Delayed access to treatments for rare diseases: Who's to blame?

摘要: The development and commercialization of drugs for rare diseases, termed ‘orphan drugs’, has historically been economically unattractive. However, because the introduction legislation that provides financial regulatory incentives orphan drugs, new developments are making their way through approval processes. Unfortunately, delays in availability treating disease continue to persist. This paper reviews approach several jurisdictions an effort determine relative effectiveness providing patient access. Generally speaking, authorities across have recognized need enhance timely access safe, effective treatment patients with diseases able shift timelines care. greater impediment drug appears be funding, particularly publicly sponsored health-care systems. Redundancies federal provincial can result significant drugs. Clearly, more must done accelerate treatments so desperately needed by patients. Public payers held accountable process decisions—especially therapies.