New treatments for serious conditions: ethical implications

摘要: Approval of Spinraza (nusinersen) for treatment spinal muscular atrophy prompts consideration a number ethical issues that arise whenever new is proposed serious condition, especially one rare and can devastatingly affect children. Patients, families, clinicians, researchers, institutions policymakers all must take account the ways newly available treatments informed shared decision-making about therapeutic research options. The to consider include: addressing what still uncertain unknown; possibility potential benefits will be exaggerated harms underemphasized in media, by advocacy organizations, consent forms processes; high cost many novel drugs biologics; effects including conditions variable phenotype state-mandated newborn screening panels; how change standard care, altering not known disorder posing challenges at both individual policy levels. good news brings thus requires additional attention its implications, improve counseling options patients involved their care.