FDA孤儿药认定请求持续飞涨
出自识林
FDA孤儿药认定请求持续飞涨
笔记 2016-07-22 Lachman CONSULTANTS FDA孤儿药认定（ODD）在过去几年中以年度30%的速度跳跃性飞涨并不少见。FDA孤儿药产品研发办公室（OOPD）主任Gayatri Rao医师指出，“2014年比前一年的数量增加了30%。而这一纪录于下一年被打破，2015年我们收到将近470件请求。而且这一步伐并没有放缓的迹象。实际上，将2016年到目前为止的新收到的请求数量与2015年同期相比，似乎还有30%的增加。” 当申办人证明一个特定药品将用于治疗一种罕见病（在美国不超过20万名患者）的特定适应症时，方可授予孤儿药认定。孤儿药认定可以令药品申办人获得孤儿药法案规定的多种研发激励，包括符合资格的临床试验的税收减免。获得孤儿药认定的处方药产品上市申请不需要缴纳处方药使用者付费，除非申请包括除药品已认定的罕见疾病或情况之外的其它适应症。 显然，这似乎显示了对孤儿药认定兴趣的增加，对于许多遭受罕见疾病或障碍的患者也是一个好迹象。ODD请求数量的上涨给OOPD及时处理这些迅速增加的请求带来了巨大压力。由于孤儿药计划不是一个付费计划，因此满足需求的资源十分有限。Rao医师表示，虽然对于审评没有正式目标，但是OOPD的内部目标是在自接收之日90天内审评75%的请求。然而，由于请求的显著增加，OOPD最近将这一目标修订为自接收之日120天内审评75%的请求。 另外，Rao医师指出，大多数ODD的审评经历2轮审评周期，为提高效率，她恳请申办人全面审查ODD要求，提供可以在首轮审评中解决所有要求的完整材料。随着请求数量的增加，而经费仍保持不变，希望未来审评时间表不会进一步延长。 Gayatri Rao医师 FDA博客文章：The Rise in Orphan Drug Designations: Meeting the Growing Demand Lachman CONSULTANTS - Bob Pollock先生 Orphan Drug Designations Skyrocket Orphan Drug Designations (ODD) at the FDA has soared over the past few years with yearly jumps of 30% not uncommon. In a blog posted today on FDA's website, Dr. Gayatri Rao, Director for the Office of Orphan Products Development (OOPD) notes that: “In 2014, we saw a 30% increase over the prior year’s record number. Yet, that record was broken the very next year when we received close to 470 requests. And the pace does not seem to be slowing. In fact, comparing the number of new requests received so far in 2016 with the corresponding date in 2015, there appears to be yet another 30% increase.” Clearly, this points to what appears to be an increased interest in orphan drug designation and is a good sign for the patients suffering from many rare diseases or disorders that currently have no treatment. The rise in the number of ODD requests places enormous pressure on OOPD to act on those burgeoning requests in a timely manner. Since the Orphan Drug Program is not a fee-based program, there are limited resources available to meet the demand. Dr. Rao noted that, while there are no formal goals for review, the OOPD had an information target goal of reviewing 75% of requests within 90 days. However, due the significant increase in requests, OOPD has recently revised that goal to 75% review within 120 days from date of receipt. She also noted that most reviews for ODD undergo 2 review cycles and, to improve efficiency, she implores sponsors to fully review the requirements for ODD and to include a complete submission that addresses all requirements in the first cycle. Just as a reminder, an Orphan Drug Designation is granted when a sponsor demonstrates that a specific drug product will be used to treat a specific indication for a rare disease (less than 200,000 patients in the US). Dr. Rao's remarks can be found here and the requirements for ODD can be found here. With the number of requests increasing and the funding remaining the same, hopefully review timelines will not extend further in the future. |