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Kellie Combs: Hi, I’m Kellie Combs, a partner in the life sciences regulatory and compliance practice in Ropes & Gray’s Washington, D.C. office. I’m joined today by Josh Oyster, a partner in our group also based in D.C., and Sarah Blankstein, a counsel based in Boston, for a special edition of our Non-binding Guidance podcast series. Today, we’re providing a preview of an exciting upcoming event: the second annual Ropes & Gray Rare Disease Forum, scheduled for May 7 in Cambridge, Massachusetts.
We held our first Rare Disease Forum last June, where we heard from Ropes & Gray and industry panelists on many legal and regulatory topics of interest in the rare disease space. Like last year, the 2024 Rare Disease Forum will feature Ropes & Gray attorneys and industry experts on a series of panels discussing key issues relevant for companies involved in rare disease drug development, marketing, and dealmaking.
Josh, let’s start with you: Why a forum on rare disease?
Josh Oyster: Happy to jump in, Kellie. There are a number of reasons to focus on rare diseases, but first and foremost, rare disease drugs and biologics make up a huge proportion of new product development. In 2023, orphan drugs made up the majority of novel approvals by both CBER, FDA’s biologics center, and CDER, FDA’s drug center. Twelve of the 17 biologics approved by the CBER in 2023—or 71%—were orphan drugs, and 28 of CDER’s 55 novel approvals—that’s a little over half, at 51%—were orphan drugs.
More to the point: the newest and most cutting-edge science and technology, development strategies, and creative approaches to pricing, market access and commercialization are really concentrated in rare diseases. And, that’s no secret to the FDA either. FDA has been quite active when it comes to policy development and program creation for rare disease products, and 2024 promises to bring even more.
So, we will certainly have lots to discuss at this year’s Forum.
Sarah, can you tell us about some of the new developments we can expect to hear about at this year’s Rare Disease Forum?
Sarah Blankstein: Of course. I’m really excited for this year’s panels and discussions. Like last year, we will be hosting a pre-Forum introductory workshop. The goal of that workshop is to provide a high-level overview of the regulatory framework relevant to rare disease products in the U.S. We’ll cover topics like orphan designation, expedited pathways, product promotion and disease awareness communications, and fraud and abuse. We hope that this workshop will give attendees who are interested a primer so that they can get the most out of the rest of the Forum. This year, we’re also including in the workshop a discussion of some of the newer rare disease-focused programs and initiatives at FDA, things like the Support for Clinical Trials Advancing Rare Disease Therapeutics (“START”) pilot program.
After the morning workshop, we’ll have lunch and then a fireside chat with Pamela Gavin, the Executive Vice President of the National Organization for Rare Disorders. Following that fireside chat, we have five panels planned for the afternoon. There’s a panel on clinical trial issues relevant to rare disease products that I think will be really interesting. For those who were at last year’s Forum, we talked about clinical trial issues on one of the panels last year as well, but we’ll cover new and emerging topics this year, and that includes decentralized clinical trials, recruitment issues, clinical trial diversity (where, of course, we’re waiting for some FDA guidance on diversity action plans), considerations for demonstrating effectiveness, and research misconduct and data integrity issues. There’s a lot that has been happening at the FDA on these topics just in the last several months. Among other things, FDA has put out new draft and final guidances on rare disease product development, using a single adequate and well-controlled trial with confirmatory evidence to support approval, decentralized trials, externally controlled trials, master protocols, and real-world evidence. We discussed these and other developments related to rare disease product development and pricing, and what we might see in 2024, in a podcast last month.
Josh Oyster: Thanks, Sarah. I’m glad you mentioned pricing, because I know that pricing and market access present unique issues in the rare disease context, and there’s been a lot happening there as well. We’ll be having a panel at the Forum on these go-to-market issues, which I’m really looking forward to. That panel will cover both U.S. and European issues related to drug pricing and market access, as well as evolving trends in pre-market and commercial strategy. And, of course, the panel will delve into the Inflation Reduction Act and what it means for companies developing products for the U.S. market.
Elsewhere, we’ll also have a panel this year on deal trends and considerations, with a focus on recent transactions in the rare disease sector, considerations for deal structures, as well as FTC enforcement trends, which has been a hot topic in the rare disease market of late.
Now, the panels Sarah and I have been talking about so far have really been focused on things happening in the premarket phase and around the time of product approval, but what about when you have a rare disease product that’s already reimbursed and on the market? What are some of the issues you’ve seen coming up Kellie, and what will we be hearing about them at the Rare Disease Forum?
Kellie Combs: Thanks, Josh. While many of the issues that come up for companies marketing rare disease products are the same that come up for any drug or biological product, there are some unique dimensions to commercializing a product to treat rare disease. These include special compliance considerations like interactions with patients and patient groups, as well as patient support programs and sponsored testing programs, in particular, which have seen some recent government enforcement activity.
In addition to our panel at the Forum on compliance topics, we’ve added a new panel this year focused entirely on advertising and promotion issues for drugs to treat rare diseases. Here, we’ll address a variety of hot topics that I see coming up all the time in the rare disease space, including the use of patient ambassadors, disease awareness campaigns, direct-to-patient communications, and proactive scientific exchange, including FDA’s recent draft guidance on communication of scientific information on unapproved uses or the “SIUU Guidance.” We discussed the SIUU Guidance in depth in an alert and companion podcast last year, and also addressed that guidance and other ad/promo topics we expect to play out in 2024 on a podcast last month.
Turning back to the forum, though, we’ll also talk this year about some of the issues that companies face when getting a new approval—especially in cases where it’s their first product launch—and the issues that they face in standing up a commercial organization to support their product. I’m talking here about things like establishing a medical or promotional review committee, preparing and reviewing launch materials, and making sure that the necessary policies and procedures are in place.
Sarah Blankstein: Thanks, Kellie. This year’s Rare Disease Forum is shaping up to be really interesting, and a great way for lawyers as well as regulatory and compliance professionals at companies developing and marketing rare disease products to stay abreast of the latest issues in this space. And, if the panels alone aren’t enough of a draw, we’ll be capping things off with a networking reception after the Forum concludes.
Kellie Combs: Yes, that’s right, Sarah. We hope that you will join us this year; whether or not you attended last year, we really think there’s something for everyone. And there certainly have been a lot of exciting developments over the last year. We’ll end today’s podcast here—we just wanted to give everyone a quick teaser of the event and encourage our listeners to join us in Cambridge on May 7. If you’d like more information about the upcoming Rare Disease Forum, you can visit our second annual Rare Disease Forum webpage on ropesgray.com.
We want to thank all of our listeners for tuning in as well. For more information about our life sciences regulatory and compliance practice, please visit our practice page at ropesgray.com. You can listen to Non-binding Guidance and other RopesTalk podcasts through our podcast newsroom on our website, or you can subscribe wherever you listen to podcasts, including on Apple and Spotify. Thank you again for listening, and we’ll see you next time.