Abstract
Background New scientific progress in the “precision medicines” direction and a better diagnosis of rare diseases have led Regulators and Payers to focus their vigilance towards the real life setting. Although Regulators and Payers are taking into consideration patient pressures for rapid access to treatment, nevertheless they must balance the difficulties of taking significant decision coping with uncertainties when deciding on pricing and reimbursement processes.
Highlights
New scientific progress in the “precision medicines” direction and a better diagnosis of rare diseases have led Regulators and Payers to focus their vigilance towards the real life setting
Results the use of MEAs representing a significant tool for the management of a sustainable pharmaceutical expenditure and for the generation of further clinical evidences by registries’ adoption, they can be considered the key element to overcome this concern
Materials and methods A large number of mechanisms (Managed Entry Agreements) have been developed to limit the reimbursement of medicines to those subpopulations that are most likely to benefit from treatment
Summary
New scientific progress in the “precision medicines” direction and a better diagnosis of rare diseases have led Regulators and Payers to focus their vigilance towards the real life setting. Regulators and Payers are taking into consideration patient pressures for rapid access to treatment, they must balance the difficulties of taking significant decision coping with uncertainties when deciding on pricing and reimbursement processes. Among all participants on how to facilitate the access of orphan drugs in the real life setting, given the high cost and the lack of evidence of these products
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