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RAPS Turns 40 and Holds Successful Annual “Convergence” in San Jose: A Recap

October 12, 2016 | Mark A. Cierpial, PhD, RAC; Principal Regulatory Consultant | Regulatory Affairs

Congratulations to the Regulatory Affairs Professionals Society (RAPS) on turning 40 this year, and for conducting another successful annual meeting (or “Regulatory Convergence” as it’s called) at the end of last month. This year’s Convergence was focused around the theme of innovation, so the location, San Jose, CA, in the heart of Silicon Valley, was the perfect backdrop.

map RAPS’ Regulatory Convergence (#2016RAPS) 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.

This year, over 20 different health-related regulatory agencies were represented from around the world, including Argentina, Singapore, India, and Saudi Arabia. The US FDA, Europe’s EMA, and Japan’s PMDA were, of course, well represented.

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.

Keynote Address

The Convergence was kicked off by Silicon Valley author, strategic thinker, and influential blogger R “Ray” Wang, who works with Global 2000 companies on areas including innovation, business model design, engagement strategies and customer experience. His best-selling book, Disrupting Digital Business, provides insight into the digital disruption ahead and innovation required for success.

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Wang’s keynote remarks touched on the rapid development of innovation and how regulation and regulatory professionals can and must keep pace. “Disruptive” technologies that are currently challenging regulatory affairs professionals include gene editing, the use of social media and the web in regulated activities, 3D printing, and adaptive clinical trials, to name a few.

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. As you can see, some of these were similar to the themes discussed at this year’s DIA annual meeting (read our write-up of that conference here).

Update on Rare Disease and Orphan Drug Designations (US, EU, and Japan)

There was a very informative session on rare disease and orphan drug designations in the ICH regions given by Dr. Gayatri Rao, Director of the FDA’s Office of Orphan Products Development (OOPD), Dr. Sabine Haubenreisser, EMA’s Liaison Official to the FDA, and Dr. Jun Kitahara, from PMDA’s Office of International Programs. Each provided an overview of the designation process in their country or region, and discussed the latest initiatives they are implementing and challenges they are facing.

One common challenge being faced by global regulators is the impressive rise in the number of orphan drug requests being submitted. In the US, for example, which was the first region to have an orphan drug program, annual designation requests have increased more than 6-fold over the past 20 years.

This is putting a strain on OOPD review resources, which prompted Dr. Rao to recently blog that she was increasing her office’s stated goal for completing 75% of designation reviews from 90 days of receipt to 120 days of receipt. During her presentation at the meeting, Dr. Rao informed the audience that OOPD is now even struggling to meet the new 120-day goal for review.

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Dr. Rao and Dr. Haubenreisser discussed a new collaboration between the FDA and EMA aimed at improving the sharing of information on various aspects of the development and scientific evaluation of medicines for rare diseases. The new collaboration was formally announced by the agencies on September 26th, a week after the RAPS Convergence.

Among other things, the agencies will be discussing trial designs and statistical methods for small population studies, the selection and validation of trial endpoints, and how regulatory flexibility can be used to accelerate the approval of drugs for rare diseases. As we have blogged previously, there has been keen interest in the rare disease space recently, making this new collaboration significant and timely.

US Breakthrough Designation and EMA PRIority MEdicines (PRIME) Initiative

Accelerating development and approval of drugs intended to treat serious or life-threatening diseases for which there is significant unmet medical need is a priority of regulatory agencies around the world. In the US, we’ve had the Breakthrough Therapy Designation (BTD) program since 2012. In 2015, nearly a quarter of the new drugs approved by the FDA were designated as breakthrough therapies.

Dr. Haubenreisser from the EMA gave a very informative talk on the PRIority MEdicines initiative, or PRIME, which was adopted earlier this year by the European agency.

The program has similar goals as the FDA’s BTD program, and is meant to foster the development of medicines with high public health potential.

prime

Dr. Haubenreisser explained that the program is focused on the concept of “accelerated assessment.” Instead of waiting until just prior to MAA submission to determine whether a new drug qualifies for accelerated review, under PRIME, eligible candidates will be identified early in the development process and offered enhanced regulatory support through advice at key development milestones.

Products being developed by small-to-medium size entities or academic institutions can be eligible for PRIME at the proof-of-principle stage of development (Phase 1), whereas products being developed by larger entities must establish proof-of-concept (evidence of clinical response in exploratory clinical trials) before entering the PRIME scheme.

Stairways and doors leading to nowhere …
homesI did make it over to the Winchester Mystery House during my visit to San Jose. Before entering, we were admonished more than once to stay with the tour guide so as not to get lost in the labyrinth of hallways and rooms. I snapped the picture on the right of a second-floor door leading to, well, nowhere.

The regulatory labyrinth leading to product approval can be just as tricky to navigate. Get yourself an experienced tour guide and stay close … just saying … the door really did look perfectly reasonable to walk through!

Back to the Meeting … and Off-Label Promotion

Where is the line between informing physicians of a drug’s potential benefit in an off-label use and illegal promotion?

Pharmaceutical companies have argued that they have a first amendment right to disseminate information on off-label uses of their products, provided the information is not false or misleading.

The FDA’s long-standing position is that promotion of off-label uses misbrands the product and opens manufactures up to enforcement action.

Two well-done sessions explored the topic of advertising and promotion compliance, one of which focused on recent court cases regarding off-label promotion and how they may impact marketing efforts for drug manufacturer’s future campaigns.

The FDA has lost a number of high-profile cases in the area of off-label promotion, with courts siding with pharmaceutical companies such as Amarin and Vascular Solutions.

However, the FDA has not completely acquiesced on the topic and there are likely additional lawsuits to come, which puts regulatory compliance professionals in a tricky situation when it comes to advising their companies on advertising strategy.

Other regulatory/legal topics covered at sessions included physician payments under the Sunshine Act, how to avoid landmines in your FDA records and emails, dietary supplement claims, the Park Doctrine, and the Foreign Corrupt Practices Act. Too much to get into here!

RAPS New Executive Director

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This was the first Regulatory Convergence for Paul Brooks, who took over as RAPS Executive Director (ED) on September 12th (just 5 days before the meeting!)

The outgoing ED, Sherry Keramidas, retired earlier this year after leading RAPS in stellar fashion for the last 20 years. Check out her outgoing interview here.

Congratulations and best of luck Paul!

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 along the DC Waterfront in National Harbor, MD next September.

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.

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Category: Regulatory Affairs
Keywords: Regulatory Affairs, RAPS Convergence, Orphan Drug Designations, Rare Diseases, Off-Label Promotion, PRIME, Breakthrough Therapy Designation

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