Brussels, Belgium

21st September 2018

Health Technology Assessment Dossiers



Beyond New Drug Applications, biostatistics and statistical programming professionals across the pharmaceutical industry are now heavily engaged in the preparation of Health Technology Assessment (HTA) dossiers. HTA dossiers are submitted to HTA agencies across Europe and other countries (including Australia, Korea and soon Japan) in support of pricing and reimbursement negotiation of newly approved drugs as well as later on during the drug life cycle.

The German AMNOG Pharmaceuticals Market Reorganization Act requires pharmaceutical companies marketing their products in Germany to submit evidence showing that the new product is more effective than the existing standard of care. In parallel, other HTA agencies (like the UK NICE agency) are rather expecting the presentation of cost-effectiveness modeling as part of HTA dossiers submitted to them. In both cases, the biostatisticians and statistical programmers organisation will be leveraging the analysis and reporting used in support of the compound clinical study reports to contribute to the development of the HTA dossiers, directly with additional clinical data analysis or indirectly by providing clinical data and analysis as input to economic models. The analysis will also go beyond what has been presented in the original clinical study reports, for instance in using methodologies of indirect treatment comparisons and network meta-analysis to allow the comparison of the company’s own drug with existing standard of care on the various markets.



The Role of Biostatistics in Health Technology Assessment: Present and Future – William Malbecq & Kristel Vandormael, MSD

Real-world Patient Treatment Journeys in Combination with Health Assessments at Critical Drug-changing Time Points – Berber Snoeijer, PHARMO Institute

Matching-Adjusted Indirect Comparison (MAIC) in HTA and its Application Using SAS Procedures – Rachid Massaad, William Malbecq, Harris Kampouris & Dave Gelb, MSD

Developing a Global Value Dossier (GVD) to Best Meet Local HTA Requirements – Fred Sorenson, Xcenda

Framework for Developing and Maintaining a Macro Library‚Ä®in Health Technology Assessment – Thierry Paquet & Amit Bhaniani, MSD

Comparison of Different Methods in Meta-analysis of Single-arm Trials with Low Event Rate Binary Outcome; A Case Study in Cervical Cancer Trials – Grace Antony & Edwin Hoeben, MSD

Use of Dictionaries in Automated RTF Table Translation – Qian Wang & Ligia Adamska, MSD

Generating the Relevant Comparative Evidence to Support Market Access in Absence of a Randomized Comparative Trial Versus the Right Comparator or, Versus any Comparator – Joris Diels, Nolen Perualila, Pushpike Thilakarathne & Suzy Van Sanden, J&J

RTF Table Translation of Clinical Trial Results: An Automated Approach – Qian Wang & Amit Bhaniani, MSD

A Single Study that Solves Multiple End Point Preferences — Statistical Solution for Binary End Point Models – Martin Dörr, Clinipace

How to Drive a Longitudinal ADaM Data Set for Inverse Probability of Censoring Weighting Analysis – Yirong Cao & Shahrul Mt-Isa, MSD


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