Changes in the regulatory environment: The EU economic assessment study

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1 Changes in the regulatory environment: The EU economic assessment study Dr Peter Varnai Technopolis Group 8 February 2018

2 Introduction Present the independent study of the economic impact of the Paediatric Regulation commissioned by DG SANTE in Oct Study analysed Regulatory compliance costs to industry The value and the costs of the rewards and incentives Societal and broader economic benefits Costs and benefits of the current system Overall how much the rewards cost to society and healthcare payers? are these comparable to the costs that companies bear to comply with regulation or to engage in voluntary research? 2

3 Regulatory compliance costs to industry ( ) Administrative and R&D cost data collected via survey Data from 26 organisations: 19 companies and 7 FP7 participants Several EFPIA member companies; 1 non-profit; and 6 SMEs Data on 85 PIPs from 24 organisation Data on 36 waiver applications from 11 organisation Number of PIPs by stage Not started 2. Ongoing 3. Discontinued 4. Completed (final compliance check) Under development Number of PIPs by paediatric product stage 6 2. Application for marketing authorisation submitted 4 3. Marketing authorisation in EU obtained Marketed in 5. Discontinued at least one EU member state 3

4 Regulatory compliance costs to industry Overview of calculated costs of all PIPs PIPs* Total administrative and R&D costs of PIPs for the industry per year 2,103m Average number of first PIP decisions per year** Average cost per PIP 19,608k Average administrative cost per PIP 728k Average R&D cost per PIP 18,879k Number of clinical studies agreed per PIP 2.9 Average cost per study 6,831k * Data based on period ** Based on data from the Draft 10-year report, EMA 4

5 Regulatory compliance costs to industry Overview of calculated costs of all waiver applications Waiver* Total administrative costs of waiver applications for the industry per year Average number of waiver decisions per year** ,585k Average administrative cost per waiver application 70k Average cost per waiver application as a percentage of the average administrative cost per PIP application 10% * Data based on period ** Based on data from the Draft 10-year report, EMA 5

6 Millions Regulatory compliance costs to industry Breakdown of the estimated total regulatory costs incurred (based on data for ), per year in millions of euros Administrative costs - waiver Administrative costs - PIP In-vitro studies and animal studies 77 Development of a paediatric formulation 341 Phase II paediatric clinical trials Phase III paediatric clinical trials Other R&D costs Total costs Total estimated administrative and R&D costs of PIPs that are already discontinued: 144m, 7% of total costs Since many on-going PIPs will be discontinued at later stages, this figure is an underestimate of regulatory costs of discontinued PIPs 6

7 Millions Regulatory compliance costs to industry Estimated cost of a paediatric study Estimated cost of a paediatric study based on RAND 2004 is 5m [ 1-9m] Average cost - based on Li et al. (2007) was not intended to be representative of total industry Average cost - based on 2016 survey includes cost incurred in relation to phase II and phase III paediatric clinical trials and other costs incurred in relation to completing a paediatric investigation plan. 7

8 Regulatory compliance costs to industry Cost comparison: paediatric vs adult* clinical trials Phase II paediatric Phase II Phase II cost ratio Phase III paediatric Phase III Phase III cost ratio Average 7.3m 66.2m 11% 15.7m 288.4m 5% Standard deviation DiMasi, J., Grabowski, H., and Hansen, R. (2016). Innovation in the pharmaceutical industry: new estimates of R&D costs. Journal of Health Economics R&D costs of 106 new drugs were obtained from a survey of 10 biopharmaceutical firms Costs for compounds that were abandoned were linked to costs of approved compounds 8

The value of the rewards to industry IMS Health data made available with sales volumes Q1 2008 Q3 2014 (retail & hospital) across the EU

8 products (out of 13 granted until 2015) For orphan (4 products) and PUMA (2 products) rewards it was not possible to calculate the

9 The value of the rewards to industry IMS Health data made available with sales volumes Q Q (retail & hospital) across the EU Used most sold dosage and converted packages into units Available data can (only) be used to calculate economic value of: SPC extension: 8 products (out of 13 granted until 2015) For orphan (4 products) and PUMA (2 products) rewards it was not possible to calculate the economic value due time of MA and time to patent expiry Geographical spread of granted SPCs in the EU, * collaboration with Ecorys BV

10 3/08 6/08 9/08 12/08 3/09 6/09 9/09 12/09 3/10 6/10 9/10 12/10 3/11 6/11 9/11 12/11 3/12 6/12 9/12 12/12 3/13 6/13 9/13 12/13 3/14 6/14 9/14 3/08 6/08 9/08 12/08 3/09 6/09 9/09 12/09 3/10 6/10 9/10 12/10 3/11 6/11 9/11 12/11 3/12 6/12 9/12 12/12 3/13 6/13 9/13 12/13 3/14 6/14 9/14 Volume share Volume Share The value of the rewards to industry Generic entry: volume share changes Italy Sweden 100% 80% 60% 40% 20% 0% 100% 80% 60% 40% 20% 0% % non-generic % generic Time (quarter/year) % non-generic % generic Time (quarter/year) Sweden: generics quickly overtake branded volumes Italy: volumes have not reached new equilibrium by the end of analysis period Based on IMS HEALTH data

11 3/08 6/08 9/08 12/08 3/09 6/09 9/09 12/09 3/10 6/10 9/10 12/10 3/11 6/11 9/11 12/11 3/12 6/12 9/12 12/12 3/13 6/13 9/13 12/13 3/14 6/14 9/14 3/08 6/08 9/08 12/08 3/09 6/09 9/09 12/09 3/10 6/10 9/10 12/10 3/11 6/11 9/11 12/11 3/12 6/12 9/12 12/12 3/13 6/13 9/13 12/13 3/14 6/14 9/14 Percentage changes Percentage changes The value of the rewards to industry Generic entry: price changes 120% Italy 120% Sweden 100% 100% 80% 80% 60% 60% 40% 40% 20% 20% 0% 0% Time (quarter/year) % total (weighted) % non-generic % generic Time (quarter/year) % total (weighted) % non-generic % generic Branded prices drop by 60% in both cases but generic prices differ Sweden: revenue from generics overtake branded one Based on IMS HEALTH data

12 The value of the rewards to industry Economic value (x1,000 ) data available for 8 products resulted in a total economic value of 517m total extrapolated economic value for 13 products with patent expiry before 31/12/2015 is 925m Drug A (8) 58,122 Drug B (8) 14,105 Drug C (7) 9,355 Drug E (11) 146,497 Drug F (10) 105,217 Drug G (9) 113,221 Drug H (9) 31,183 Drug I (4) 39,548 Total 517,249 Average 64,656 economic value generated by SPC extension is the difference between the real situation with SPC extension and a hypothetical situation without SPC extension Based on IMS HEALTH data

13 The societal and economic benefits Delphi consultation of (116) non-industry experts (researchers, clinicians, hospital pharmacists, regulatory affairs officers, patient representatives) Positive view on increased research, awareness, and the availability of more high quality information and additional funding Negative view on the replacement of existing paediatric treatments with less toxic and more effective alternatives, and the number of children treated with right medicine at the right time and with the right dose 90% 80% 70% 60% 50% 40% 30% 20% 10% Given your answers above, do you think that in the long-term there will be measurable benefits as a result of more effective treatment of children: No Yes Unclear 0% An increase in children's school attendance A reduction in the time carers need to take off work to care for their children An increase in qualityadjusted life years for children A reduction in mortality of children with lifethreatening illnesses

14 Cost-benefit model and assessment 3 level analysis of benefits: 1. All PIPs (through investment mainly by the pharmaceutical industry) contribute to wider public good benefits/ social returns 858 PIPs (completed, ongoing, discontinued, etc) in PIPs with positive statement of compliance (but not all requiring additional healthcare costs) have had positive effect on society via patient SmPC/PL change/safer paediatric medicine 85 PIPs related to unique INNs 3. PIPs with reward secured can be assessed as contributed to specific paediatric benefits and (will) have incurred healthcare costs 39 SPC, 5 Orphan, 3 PUMA until 2015 with 13 SPC extensions realised 8 products used in detailed modelling in this study * collaboration with empirica GmbH

15 Cost-benefit model and assessment PIPs with reward secured can be assessed as contributed to specific paediatric benefits and (will) have incurred healthcare costs: 8 products (SPC) with detailed data: Estimated total cost of 6-month SPC extension ( ) to society: 590m Total cumulative benefits after 10 years: 199m cash benefits (avoidable hospital, out-patient treatment, etc) avoidable non-cash benefits (avoidable mortality, reduced QoL, time by informal carers) large variations for therapeutic area/ medicinal products! some with 32-66m net benefit over 10 years, others with negative 4-166m intangible benefits and societal return: eg, chronic respiratory disease with a relatively high share of episodes relating to paediatrics vs migraine with lower share of episodes relating to paediatrics

16 Cost-benefit model and assessment Results of extrapolations: Consider 108 additional PIPs with positive statement of compliance: intangible benefits 970m vs the estimated extra monopoly costs of 750m The overall balance for society in the first 10 years of the Regulation is: estimated benefit of 500m vs estimated cost of 590m R&D spillovers resulting from all PIPs: 30% social rate of return on investment to society equates to 336m additional annual benefit (additional jobs, growth and innovation, crossing industries)

17 Key thoughts on the implications of this study Few applications for PUMA and Orphan rewards in first 10 years Few voluntary drug development incentivised by the Regulation No prioritisation of specific paediatric drug development Cost-benefit ratio to healthcare system depends largely on the medicinal product Over half of PIPs data generated also (plan to be) used to apply to the FDA PIPs conducted by pharmaceutical industry can be considered as an R&D investment PIPs resulted in behaviour change, paediatric research capacity building, new structures and research networks established Engagement of representatives of paediatric population in drug development EU market is becoming attractive for FDI because of stable regulatory framework Caveats: Heterogeneity due to products therapeutic area and adult product development stage at the time of PIP application Most PIPs are still ongoing and about 16% discontinued

18 Amsterdam Berlin Bogotá Brighton Brussels Frankfurt/Main Paris Stockholm Tallinn Vienna 18

19 The societal and economic benefits Industry view of societal benefits % of PIPs involved a collaboration with a research network Informal networks and consultations with paediatricians European Paediatric Formulation Initiative (EUPFI) Task-force in Europe for Drug Development for the Young (TEDDY) 0 Change in culture New and improved medicine New knowledge for industry New knowledge for prescribers Network effect Other Medicines for Children Research Network (MCRN) Innovative Therapies for Children with Cancer (ITCC)

20 Cost-benefit model and assessment A detailed model was developed that aggregates gains and losses, primarily to the healthcare payer: 1. Cost (monopoly rent) of 6-month SPC extension Minus co-payments for patients Equals net cost to system (NHS, statutory health insurances) 2. Benefit indicators linked to reduced adverse drug events in children and/or better treatment Avoided hospitalisation costs Avoided outpatient treatment and ambulatory services costs Avoided reduced quality of life (disability, permanent damage) Avoided mortalities (Value of a statistical life per year, Average years lost per death) Avoided lost time by informal carers Additional benefit per paediatric episode due to better treatment

21 The societal and economic benefits In the long term 1. Benefits to patients and consumer groups Greater awareness in health literacy of the general population Involvement of the paediatric population in discussions on drug development 2. Benefits to industry Change of culture, more inclusive focus on developing new and improved medicine for the paediatric population EU market is becoming more attractive for FDI because of stable regulatory framework; opportunities for commercial clinical trial investment and attracting research from outside the EU 3. Increase in jobs and growth as a result of the increased investment in R&D

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