edition of the Report covered 20 economies:the 1

43、9 individual economies of the Group of Twenty(G20)and Algeria.One-quarter of the 2019 ReportCategory 4:Pricing,reimbursement,and access regulation to a national biopharmaceutical marketis dedicated to measuring the extent to which national biopharmaceutical regulations,including pricing and reimburs

44、ement(P&R)policies,limit access to a domestic market and provide preferences for local producers.4Rebenchmarking the Report in a Post-COVID-19 WorldSince the results of the Report were published in 2019,the world has fundamentally changed.The COVID-19 pandemic reshaped how many governments and polic

45、ymakers view public health and access to medical technology,including biopharmaceuticals.The pandemic illustrated the immense and direct value of the research-based biopharmaceutical industry to global health.Developing new medicines is a long-term,high-risk,resource-intensive process.The fixed cost

46、s in terms of laboratory,research facilities,and researchers are immense.In 1979,the total cost of developing and approving a new drug stood at USD138 million.Almost 25 years later,in 2003,this figure was estimated at USD802 million.5 More recent research from Tufts University suggests that it costs

47、 USD2.6 billion,on average,to develop a new drug.6 On average,only one to two of 11every 10,000 synthesized,examined,and screened compounds in basic research will pass through all stages of R&D and go on to become a marketable drug.Developing a new medicine can take a decade or more.Up until the COV

48、ID-19 outbreak,this timeline was the norm for vaccine development and the biopharmaceutical R&D process in general.Within this historical context the speed with which COVID-19 vaccines and treatments have been developed over the last two years has been truly breathtaking,and it is impossible to over

49、state the enormity of these accomplishments.It shows the extensive scientific capacity developed by the biopharmaceutical industry and the biotech community and the ability to scale up quickly and decisively to understand and develop a treatment for a novel virus that was not prevalent in human bein

50、gs 24 months earlier.At the scientific,manufacturing,distribution,and organizational levels,what the industry together with its partners in academia and the public sector has been able to achieve amounts to a modern-day miracle.Yet the science and technological capacity that allowed industry,public

51、research organizations,and academic researchers to carry out this development are based on decades of investment in R&D experience and innovation.Just as for other forms of technological innovation,it is highly unlikely that this biopharmaceutical R&D would have taken place without the right enablin

52、g environment in place.These technologies and products are the fruits of a preexisting innovation ecosystem that is centered on IP rights,which provide innovators with an opportunity to earn a return on investment and establish a vehicle for knowledge-related commercial transactions.Without strong a

53、nd clear IP rights and the economic functionality they provide,it is unlikely that any of those products and technologiesor the underlying sciencethat have been so essential in keeping societies functioning and fighting the COVID-19 pandemic would exist today.The IP-enabled technologies used to figh

54、t COVID-19 will also further the development of the next generation of breakthrough treatments and cures.While a patent entitles the innovator to a 20-year term of protection,upon expiration,the knowledge included in the patent becomes available for anyone in the public to use to further their own i

55、nnovation.In this way,patents further the public consumption of knowledge and ensure that future innovators can benefits from the research of their predecessors.Given this new global context and the looming introduction of national biopharmaceutical price controls in the United States,the U.S.Chambe

56、r of Commerce has rebenchmarked nine of the original 20 economies against the four indicators included in Category 4:pricing,reimbursement,and access regulation to a national biopharmaceutical market.The results of an economys score on these four indicators comprise their overall score in the Report

57、.The nine economies benchmarkedAustralia,Canada,France,Germany,Italy,Japan,South Korea,the UK,and the U.S.play an outsized role in determining the viability of the economic environment for new drug development.The updated research makes clear for politicians,policymakers,and stakeholders the differe

58、nces in how these economies have regulated access to their biopharmaceutical markets through national pricing,reimbursement,and procurement policies,as well as the direct and highly negative consequences for medical innovation and patient access to new products and technologies associated with many

59、of these approaches.12Report Results and Key TakeawaysFigure 1 shows the results of the re-benchmarking of the four indicators included in Category 4:pricing,reimbursement,and access regulation to a national biopharmaceutical market for the original nine high-income OECD Members included in the 2019

60、 Report:Australia,Canada,France,Germany,Italy,Japan,South Korea,the UK,and the U.S.Figure 1:Overall Scores,Category 4:Pricing,Reimbursement,and Access Regulation to a National Biopharmaceutical MarketU.S.UKGermanyFranceItalyCanadaAustraliaJapanSouth Korea80%100%60%40%20%0%94.95%63.94%63.94%63.94%57.

61、69%51.59%47.95%35.00%34.55%As Figure 1 shows,strict price controls are in place and widely used in most OECD economies.In Europe,France,Italy,Germany,and the UK all impose direct or indirect controls on the price of biopharmaceuticals.Access to national health systems is also restricted by reimburse

62、ment limits,health technology and cost effectiveness assessments,and reference pricing.Similarly,Australia,Canada,and Korea all impose harsh price controls.Until 2022,the U.S.was the sole economy included in the Report that did not impose direct national price controls or other policies adopted in t

63、he name of biopharmaceutical cost containment.Consequently,the U.S.achieves an overall score of 94.95%.This is more than 30 percentage points higher than France,Germany,and UK;all tied at 63.94%.Notably,almost half the sampled economiesCanada,Australia,Japan,and South Koreaachieve a score of around

64、1350%or less;Canada scored the highest of those four economies at 51.59%.However,following the implementation of the IRA,the U.S.score will decrease significantly as a result of the new price control provisions.As a result,U.S.consumers will face less choice of new medicines,similar to consumers in

65、other OECD economies.Review of the four individual indicators that together constitute Category 4:pricing,reimbursement,and access regulation to a national biopharmaceutical market echoes these broader findings.Table 1 shows the individual scores for the economies sampled for each of the four underl

66、ying indicators that together make up the category.Table 1:Individual Indicator Scores,Category 4:Pricing,Reimbursement,and Access Regulation to a National Biopharmaceutical Market7Overarching philosophy and direction of health and biopharmaceutical system:cost cutting versus recognizing and incorpo

67、rating new products and biopharmaceutical innovationPricing and reimbursement decision-making and processBiopharmaceutical-specific procurement preferences Availability of new,innovative biopharmaceutical treatments and products Australia25%25%100%42%Canada25%25%100%56%France50%50%100%56%Germany50%5

68、0%100%56%Italy50%25%100%56%Japan25%0%75%40%South Korea0%0%100%38%UK50%50%100%56%U.S.100%100%100%80%Table 1 shows how challenging the market access environment is for biopharmaceutical rightsholders as measured by the individual indicators.For example,apart from procurement preferenceswhere only Japa

69、n scores less than 100%all economies bar the United States score 50%or less on indicators measuring the pricing and reimbursement environment.South Korea and Japan stand out.Both economies score poorly on the overarching philosophy and direction of their health and biopharmaceutical systems.Similarl

70、y,the P&R process in both economies remains opaque with little in the way of meaningful opportunity and engagement with nongovernment stakeholders.Comparing the 2022 Report with the 2019 scores shows that the biopharmaceutical market access environment in many economies has deteriorated.Figure 2 com

71、pares the nine sampled economies 2022 and 2019 scores.14Figure 2:2022 Versus 2019 Results,Category 4:Pricing,Reimbursement,and Access Regulation to a National Biopharmaceutical MarketU.S.FranceGermanyUKItalyCanadaAustraliaJapanSouth Korea80%100%60%40%20%0%95.91%94.95%63.18%63.94%69.09%63.94%67.27%63

72、.94%56.48%57.69%58.09%51.59%47.95%47.95%47.50%35.00%34.55%34.55%20192022As Figure 2 shows,most of the nine sampled OECD economies have seen their scores drop over the last two years.Notably,Japan and Canadas scores decreased substantially,with decreases of 12.50%and 6.50%,respectively.In Japan,this

73、steep drop was driven by the finalization and adoption of a new biopharmaceutical cost-benefit assessment system,price preferences for locally developed products,and a lack of meaningful consultation and engagement 15with relevant stakeholders in developing these new policies.8 Specifically,a new he

74、alth technology assessment(HTA)procedure became operational and has become an integral part of the national pricing and reimbursement process.This new HTA system is primarily focused on cost as a basis for analysis.It includes rigid selection criteria and focuses almost exclusively on high-cost prod

75、ucts.Additional socioeconomic impact analysis does not form part of the assessment criteria.Similarly,changes to eligibility and evaluation criteria relating to the Price Maintenance Premium programan initiative introduced in the early 2010s aimed at rewarding innovation through additional price pre

76、miumsnow provide indirect price preferences for products developed in Japan.Both the U.S.government and the research-based biopharmaceutical industry have noted the lack of transparency and meaningful stakeholder involvement in developing these new policies.Similarly,in Canada,the biopharmaceutical

77、market access environment has deteriorated since 2019.Over the past several years,Canadian authorities have reformed how patented medicines are evaluated and priced through the Patented Medicine Prices Review Boards(PMPRB)evaluation methodology.These reform efforts have focused almost exclusively on

78、 cost and expenditure reduction.While successful legal challenges have limited the scope of some of these proposals,the changes to the basket of economies the PMPRB uses for international price comparisons have been retained and are now in effect.Specifically,the reforms have expanded the size of th

79、e basket and removed the United States and Switzerland as comparator economies.New economies added are Australia,Belgium,Japan,the Netherlands,Norway,and Spain.Given the strict price controls in these new economies and the removal of the United States and Switzerland as comparator economies,these ch

80、anges will substantially lower the overall price comparisons and thus the overall biopharmaceutical price level in Canada.These changes came into force on July 1,2022.Germany and the UK have also seen their scores drop by 5.15%and 3.33%,respectively.This drop was driven by a decrease in the availabi

81、lity of new oncology medicines in both economies compared with those of the U.S.The latest available data on the launch of new oncology products show how substantially fewer innovative medicines have been launched in Europe in the last five-year period than in previous reporting periods measured in

82、the 2019 Report.Specifically,the Global Oncology Trends 2022 reportpublished by IQVIA in May 2022shows how out of 104 new products launched globally since 2017,80%were launched in the U.S.,but only 58%were launched in Europe,including both Germany and the UK.16Fewer Innovative Medicines and Longer W

83、ait TimesThe True Cost of Price ControlsThe imposition of price controls have a direct impact on how,when,and which medicines and medical products are available to patients in a given health system.For instance,a 2018 study examining the availability of 46 new cancer medicines in four EU Member Stat

84、es(Belgium,Estonia,Scotland and Sweden)between 2000-2014 found large discrepancies with respect to both the number of products launched in each country and when products were made available within each health system.In no country were all 46 new products launched.In Estonia,for instance,almost half

85、of the sample(19 medicines out of 46)were never launched.Similarly,in all of the countries for all of the medicines launched it took,at best,more than a year for these new products to reach patients.In Sweden,which on launch time was the best performing of the four,it still took,on average,14.3 mont

86、hs from time of EU market authorization until the product was first used within the health system.In Belgium this was,on average,over two years(26.8 months)and in Estonia it was even more over five years(63.9 months).Similarly,looking at access to orphan drugs and new medicines for rare diseases,evi

87、dence suggests that there is considerable variation in levels of access across the EU.For example,a 2017 study by the Office of Health Economics(a British research institute)compared access to 143 orphan products that were approved for marketing in the EU between 2000 and 2016 across the then EU-5(i

88、ncluding a division between England,Scotland and Wales that comprises the UK).Overall,the study found that access to authorized orphan products through public reimbursement varied substantially between the sampled Member States,ranging from 93%in Germany to 33%in Wales.Similarly,the average duration

89、 between the granting of marketing authorization by the EMA and reimbursement decision by the national authority was 23.4 months.The duration was considerably longer for orphan medicines when compared to non-orphan medicines.For example,in the UK,the median number of months between the marketing aut

90、horization and the first NICE appraisal was 20.2 months for orphan medicines compared to 12.7 months for non-orphan medicines.Indeed,the historical data and evidence is very clear on this.17Several studies examining the availability of innovative drugs show a distinct disparity between(1)economies w

91、ith market access and regulatory environments that seek to strike a balance between maintaining financial stability and rewarding innovation and(2)more restrictive economies that prioritize policies adopted in the name of biopharmaceutical cost containment.For instance,a 2008 study by the National B

92、ureau of Economic Research concerning drug launches suggests that free-pricing countries historically have a larger number of launched innovative drugs than countries with a more challenging P&R environment.For the period examined(19922003),Germany9 and the U.S.had a larger number of launched drugs

93、for subclasses of innovative drugs(88 and 86,respectively),while Japan,Portugal,and France,which all had in place strict price regulations,saw fewer molecules launched(53,62,and 69 respectively).10 Similarly,statistical modeling investigating the impact of price controls on product launches in sever

94、al OECD and middle-income economies found that price controls(and other supply-side controls)have a significant impact on potential product entry,reducing the likelihood of entry by roughly 75%compared with a market having no price controls.11 A large 2010 study from the London School of Economics a

95、nd Political Science examining historical trends in launch lags of innovative drugs in 20 economies suggests that economies with more challenging regulatory and market access environments have longer delays in accessing innovative drugs.Figure 3 shows the time lags in market access for the latest pe

96、riod studied,19952008.Figure 3:Historical Trends in Launch Lags of Innovative Drugs,19952008,Select Economies12*Mean Launch Lag(in Years)for New Active Substances Launched in the U.S.&UK9.08.07.06.05.04.03.02.01.00.0JapanSpainTurkeyGreeceAustraliaCanadaSwitzerlandItalyFranceGermany7.96.35.74.13.63.4

97、3.02.92.91.4*Mean launch lag refers to the average time(in years)between first global launch(indicated by first sales in IMS database)and first sales in a given economy for 266 new molecules approved for marketing in the U.S.and UK in 19952008.18As Figure 3 shows,markets like Japan,Australia,Spain,T

98、urkey,and Greece have historically had longer lags than other markets.While these academic studies focus on the long-term historical launch and drug availability records,launch lags and differences in product availability between markets are still common today.In fact,despite globalization,global re

99、gulatory harmonization,and improved access to global biopharmaceutical supply chains,these lags in availability are,in some ways,even more pronounced today.This is particularly the case for more technically complex and innovative drugs such as biologics and cancer medicines.Just like the long-term h

100、istorical record,the most recent launch and product availability data show how economies with price controls and a more challenging biopharmaceutical market access environment consistently see substantially lower levels of product penetration and drug availability for patients.For example,evidence c

101、ollected by IQVIA and published by Life Sciences Ontario in 2020 shows that many new health technologies and medicines are never launched at all in economies with strict price controls in place.This is a critical takeaway when examining the true cost of introducing price controls:Many new products a

102、nd medical innovations never make it onto the market.As Figure 4 shows,markets like Canada,Japan,South Korea,Australia,and EU member states have seen significantly fewer overall biopharmaceutical product launches than the United States over the past 20 years.Figure 4:Percentage of New Active Substan

103、ces Launched,20002019,Select Economies13U.S.GermanyUKItalyFranceCanadaJapanSouth KoreaAustralia80%100%60%40%20%0%89%85%82%76%70%66%61%56%47%19This disparity between the U.S.and other developed OECD economies is even more pronounced when looking at more advanced and specialist products such as biolog

104、ics and oncology drugs.Figure 5 shows the percentage of new biologic products launched in individual economies between 2000 and 2019.Apart from Germany and the UK,economies such as France,Italy,Canada,Japan,South Korea,and Australia have seen significantly fewer biologics launched than the United St

105、ates had over the past 20 years.Moreover,economies with the most severe price controls in placesuch as South Korea and Australiahave seen fewer than half of new biologics launched in the same period.Australia,in particular,stands out.Over the 20-year period studied,only 38%of new biologics were laun

106、ched in Australia.Apart from South Korea(at 49%),this rate is not comparable to other high-income economies.Instead,it is closer to the launch rate of developing and emerging markets such as Argentina(35%)and Thailand(40%),both of which have far less purchasing power and far fewer resources invested

107、 in their health systems.Figure 5:Percentage of New Biologic Active Substances Launched,20002019,Select Economies14U.S.GermanyUKItalyFranceCanadaJapanSouth KoreaAustralia80%100%60%40%20%0%90%90%84%73%73%62%59%49%38%20Furthermore,there is a similar disparity for oncology products between the U.S.and

108、economies with biopharmaceutical price controls in place.Figure 6 shows the percentage of new oncology products launched in the sampled economies between 2000 and 2019.Apart from Germany and the UK,economies such as France,Italy,Canada,Japan,South Korea,and Australia have seen significantly fewer on

109、cology products launched than the U.S.had over the past 20 years.Moreover,just as with biologics,economies with the most severe pricing,reimbursement,and price controls in placesuch as South Korea and Australiahave seen substantially fewer oncology products launched during the same period.Figure 6:P

110、ercentage of New Oncology Active Substances Launched,20002019,Select Economies15U.S.GermanyUKFranceItalyCanadaJapanSouth KoreaAustralia80%100%60%40%20%0%91%91%87%77%75%69%68%59%41%21The most recent data on the launch of new cancer medicines echo these findings and show how even Germany and the UKwhi

111、ch in the past have been the closestare falling behind the U.S.IQVIAs latest Global Oncology Trends 2022 report published in May 2022 shows how the disparity in product launches between the U.S.and other economies with national price controls in place is,in fact,growing.Figure 7 shows the total numb

112、er of new oncology active substances launched in the past five years(20172021)and the proportion available in the U.S.compared with those in the EU and the UK.As Figure 7 shows,out of 104 new products launched globally since 2017,80%were launched in the U.S.,but only 58%were launched in Germany,Fran

113、ce,Italy,and the UK.Figure 7:Number of New Oncology Active Substances Launched Globally,20172021,Select Regions16Global8000Germany,France,Italy,UKU.S.1048358These data paint a clear picture:There is a large disparity in product launches and availability of new medicines between the U.S.an

114、d other OECD member states that have national price controls in place.However,the launch of a product on a given market does not always equate to actual patient access.In addition to setting and controlling prices,an important barrier to free and fair market access consists of reimbursement evaluati

115、on.Many payers(whether public or private)have an elaborate evaluation to determine whether a product should be included on a given formulary and the rate of reimbursement.In a growing number of health systems,the reimbursement process often involves a pharmacoeconomic or HTA evaluation that assesses

116、 a given products cost effectiveness.Depending on the design and function of the HTA body and underlying assessment methodology used,this assessment can include a cost-effectiveness threshold and benchmark to assess the cost effectiveness of new products through quality-adjusted life years(QALYs).A

117、QALY is a matrix that measures the value of a given product based on two dimensions:the time added to patients lives due to the use of a given product and the quality of life experienced during these years.Outside of the U.S.,most governments restrict access to their respective national health syste

118、ms through reimbursement 22limits,health technology and cost-effectiveness assessments,and reference pricing.In Europe,France,Italy,Germany,and the UK all make use of such tools,as do Australia,Canada,and South Korea.Critically,there can be a long lag between market authorizationthat is,the date by

119、which a new product is approved for use and launched in an economyand inclusion for public reimbursement.For health systems that are predominantly publicly funded and organized,the latter date,not the product launch date,determines when most patients can actually access a new product.Looking at the

120、available data and evidence,it is clear that many economies have a considerable lag between product launch and inclusion for public reimbursement.For example,Figure 8 shows the results of the European Federation of Pharmaceutical Industries and Associations(EFPIA)and IQVIAs annual Patients W.A.I.T.(

121、Waiting to Access Innovative Therapies)Survey from 2022.This survey measures the rate of availability and patient access to new and innovative medicines in Europe.17 As Figure 8 shows,the number of days patients in Europes largest economies(Germany,France,Italy,Spain,and the UK)wait,on average,to ac

122、cess innovative biopharmaceuticals is considerable.It ranges from 133 days in Germany as long as 500 days in Spain.Figure 8:Time to Availability(Days From Market Authorization and Availability to Patients Through Public Reimbursement)3002001000GermanyEnglandScotlandItalyFranceSpain1333404

123、23As Figure 8 shows,apart from Germany,patients in Europes largest economies must wait almost a year or more before they can access new medicines.Long delays are particularly more pronounced in France and Spain.Outside of Europe,there are also long access lags in both Australia and Canada

124、.In Australia,for instance,a 2018 study found that only 46%of all drugs registered in the country between 2012 and 2017 were reimbursed(with a similar share for first-in-class drugs).19 On average,the reimbursement evaluation for the products studied took 426 days,considerably longer than the OECD a

125、verage for this period.The results are similar in Canada.For example,a 2016 report conducted by IMS Health Canada for Innovative Medicines Canada shows how Canadian patients have access to fewer innovative treatments than in other OECD economies.20 The study finds that there are long lags between ma

126、rket authorization and inclusion for public reimbursement.On average,for the period studied(20102014),it took 449 days from market authorization to reimbursement.Looking at access across all Canadian provinces(formulary and reimbursement decisions are made provincially in Canada),the study finds tha

127、t only 37%of drugs were reimbursed and available to 80%or more of the population.There were particular gaps in availability for more advanced treatments,including cancer medicines and biologic products.Only 59%of new cancer medicines were available to 80%or more of the population.For new biologics,t

128、his ratio was even lower at 23%.More recent data suggest that the time taken for reimbursement evaluation in Canada has gotten longer.Data compiled by Innovative Medicines Canada suggest that for the period 20122018,the average time from market authorization to reimbursement in Canada was 632 days.2

129、1ConclusionAs the 2022 results from the rebenchmarking of the Patient Access Report demonstrate,of the nine high-income OECD economies sampled,the U.S.was the sole economy that did not impose direct national price controls or other policies adopted in the name of biopharmaceutical cost containment.P

130、rior to the implementation of the IRA,the U.S.achieves a score of 94.95%.However,following the implementation of government price controls,the U.S.score will fall notably,with U.S.patients suffering as a result.While the U.S.currently score,more than 30%higher than France,Germany,and the UK,the IRA

131、price control provisions will result in U.S.consumers facing similar barriers to access as those in other OECD economies.Price controlsincluding international reference pricingimpose a fundamental market access barrier.Such policies have a real and negative impact on the availability of new,innovati

132、ve drugs and medical technologies for patients and consumers in the affected market.As this briefing document and the historical evidence demonstrate,an economys score on the Barometer corresponds with access to and availability of medical innovation.There is a largeand growingdisparity in product l

133、aunches and market availability of new medicines between the U.S.and other advanced OECD member states with strict price controls in place.The price controls included in the Inflation Reduction Act will have a detrimental impact on biopharmaceutical innovation,leading to fewer new products and medic

134、ines developed and introduced in the U.S.The research and benchmarking that this briefing document are based on were conducted before the enactment of the Inflation Reduction Act.As the federal government moves forward with implementing the biopharmaceutical price controls provisions of the legislat

135、ion in 2023,the U.S.Chamber of Commerce will recalibrate and rebenchmark the Report considering the full scope of these legislative and regulatory changes.An updated Report to be released in the second half of 2023 will provide stakeholders with an understanding of the negative impact that the Infla

136、tion Reduction Act has had on the biopharmaceutical policy environment in the United States.U.S.leadership in biopharmaceutical innovation is a result of the robust free market framework and strong protection of intellectual property.The price control measures included in the Inflation Reduction Act

137、and any subsequent attempts to set the price of medicines via federal regulationwill undermine the innovative ecosystem that empowered the U.S.to become one of the most innovative countries in the world.Prior to 2022,the U.S.framework allowed competition,rather than government intervention in the pr

138、ice-setting process,to drive down the cost of innovative medicines.As this briefing document illustrates,the existing U.S.framework has resulted in patients having primary and extensive access to new lifesaving treatments and cures.Government officials must consider the implications of price control

139、s on patients before proceeding with the implementation of a framework that would jeopardize U.S.leadership in biopharmaceutical innovation and patient access to treatments.2425AppendixMethodology,Sources,and Indicators ExplainedThe 2019 Report consisted of 16 indicators and four separate categories

140、.For the purposes of rebenchmarking the Report in 2022,only the four indicators under Category 4:pricing,reimbursement,and access regulation to a national biopharmaceutical market are benchmarked in this document.Indicators ExplainedThis section explains how each of the four indicators in the 2022 R

141、eport is measured and scored.Category 4:Pricing,Reimbursement,and Access Regulation to a National Biopharmaceutical MarketOverarching philosophy and direction of health and biopharmaceutical system:cost cutting versus recognizing and incorporating new products and biopharmaceutical innovationthe ext

142、ent to which a given economys health and biopharmaceutical system(including de jure laws,regulations,rules,official guidelines,and/or de facto practices)is geared toward cost cutting(regardless of pricing and reimbursement system and methodology used)versus recognizing and incorporating new products

143、 and biopharmaceutical innovation.The latter can be defined,for example,through market-based pricing or appropriate recognition of innovation within an economys pricing and reimbursement system and decision-making process.This is a mixed indicator.Pricing and reimbursement decision-making and proces

144、sthe extent to which a given economys pricing and reimbursement process and decision-making(1)are transparent,(2)follow due process,and(3)use comparisons that are made(regardless of methodology used)on the basis of reaching the lowest possible price or rate of reimbursement or on a basis that values

145、 the level of innovation.This is a mixed indicator.26Biopharmaceutical-specific procurement preferencesthe extent to which the procurement of biopharmaceutical products(including de jure laws,regulations,rules,official guidelines and/or de facto procurement practices)by public and/or private payers

146、gives preference to local manufacturers.Examples of such preference include(but are not limited to)higher prices paid to local manufacturers,tendering preferences for local manufacturers,and restrictions on foreign vendors ability to participate in public procurement.This is a mixed indicator.Availa

147、bility of new,innovative biopharmaceutical treatments and productsmeasured by the availability of new cancer medicines on the domestic market.For the U.S.,France,Germany,Italy,and the UK,this is measured using the Global Oncology Trends 2022 report published by IQVIA in May 2022.For the remaining ec

148、onomies where data are not available,this is measured by the original 2019 Report indicator(i.e.,availability of new oncology products within two years of global launch between 2012 and 2016).22 This is a numerical indicator.Scoring MethodologyThe scoring methodology used in the original 2019 Report

149、 has been retained.Each indicator can score a value between 0 and 1,and the cumulative score of the Report ranges from a minimum of 0 to a maximum of 4.Indicators can be scored using two distinct methods:numerical and mixed.Numerical indicators are those that are based on a quantitative source and c

150、ompared to a baseline used.The score for each economy on this indicator is calculated by dividing the quantitative score for each economy by the relevant baseline using the numerical formula n quantitative score/the baseline used.The Report includes only one numerical indicator,which measures the pe

151、rcentage of availability of innovative oncology medicines in the economies benchmarked.The remaining indicators that make up the rebenchmarked Report are mixed indicators.The final score for these indicators is based on an even split between these:1.Primary and/or secondary legislation(regulation),r

152、elevant rules,and guidelines in place2.The actual application and enforcement of the relevant primary and/or secondary legislation,rules,and guidelines in placeThe use of mixed indicators provides flexibility when scoring and allows the Report to more effectively accommodate gray areas in economy pe

153、rformance for a given indicator.Specifically,it is possible to assign a partial score rather than only 0 or 1.Five possible scores are available within a mixed indicator:0,0.25,0.5,0.75,and 1.The range of scores available for mixed indicators means that greater nuance can be used when individual ind

154、icators are scored;the practical result is that economies can receive partial scores for an indicator,which in some cases are a better approximation of their given reality.27SourcesScoring in the Report is based on both qualitative and quantitative evidence.To provide as complete a picture of an eco

155、nomys market access environment as possible,this evidence is drawn from a range of sources.All sources used are publicly available and accessible to all.The following is an outline of the types of sources used.Government(Legislative/Regulatory)Sources from legislative and executive government branch

156、es and agencies include the following:Primary legislation Secondary legislation(regulation)from executive,legislative,and administrative bodies Reports,rules,and published guidelines from government agencies(and,where relevant,parliamentary committees),including in particular government institutions

157、 dealing with foreign investment rules and restrictions Internal departmental guidelines,assessment protocols,and policiesJudicial/LegalSources from judicial authorities and legal practitioners include the following:Court cases and decisions,including:Legal opinions written by judges Legal analysis

158、and opinions written by legal practitionersInternational Institutions and Third PartiesThese sources include the following:Data,studies,and analysis from international organizations such as the OECD,WTO,IMF,UNCTAD,and WIPO Publicly available reports,studies,and government submissions by industry org

159、anizations Reports from NGOs and consumer organizationsAcademicAcademic sources include the following:Academic journals Legal journalsNewsNews sources include the following:Newspapers News websites Trade press28Endnotes1.See Department of Health and Human Services,Centers for Medicare&Medicaid Servi

160、ces(2020,November 27),42 CFR Part 513 CMS5528IFC,RIN 0938AT91.Most Favored Nation(MFN)Model,Federal Regis-ter,vol.85,Rules and Regulations.The economies included in the basket for the first year of analysis were Australia,Austria,Belgium,Canada,Den-mark,Finland,France,Germany,Iceland,Ireland,Israel,

161、Italy,Japan,Republic of Korea,Luxembourg,Netherlands,New Zealand,Norway,Spain,Swe-den,Switzerland,and the UK.2.See H.R.3117th Congress(20212022):Elijah E.Cummings Lower Drug Costs Now Act,introduced April 2021,Sponsor Rep.Pallone,Frank,Jr.D-NJ-6.3.Large trading partners,including the U.S.and EU,keep

162、 detailed records of the major market access trading barriers in place around the world.In the U.S.,the United States Trade Representa-tive(USTR),Department of Commerce,and State Department all independently and jointly provide thorough assessments of the international trading environment through th

163、e National Trade Estimate Report on Foreign Trade Barriers,Investment Cli-mate Statements,and Country Commercial Guides.Similarly,the European Commission lists barriers to trade in its Market Access Database(MADB).Major international institutions track barriers to trade and foreign direct investment

164、.For exam-ple,the OECD has developed an FDI Regulatory Restrictiveness Index(FDI Index),which measures the extent to which foreign direct investment is limited through foreign equity limitations;screen-ing or approval mechanisms;restrictions on the employment of foreigners as key personnel;and opera

165、tional restrictions,e.g.,restrictions on branching and on capital repatriation or on land ownership(see B.Kalinova et al.(2010)OECDs FDI Restrictiveness Index:2010 Update,OECD Working Papers on International Investment 2010/03).The WTO allows for the recording and tracking of trade disputes,the regi

166、stration of nontariff measures,and other trade related information.4.For full details of the indicators included in the Report and methodology,see the appendix.5.J.A.DiMasi et al.(2003).The price of innovation:New estimates of drug development costs.Journal of Health Economics 22(2):151185.6.J.A.DiM

167、asi,H.G.Grabowski,and R.W.Hansen(2016).Innovation in the biopharmaceutical indus-try:New estimates of R&D costs.Journal of Health Economics,47,:2033.7.A full description and definition of each of these indicators are available in the appendix.8.See USTR(2022),2022 National Trade Estimate Report on F

168、oreign Trade Barriers,302303;PhRMA(2022),2022 Special 301 Report,4557.A Towse(2019),“HTA in Japan:Failing to Meet International Good Practice?”OHE News,March 15,2019;M Hasegawa et al.(2020),“Formal Implementation of Cost-Effectiveness Evaluations in Japan:A Unique Health Technology Assessment System

169、,”Value in Health,January 2020,23(1),pp.4351.9.2010 reforms changed the German biopharmaceu-tical policy environment dramatically,but during the period studied,the P&R system was based more on market-based pricing.10.P.M.Danzon and A.J.Epstein(2008),“Effects of Regulation on Drug Launch and Pricing

170、in Interde-pendent Markets,”National Bureau of Economic Research,May 2008.11.M.Kyle(February 2007),Pharmaceutical Price Controls and Entry Strategies,Review of Econom-ics and Statistics,89(1):8899.12.N.Varol,J.Costa-Font,and A.McGuire(2010),Do International Launch Strategies of Pharmaceutical Corpor

171、ations Respond to Changes in the Regula-tory Environment?LSE Health,Working Paper No.19/2010.See appendix.13.Life Sciences Ontario(2020),“New Medicine Launches:Canada in a Global Context,”slide 29,June 22,2020.Based on a sample of 516 global launches between January 1,2000,and December 31,2019.Data

172、extracted and provided to Life Sci-ences Ontario by IQVIA.14.Ibid.Slide 34.Based on a sample of 164 global launches between January 1,2000,and December 31,2019.Data extracted and provided to Life Sci-ences Ontario by IQVIA.2915.Ibid.Slide 36.Based on a sample of 122 global launches between January 1

173、,2000,and December 31,2019.Data extracted and provided to Life Sci-ences Ontario by IQVIA.16.IQVIA Institute for Human Data Science(2022),Global Oncology Trends 2022,Outlook to 2026,May 2022,IQVIA,7.17.M.Newton et al.(April 2022),“EFPIA Patients W.A.I.T.Indicator 2021 Survey,”EFPIA&IQVIA.18.Ibid.Sli

174、de 12.This is IQVIAs description of its data set:“The time to availability is the days between marketing authorisation and the date of availability to patients in European countries(for most this is the point at which products gain access to the reimbursement list).The marketing authorisation date i

175、s the date of central EU authorisation.”The number of days estimated for France is based on products included under the ATU system.19.Medicines Australia(2018),Comparison of Access and Reimbursement Environments,4th ed.20.Innovative Medicines Canada&IMS Health Cana-da(2016),Access to New Medicines i

176、n Public Drug Plans:Canada and Comparable Countries:2016 Annual Report,5.21.Innovative Medicines Canada,Canadian Public Re-imbursement Timelines,pCPA Trends Update page.https:/innovativemedicines.ca/resources/crt/22.This assessment is based on market availability data and assessment by IQVIA Institu

177、te for Hu-man Data Science.For the period 20172021 this assessment is based on 104 oncology products launched globally.For the period 20122016 this assessment is based on 55 oncology products launched globally.Availability is based on sales in the market irrespective of formulary(private or pay-er)inclusion.See IQVIA(2022),Global Oncology Trends 2022,7.;and IQVIA(2018),Global Oncology Trends 2018,IQVIA Institute,24.