YourEncore Insights - Life Sciences

Alternative Regulatory Pathways for Rare Disease: Breaking the ICE

Posted by Tim Franson, MD on 4/25/16 10:30 AM

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. 

I sat down with Dr. Joe Lamendola on April 7th to discuss this approach, and you can listen/watch the full converation below

Read More

Topics: Pharmaceutical Industry, Regulatory, Rare Disease

Transparency Isn't a One-Way Proposition

Posted by Peter J. Pitts on 4/13/16 11:00 AM

Transparency in medicine isn't a one-way street.

Read More

Topics: Life Sciences, Pharmaceutical Industry, Regulatory

FDA Gets Out In Front on Single Source Problem

Posted by Peter J. Pitts on 3/29/16 11:00 AM

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.

Here’s why:

Read More

Topics: Pharmaceutical Industry, Regulatory, FDA

FDA Advisory Committee Mistakes to Avoid

Posted by Joe Lamendola, Ph.D. on 3/9/16 10:58 AM

Advisory Committee meetings are an incredibly intense and high stakes endeavor within the drug development process. Millions in R&D costs, potential revenue, and patients served can come down to an eight hour meeting. When the FDA ultimately sides with the Advisory Committee’s recommendation approximately 85% of the time, these meetings have to go well.

Over the course of my 25+ year regulatory career, I’ve participated in numerous Advisory Committee meetings across a variety of therapeutic areas. Some went smoothly, and others didn’t. In my experience, when things don’t go well, it’s usually because of three main problems.

Read More

Topics: Life Sciences, Regulatory

FDA's Abuse-Deterrent Commissioner

Posted by Peter J. Pitts on 3/4/16 9:26 AM


Yesterday the FDA Science Board discussed the agency’s approach to opioids.

FDA presenters included agency point-man Doug Throckmorton (Deputy Director for Regulatory Programs, CDER), Janet Woodcock (Director, CDER), Sharon Hertz (Division Director, Division of Anesthesiology, Analgesia and Addiction Products), Gerald Dal Pan (Director, Office of Surveillance and Epidemiology) – and newly confirmed FDA Commissioner Rob Califf.

Califf was there at the beginning of the meeting (expected) and stayed through the entire length of the day-long affair (unexpected). An important signal that he intends to be a hands-on leader.

I was chosen to speak during the open public comment part of the hearing, and spoke about using real-world information to provide providers and patients with information beyond the limited world of pivotal trials. Here are my brief remarks:

Read More

Topics: Regulatory

From Snake Oil to Fish Oil: The Need for a New Approach to Off-Label Communication

Posted by Peter J. Pitts on 2/25/16 1:23 PM

As you well know, off label communications is on my short list of regulatory policy and public health issues that keep me up at night.  

Why?  There is a leadership vacuum on the topic, and absent clear leadership, federal judges are starting to dictate regulatory policy through the Amarin case and others.  If existing policy has evolved to protect the public from snake oil, the recent Amarin decision is precarious precedent for communications about fish oil – and beyond.   

To help find better solutions to this important issue, on February 18th the new Duke-Margolis Center for Health Policy held a conference titled, “Off Label Communication in 2016: Meeting Information Needs through New Policy Options.”

Those new options are detailed in an important new paper:
Policy Options for Off-Label Communication: Supporting Better Information, Better Evidence, and Better Care

I am honored to be one of the co-authors and to have had the opportunity to speak at the event. 

Just about every speaker pointed to the need for FDA leadership though bold action and … clarity.  The paper lays out what we refer to as Guiding Principles for Lasting Solutions. They are:

Read More

Topics: Life Sciences, Pharmaceutical Industry, Regulatory

A Biotech’s Guide to FDA’s 2016 Agenda

Posted by Peter J. Pitts on 2/17/16 10:39 AM

It is often said that the FDA loves ambiguity – because it gives the agency unlimited authority.  As a former regulator, I know that’s true. However, what’s more important to the men and women of the FDA is to be an innovation accelerator. And that means the agency has to lead through … clarity.

That wish is apparent when you study the FDA’s recently released 2016 Guidance Agenda, which outlines what the agency views as its top priorities for 2016.

The document is worth perusing in its entirety, but to save you some time, I have assembled what I view as some of the more interesting items for biotechs to watch.

Read More

Topics: Life Sciences, Pharmaceutical Industry, Quality & Safety, Regulatory

Regulatory R.A.N.T. - Biomarkers, Pediatrics, Benefit-Risk - Oh my!

Posted by Peter J. Pitts, Tim Franson M.D. on 2/11/16 11:44 AM

To protect sustainable innovation, it is vital to look beyond present conditions and develop proactive interventions for anticipated environmental changes. With development times for new biomedical medicines and devices now spanning years (and sometimes over a decade), regular audits (at least yearly) are essential in recognizing and adapting to key "climate changes."

Given the importance of both identifying new trends and their impact on the development and commercialization of every product and process, YourEncore gathered an eclectic entourage of experts with deep experience in regulatory and industry leadership after the recent RAPS meeting in Baltimore. We call these sessions R.A.N.T.s - Relevant Assessments, New Trends.  We turned the panel discussion into a white paper, full of insider insights on the changing face of biomarkers, pediatric studies and benefit-risk, all of which are integral factors in development planning that exist in a state of constant flux.

The discussion is particularly timely if you've read the FDA's recently released 2016 Guidance Agenda.  We trust you will find this material useful in preparing for the upcoming guidance season as well as informing your product/portfolio management and regulatory strategies.

Download the white paper now. 

Read More

Topics: Life Sciences, Pharmaceutical Industry, Regulatory, Rare Disease, Pharmacovigilance

FDA and NBCDs: Do Not Pass GAO

Posted by Peter J. Pitts on 1/21/16 12:15 PM

GAO Asked to Assess Viability of FDA’s Complex Generics Pathway

The FDA has begun approving generic versions of non-biologic complex drugs (NBCDs) using the same regulatory approval pathway used for small molecule drugs. However, the House Energy & Commerce Committee is asking the Government Accountability Office (GAO) to evaluate if this approach is sufficient.

Read More

Topics: Pharmaceutical Industry, Regulatory

Transparency vs. Smoke and Mirrors: Clinical Trials Debate

Posted by Peter J. Pitts on 12/22/15 9:00 AM

The debate around clinical trial transparency is certainly nothing new. But is the answer, as some call for, "more transparency and oversight?"

Read More

Topics: Pharmaceutical Industry, Expert, Regulatory