Moderator: Dennis Meletiche, PharmD, Chief Strategy Advisor, Maple Health Group, LLC, Cambridge, MA, USA
Speakers: Rosa Maria Galindo Suarez, MHA, Senior Consultant, PERMA Consultores, Mexico City, Mexico; Diego Rosselli, MD, MEd, MHP, Professor of Health Economics, Pontificia Universidad Javeriana, Medical School, Bogota, Colombia; Maria Isabel Acevedo, BA, MEd, Presidente, FAME Colombia & Active Member, FECOER, Bogota, Colombia; Andre Liamas, BA, MSc, Director, Brazil Market Access & Corporate Affairs, Biogen Idec, São Paulo, Brazil
The purpose of this educational symposium is to discuss the challenges faced by health technology assessment (HTA) agencies and health care decision makers in Latin America in evaluating novel therapies for rare/orphan diseases and best practices in assessing the value of these treatments. The symposium will present the orphan drug policies that exist in Latin American countries today, with a focus on the impact of these policies on time to access and reimbursement relative to other markets. In addition, the symposium will highlight several methodologies that have been developed for the assessment of rare disease products that have been used in other regions and will present spinal muscular atrophy (SMA) as a case study. The panelists will share the importance of implementing a robust evidence package and holistic assessment framework (including a multi-stakeholder view and multi-criteria analysis) for addressing uncertainties in clinical and economic outcomes in order to ensure successful access and reimbursement of novel rare/orphan disease therapies.