The Centers for Medicare and Medicaid Services (CMS) late Tuesday proposed a new rule that would increase payments for medical devices designated by the US Food and Drug Administration (FDA) as breakthrough devices. CMS explains how at the time of approval for these devices with a breakthrough designation, real-world data on outcomes in different patient populations is often limited. So, it can be challenging for device firms to meet the requirement for demonstrating a ...

A citizen petition filed last week by law firm Goodwin Procter on behalf of Pennsylvania-based drugmaker Braeburn calls on the US Food and Drug Administration (FDA) to revoke the orphan designation granted to Indivior’s opioid use disorder treatment Sublocade (buprenorphine extended-release injection).   “Sublocade is not now, nor was it ever a bona fide orphan drug, particularly since more than two million Americans currently are afflicted by opioid addiction,” the pe...

US Food and Drug Administration (FDA) Commissioner Scott Gottlieb and Center for Biologics Evaluation Research (CBER) Director Peter Marks on Wednesday warned that the agency will step up its enforcement efforts against companies illegally marketing stem cell therapies.   Gottlieb and Marks also said the agency will look into new ways to “delineate an efficient development path” for low-risk stem cell therapies being developed by firms that have filed investigational n...

The US Food and Drug Administration (FDA) on Thursday issued draft guidance providing recommendations to drugmakers looking to develop nicotine replacement therapy (NRT) products to help patients quit smoking.   The 19-page draft guidance comes after FDA held a public consultation on its approach to evaluating NRT products in November 2017 and a public hearing on the matter in January 2018.   FDA says the draft guidance “takes into consideration the feedback re...

The US Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER) on Wednesday released plans for what guidance is to be expected in 2019, including six final guidance documents related to gene therapies. The gene therapy guidance documents ( draft versions were published in July ) the agency expects to finalize include: Long Term Follow-Up After Administration of Human Gene Therapy Products; Guidance for Industry Chemistry, Manufacturing, an...

The European Commission on Tuesday launched a targeted consultation to provide input on a revision of its guideline on the content and format of applications for orphan drug designation, as well as the transfer of designations between sponsors.   "The comments received will be taken into account by the European Commission in the finalization of the guideline on the format and content of application for designation as orphan medicinal products and on the transfer of des...

Plans at the US Food and Drug Administration (FDA) to “modernize” the most commonly used pathway for new medical device clearances “might require new authority,” FDA leadership said on Monday. FDA Commissioner Scott Gottlieb and Jeff Shuren, director of FDA’s Center for Devices and Radiological Health (CDRH), issued a joint statement to announce 2018 progress on novel device approvals, pointing out that the breakthrough 510(k) is one of two submission types that was new...

A consensus of industry participants at a US Food and Drug Administration (FDA) webinar on Thursday pressed for metrics on the medical device priority review program run by FDA’s Center for Devices and Radiological Health (CDRH). The webinar led by Maureen Dreher, policy analyst at CDRH’s Office of Device Evaluation, underscored the need for additional transparency around the Breakthrough Device Program. This request was supported across several participants during the ...

The US Food and Drug Administration (FDA) on Tuesday finalized guidance detailing its breakthrough devices program and announced plans to bring safer medical device alternatives to market via a new safer technologies program (STeP).   “We continue to encourage device manufacturers to consider the breakthrough devices program for innovative, lifesaving devices, and, in the near future, our goal is to have STeP as an option for those devices with new advances and adaptat...

Industry group BIO recently submitted comments on a series of new draft guidance documents on gene therapy released by the US Food and Drug Administration (FDA) in July. The six guidance documents focus on developing hemophilia, rare disease and retinal disorder gene therapies, and also include one on chemistry, manufacturing and control (CMC) information, one on long term follow-up observational studies collecting data on adverse events and one on the testing of retr...

Following the Pentagon’s criticism of the US Food and Drug Administration’s (FDA) sluggishness in approving a French freeze-dried plasma product, which some have said other countries’ troops have used for years, FDA and the Department of Defense’s (DoD) Office of Health Affairs on Tuesday launched a joint program to prioritize the efficient development of such medical products. The framework for the program was established, via H.R.4374 , after DoD sought to crea...

The US Food and Drug Administration (FDA) on Tuesday released draft guidance indicating that it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases except for under certain conditions. Those conditions are if the use of the drug in the pediatric subpopulation are for a valid orphan subset, meaning "that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of th...