Rule of Prevention: a potential framework to evaluate preventive interventions for rare diseases.
Eddie Gibson, Daniel A Ollendorf, Steven Simoens, David E Bloom, Federico Martinón-Torres, David Salisbury, Johan Louis Severens, Mondher Toumi, Daniel Molnar, Kinga Meszaros, Woo-Yun Sohn, Najida Begum
Author Information
Eddie Gibson: Wickenstones Ltd, Abingdon, UK.
Daniel A Ollendorf: Institute for Clinical Research and Health Policy Studies (ICRHPS), Center for the Evaluation of Value and Risk in Health (CEVR), Tufts Medical Center, Boston, MA, USA.
Steven Simoens: Clinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, Belgium.
David E Bloom: Harvard T.H. Chan School of Public Health, Boston, MA, USA.
Federico Martinón-Torres: Department of Pediatrics, Translational Pediatrics and Infectious Diseases, Pediatrics Department, Hospital Clínico Universitario de Santiago de Compostela, Santiago de Compostela, Spain.
David Salisbury: Royal Institute of International Affairs, Chatham House, London, UK.
Johan Louis Severens: Severens HTA Consultancy, Venray, The Netherlands.
The benefits of preventive interventions lack comprehensive evaluation in standard health technology assessments (HTA), particularly for rare and transmissible diseases. To identify possible considerations for future HTA using analogies between the treatment and prevention of rare diseases. An Expert panel meeting assessed whether one HTA assessment framework can be applied to assess both rare disease treatments and preventive interventions. Experts also evaluated the range of value elements currently included in HTAs and their applicability to rare, transmissible, and/or preventable diseases. A broad range of value should be considered when assessing rare, transmissible disease prevention. Although standard HTA can be applied to transmissible diseases, the risk of local outbreaks and the need for large-scale prevention programs suggest a modified assessment framework, capable of incorporating prevention-specific value elements in HTAs. A 'Rule of Prevention' framework was proposed to allow broader value considerations anchored to severity, equity, and prevention benefits in decision-making for preventive interventions for rare transmissible diseases. The proposed prevention framework introduces an explicit initial approach to consistently assess rare transmissible diseases, and to incorporate the broader value of preventive interventions compared with treatment.
