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RAPS Regulatory Convergence 2017: A Recap

October 11, 2017 | Mark A. Cierpial, PhD, RAC; Principal Regulatory Consultant | Regulatory Affairs

The Regulatory Affairs Professionals Society (RAPS) held another successful annual meeting (or “Regulatory Convergence” as it’s called) last month. This year’s Convergence was held at the Gaylord Resort at National Harbor on the DC Waterfront and offered more than 70 educational sessions presented by over 200 expert speakers from around the world. In addition, 135 companies, offering a wide range of regulatory services and solutions, were in attendance in the exhibit hall.

RAPS’ Regulatory Convergence (#2017RAPS) is the largest annual gathering of global healthcare regulatory professionals, drawing over 2000 attendees each year. Industry representatives, healthcare practitioners, regulatory service providers, and government regulators come together to share knowledge, stay informed on the ever-changing landscape of regulatory affairs, and network with friends and colleagues.

As you can see by the numbers to the right, the meeting content at RAPS Convergence meetings is typically skewed towards medical devices – particularly if you add in the in-vitro diagnostics (IVDS) sessions which get their own track. But there’s still enough for the drug and biologic folks in the crowd (like me).

And although the Convergence is a relatively large gathering, it is still small enough to foster one-on-one interactions between industry professionals and regulators representing their particular agencies. Regulators who presented at the meeting were easily accessible for questions following talks or in the halls.

You can read Dr. Gottlieb’s
entire speech here.

Plenary Session with Commissioner Gottlieb

Newly installed FDA Commissioner Scott Gottlieb, MD, was on hand to give a speech and answer questions during the Day 1 Plenary Lunch. In the early part of his speech, Dr. Gottlieb pointed to the high cost of drug development, particularly early phase development, as well as regulatory risks and uncertainties experienced by the pharmaceutical industry, as key impediments to patients being able to access new life-saving medicines. In response, he said that his Agency will be prioritizing ways to make the overall drug development and regulatory approval process more efficient.

One area that the Commissioner highlighted was the use of modeling and simulation. He noted that nearly all NDAs being submitted for new molecular entities now include components of modeling and simulation. Dr. Gottlieb touted the Agency’s use of advanced computing tools and statistical methodologies, and said they will continue to expand their expertise in this area.

When asked to comment on the spate of first amendment (commercial speech) court cases that have gone against the Agency (see our blog here), the Commissioner stated that “We are in a period of uncertainty right now” and “We need clear, legally sustainable regulations.” He vowed to continue vigorous enforcement of the regulations regarding off-label promotion as they are being interpreted by the courts.

Things to watch out for from the FDA according to Commissioner Gottlieb …

  • Easier access for clinicians to adverse event data via a new web-based system
  • At least 10 new disease-specific guidance documents this year, with input from patients and patient advocacy groups
  • Greater transparency in general, and specifically around letters issued to brand drug companies who try to impede generic drug entry by limiting the supply of their drugs for use in bioequivalence studies
  • Increased communication with all stakeholders in the drug development process (eg, patients, investigators, and IRBs), not just sponsors.

Meeting Sessions

Obviously, one can only attend a small proportion of the presentations made at a large professional meeting such as the Convergence. Below I highlight a few sessions that I found to be informative in order to give you a flavor for the conference, and a heads up to some changes that are on the way.

Impact of the 21st Century Cures Act and PDUFA VI on Regulatory Strategy

There was a very informative session on updates to US regulatory requirements based on the recent passage of the 21st Century Cures Act (Dec 2016) and the authorization of PDUFA VI via the FDA Reauthorization Act (FDARA) which was just passed by Congress in August of this year.

One of the big themes of the 21st Century Cures Act is patient-focused drug development and the use of real-world evidence (RWE) in regulatory decision making. Kim Quaintance-Lunn from Bayer US provided an informative overview of the key 21st Century Cures Act provisions, including those related to RWE. The day after the RAPS Convergence meeting ended, the Duke University Center for Health Policy, in cooperation with the FDA, held a 21st Century Cures Act mandated public workshop entitled, “Developing a Framework for Regulatory Use of Real-World Evidence.” Based on the outcome of meetings like this, FDA will be putting out guidance on the regulatory acceptability of RWE, and how it intends to use it in decision making.

Something else to watch out for … For all approved applications that are submitted after June 13, 2017, the 21st Century Cures Act requires FDA to include a brief statement regarding any patient experience data and related information that was submitted and reviewed for that application. We should start seeing these in approval packages (formally called SBAs) beginning in early 2018.

The Prescription Drug User Fee Act (PDUFA), originally passed by Congress in 1992, has been a highly successful piece of legislation. Patrick Frey, Chief of Staff for the Office of New Drugs in CDER, presented the graph at the right showing a shortening in approval times for new molecular entity NDAs filed in successive PDUFA cohorts. Mr. Frey reported that first-cycle approval rates are at an all-time high. Although fees have gone up considerably since 1992 (filing an NDA now costs nearly $2.5M), the FDA has been able to hire the staff needed to meet more frequently with pharmaceutical companies during the development phase, and review applications more quickly.

PDUFA must be reauthorized by Congress every five years. In exchange for user fees on various applications, pharmaceutical companies receive FDA commitment to review timelines, as well as other program enhancements.

Challenges for Gene Therapy Products

Another excellent session provided a global perspective on gene and cell therapy products, a very hot area these days. Europe is ahead of the US in terms of approved “advanced therapy medicinal products (ATMPs),” with 6 currently licensed (first approval in 2009). An additional 3 products were licensed between 2009 and 2017, but were subsequently withdrawn. The US FDA approved its first gene therapy product earlier this year – although it was reported that there are currently 560+ active INDs for gene therapy products.

Enrica Alteri, MD, from the European Medicines Agency (EMA) presented some rather sobering numbers regarding the use of gene therapy products after they have gained approval (see full article here). She pointed out that approval does not mean that the products will be available to patients, and if available, may not be paid for by European health systems. According to Dr. Alteri, developers of gene therapy products have found it difficult to convince health systems that they should invest into these highly expensive products, usually costing hundreds of thousands of euros per patient. For example, Glybera, a treatment for lipoprotein lipase deficiency, has only been used in one patient in the EU5 (France, Germany, UK, Italy, and Spain) since its approval in 2012.

It will be interesting to see how the US healthcare system responds to the availability of these costly medicines once more and more are approved by the FDA.

Developing a Regulatory Strategy? Don’t count on secret escape tunnels …

Last year, I commented on my excursion to the Winchester Mystery House while attending the RAPS Convergence in San Jose (you know what they say about all work and no play).

This year’s side trip was to Mount Vernon, a site I had never been to, despite being an undergrad in DC (many years ago, but still after Washington’s death). We went on the “National Treasure – Book of Secrets” tour which highlighted various film locations and how they were used, mixed in with some real history. (If you haven’t seen the movie, you can move on to the next section)

The tour of the mansion’s basement was the high point, where we saw the stone wall behind which lay the labyrinth of hidden passageways that Benjamin Gates lured the President into in order to “kidnap” him. I anxiously awaited getting to journey through the secret tunnels that George Washington had installed under the grounds of Mount Vernon, leading all the way to the Potomac river… But, alas (and spoiler alert) that was all made up for the movie. Who knew?

The tour was still pretty cool though.

Back to the Meeting … Other Things I learned

Most oncology drugs currently receive a waiver of pediatric study requirements, but this will soon be changing. If an investigational drug or biologic has a molecular target that is relevant to pediatric cancer, trials may be mandated, with no exception for orphan drugs. FDA said they would be publishing further guidance on this within the next year.

Did you know that “pre-pre-IND” meetings are an actual thing in CBER? I didn’t. These are informal meetings (not part of PDUFA) and are unique to CBER. Given the complex nature of the products regulated by CBER, the Center found that earlier consultation with sponsors was often needed. It was reported that CBER has a backlog of requests for these meetings, and it is currently taking approximately 3-4 months to get one.

An update was provided on the Breakthrough Therapy Designation program, which has exceeded the FDA’s expectations in terms of applications. FDA is granting about one-third of the requests, the majority of which are for oncology, hematology, and anti-viral products. About half of the requests are being rejected, primarily due to lack of initial clinical evidence to support the designation.

There are changes coming to FDA’s management procedures for formal meetings that all regulatory affairs professionals need to be aware of. Among other changes, FDA is defining “End-of-Phase” meetings as a separate type of Type B meeting, with different timelines for when Information Packages are due and when the meetings will be scheduled. Watch for our upcoming blog on this topic!

Is the RAPS Regulatory Convergence for you?

The entire agenda from this year’s Regulatory Convergence can be accessed here from the RAPS website. Check it out and see if this might be a useful meeting for you. If so, maybe I’ll see you in Vancouver, BC, next October.

In the meantime, IMPACT has experienced regulatory affairs professionals ready to consult with you. If you would like to discuss your specific needs, please contact us or give IMPACT a call today at (919) 899-9248.

Category: Regulatory Affairs
Keywords: Regulatory Affairs, RAPS Convergence, 21st Century Cures Act, PDUFA VI, FDARA, gene therapy, ATMP, Breakthrough Therapy Designation

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