The receipt of a 483 at the conclusion of a facility inspection notifies a company of conditions the FDA believes are in violation of the Food, Drug & Cosmetic Act. If 483s are not properly addressed, the FDA can take further enforcement action to protect the public health – including issuing Warning Letters. Warning Letters not only indicate potential risks to patients, but also bring unwanted publicity, erode patient trust, and potentially restrict the approval of future products significantly impacting revenue. Therefore, companies must know how to mount a competent response.
Monday’s public meeting on the FDA’s PDUFA VI Commitment Letter was a love-fest (mostly) – but as Theresa Mullin, Director of CDER’s Office of Strategic Programs, wisely noted, “the devil is in the details." Indeed.
The meeting was in three panels: Pre-Market Review and Post-Market Safety, Regulatory Decision Tools, and Administrative Enhancements.
A few highlights and comments.
Per the FDA’s PDUFA VI "commitment letter," the agency will face some real world deadlines to advance the use of real world evidence. But, since we’re dealing with the real world, let’s get real – guidance is unlikely until the end of 2022 at the earliest. (That's the timeline agreed to via the PDUFA VI negotiations.)
If Brexit, whither EMA?
Will Great Britain become like Norway and Iceland, attending committees such as the CHMP, where their views and votes are not of the same standing as those of members of the EU, or choose to follow a different route? There’s a lot at stake for the future of the MHRA and the British public health.
But what about the impact of removing MHRA expertise from EMA?
I’ve just returned from a cold, blustery and wet London where I attended the annual Pharma Access Leaders Forum.
But the meeting was hot.
In fact, piping hot when you consider the tectonic changes being felt in the world of healthcare technology assessment (HTA) and their implications on both patient access and innovation. And the linkages couldn’t be more profound.
Wither HTA in the EU? A key red thread through a series of potent discussions was real world evidence (aka, “outcomes data”). Head honcho HTA officials from across Europe (including England and Scotland – both still in Europe as of last report) returned again and again to the value of outcomes not just for the evolving world of Risk Sharing Agreements, but for the acceleration of reimbursement science.
Donna Godward and Dr. Janet Vargo recently presented a lunch briefing on improving medical devices due diligence.
Acquiring and selling innovative medical devices or diagnostics is standard business for small and large device companies – but doing it well is not standard business.
In this session, Donna Godward and Dr. Janet Vargo discussed the areas often overlooked in due diligence efforts that can lead to expensive or even show-stopper surprises once the acquisition is complete and later development and commercial efforts are underway. Understanding what to look for (or present) during due diligence will reduce risks and lead to a better evaluation for the transaction.
How has your company been preparing for EU MDR?
Minnie Baylor-Henry and Jon Lange discussed this topic during one of our most well attended webinars last month. If you missed the live event, you can now watch the recorded presentation below.
There is a heightened sense of urgency by all stakeholders to accelerate development of therapies for rare disease. The FDA shares this urgency, but it also must protect the public safety and follow the well-defined laws and regulations governing the process.
The challenge (or opportunity) is to develop creative approaches, which accelerate access and stay within the regulatory boundaries – or find ways to shape the boundaries. Intermediate Clinical Endpoints, or ICE, could represent one such approach.
National Public Radio’s Marketplace program reports:
The Food and Drug Administration recently said it’s going to prioritize any generic drug application when there’s currently just one manufacturer.