Neil McAuslane

Centre for Innovation in Regulatory Science

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Lawrence Liberti

University of Southern California

Stuart Walker

Centre for Innovation in Regulatory Science

Sam Salek

University of Hertfordshire

Hubert G. M. Leufkens

Utrecht University

Magdalena Bujar

Centre for Innovation in Regulatory Science

Ting Wang

Beijing Chao-Yang Hospital

Pieter Stolk

Utrecht University

Tina Wang

Huntington Hospital

Prisha Patel

Pfizer (United Kingdom)

Iga Lipska

State University of Applied Sciences in Elbląg

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New York University Press

Cambridge University Press

Royal Australasian College of Surgeons

University of Adelaide

Centre for Innovation in Regulatory Science

Utrecht University

London School of Economics and Political Science

Pharmo Institute

Agencia de Evaluación de Tecnologías Sanitarias

University of Hertfordshire

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VP155 Synchronization Of Regulatory Approval And Health Technology Assessment Recommendation Timing

International Journal of Technology Assessment in Health Care (2017)

Neil McAuslane Tina Wang Lawrence Liberti

INTRODUCTION: Minimizing the delay between regulatory approval and Health Technology Assessment (HTA) recommendation is critical to ensure patients access to medicines of therapeutic value. The aim of this study was to evaluate the level of synchronization between the regulatory decision and HTA recommendation. METHODS: Data were collected from the public domain for new active substances that were first appraised by the HTA agency in Scotland (SMC - Scottish Medicines Consortium), France (HAS - Haute Autorité de Santé), Germany (IQWIG - Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen), Australia (PBAC - Pharmaceutical Benefits Advisory Committee) and Canada (CADTH - Canadian Agency for Drugs and Technologies), and that reached an outcome in 2014 and 2015. The year the product was approved by the European Medicines Agency (EMA), Australian Therapeutic Goods Administration (TGA) and Health Canada were also assessed. RESULTS: In 2014 and 2015, fifty-one products with HTA recommendations were identified for SMC and IQWIG, forty-two for HAS, forty for PBAC and thirty-eight for CADTH. Of the HTA agencies studied, CADTH had the lowest percentage of HTA recommendations occurring the same year as jurisdictional regulatory approval. Of the products with CADTH recommendations in 2014, only 7 percent were approved by Health Canada in the same year. By comparison, all of the products with PBAC recommendations in 2015 were approved by TGA in the same year. For 2014 and 2015, comparing the percentage of HTA recommendations with the jurisdictional regulatory agency approval the same year showed 7 percent (2014) versus 29 percent (2015) for CADTH: 35 percent versus 37 percent for SMC: 35 percent versus 44 percent for HAS; 56 percent versus 57 percent for IQWIG; and 91 percent versus 100 percent for PBAC. CONCLUSIONS: This study shows that the parallel submission mechanism to enable synchronizing HTA and regulatory decision making is effective in Australia, whilst there remains a synchronization disconnect in other countries; although this may be improving. The extent of decision timing disconnect, influence of company strategy and type of HTA outcome were also studied. This initial analysis suggests gaps between the timing of regulatory approval and HTA recommendation for HTA agencies outside of Australia.

Health Technology

Drug approval

10.1017/s0266462317003956

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PD47 Associations Of Orphan Designation And Other Drug Development-Related Factors On Rollout Times And Health-Technology-Assessment Recommendations Of New-Active-Substances

International Journal of Technology Assessment in Health Care (2022)

Belén Sola-Barrado Tina Wang Neil McAuslane

Introduction The orphan designation has been used by the European Medicines Agency to incentivize the development of drugs treating rare diseases with high-unmet medical needs by supporting their development process and economic returns. This study evaluated the impact of the regulatory orphan designation and other drug development-related factors on the rollout times and Health-Technology-Assessment (HTA) recommendations of new active substances (NASs). Methods A total of 656 HTA appraisals from 6 European countries were collected for NASs that received regulatory approval between 2012 and 2020. Multivariable logistic (positive and positive with restrictions vs. negative HTA recommendation as dependent variable) and linear regression (rollout time as dependent variable) models examined associations with regulatory orphan designation, expedited process, product type (biotechnological vs chemical), and jurisdiction (France, England, Germany, Poland, Scotland and Sweden). Rollout time was defined as months elapsed from regulatory submission to HTA recommendation (mean± standard deviation). Results Multivariable logistic regression analysis identified disparities in HTA recommendations between countries. Every month increase in rollout time conferred a 3 percent reduction in the odds of a positive recommendation (p<0.001). Review and product type did not show associations with HTA recommendation. Interestingly, orphan products showed a 99% increase in the odds of obtaining a positive HTA recommendation compared to non-orphan (p-value=0.003). We found 244 appraisals (37%) assessing an orphan product, of which 202 (83%) received a positive HTA recommendation. Multivariable linear regression analysis indicated that orphan products presented a 4.4-month rise in rollout time when compared to non-orphan products (p<0.001). The mean rollout time in months for orphan products were 25±12 in France, 30±15 in England, 21±9.1 in Germany, 37±16 in Poland, 25±12 in Scotland and 27±14 in Sweden. Conclusions Orphan designated products showed greater odds of receiving a positive HTA recommendation compared with non-orphan. A more detailed review of orphan products could result in their longer rollout time compared with non-orphan counterparts. Considerable differences were found between HTA recommendations and rollout times between jurisdictions.

Orphan drug

Health Technology

10.1017/s0266462322003063

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Use of the Certificate for Pharmaceutical Products (CPP) in 18 Maturing Pharmaceutical Markets: Comparing Agency Guidelines with Company Practice

Therapeutic Innovation & Regulatory Science (2020)

Céline Rodier Magda Bujar Neil McAuslane Prisha Patel Lawrence Liberti

Abstract Background The certificate of pharmaceutical product (CPP) was implemented to accelerate the availability of new drugs in developing countries by providing evidence of the quality of products and reducing the time to market through reliance on a prior trusted analysis. However, the CPP format, issuing process and use have not been revised since 1997 and there are significant differences among countries in regard to requirements for CPP timing, terminology, and format. We sought to determine current CPP practices versus national regulatory guidelines and to inform recommendations for the efficient use of the CPP based on the needs of the modern regulatory environment. Methods We conducted a comparative analysis of company practice versus agency guidelines across 18 maturing pharmaceutical markets using data from the Cortellis for Regulatory Intelligence® (CRI) and the Centre for Innovation in Regulatory Science (CIRS) Emerging Markets Regulatory Review Times (EMaRReT) databases and regulatory authorities’ websites. Results Of the studied 18 countries, 16 require the CPP for submission of new registrations; many accept alternative documentation but most still require legalization of the CPP and many are not compliant with the complex CPP format. Additional complicating factors include language requirements and varying local guidelines for CPP submission timing and validity dates. Conclusions With the implementation of a number of suggested improvements, the CPP can continue to serve an important role in streamlining regulatory efficiency and provide confidence in new medicines, ensuring a more efficient and effective approval process and expediting patient access to safe and effective medicines worldwide.

Expediting

Harmonization

Certificate

10.1007/s43441-020-00196-2

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Current status of CMR survey on inter-ethnic differences in clinical responsiveness

Springer eBooks (1994)

Christine Harvey Neil McAuslane Cyndy Lumley Stuart Walker

10.1007/978-94-011-1420-2_16

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Is there a need for a universal benefit–risk assessment framework for medicines? Regulatory and industry perspectives

Pharmacoepidemiology and Drug Safety (2013)

James Leong Neil McAuslane Stuart Walker Sam Salek

ABSTRACT Purpose To explore the current status and need for a universal benefit–risk framework for medicines in regulatory agencies and pharmaceutical companies. Methods A questionnaire was developed and sent to 14 mature regulatory agencies and 24 major companies. The data were analysed using descriptive statistics, for a minority of questions preceded by manual grouping of the responses. Results Overall response rate was 82%, and study participants included key decision makers from agencies and companies. None used a fully quantitative system, most companies preferring a qualitative method. The major reasons for this group not using semi‐quantitative or quantitative systems were lack of a universal and scientifically validated framework. The main advantages of a benefit–risk framework were that it provided a systematic standardised approach to decision‐making and that it acted as a tool to enhance quality of communication. It was also reported that a framework should be of value to both agencies and companies throughout the life cycle of a product. They believed that it is possible to develop an overarching benefit–risk framework that should involve relevant stakeholders in the development, validation and application of a universal framework. The entire cohort indicated common barriers to implementing a framework were resource limitations, a lack of knowledge and a scientifically validated and acceptable framework. Conclusions Stakeholders prefer a semi‐quantitative, overarching framework that incorporates a toolbox of different methodologies. A coordinating committee of relevant stakeholders should be formed to guide its development and implementation. Through engaging the stakeholders, these outcomes confirm sentiments and need for developing a universal benefit–risk assessment framework. Copyright © 2013 John Wiley & Sons, Ltd.

Toolbox

10.1002/pds.3464

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Citations (28)

Accelerating access to new medicines: Current status of facilitated regulatory pathways used by emerging regulatory authorities

Journal of Public Health Policy (2016)

Lawrence Liberti Alasdair Breckenridge Jarno Hoekman Hubert G. M. Leufkens Murray Lumpkin

Regulatory Science

Regulatory Reform

Regulatory authority

10.1057/jphp.2016.8

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