Legislativní a etický problém dostupnosti orphan drugs a ATMPs (Advanced Therapy Medicinal Products)
| Title in English | Legislative and ethical problem of availability of orphan drugs and ATMPs (Advanced Therapy Medicinal Products) |
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| Authors | |
| Year of publication | 2024 |
| Type | Appeared in Conference without Proceedings |
| MU Faculty or unit | |
| Citation | |
| Description | The presentation addresses the legislative and ethical issues of the availability of orphan drugs and advanced therapies (ATMPs) in the Czech Republic. It describes the legal framework, including § 39da of Act No. 48/1997 Coll., which allows reimbursement of mass-produced drugs without requiring a cost-effectiveness analysis, while the rules for individually prepared drugs remain unclear. It also points to the lack of transparency in the use of Section 16, which is used for exceptional reimbursement of medicines. Ethical constraints include the limited availability of medicines for patients with ultra-rare diagnoses, particularly in paediatric oncology, and the need for personalised treatment. The presentation highlights the importance of collaboration between academic and commercial entities to produce individually formulated drugs for these patients. |
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