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In Case You Missed It: Four Themes from the DIA 2016 Annual Meeting

July 7, 2016 | Jennifer Moen, PhD, RAC, Principal Clinical Research Scientist | Medical Writing Services, Regulatory Affairs, Drug Development Consulting

Did you miss the DIA 2016 Annual Meeting? If so, no worries! This post presents 4 themes that were evident throughout the conference. Please remember that these are based on my own personal DIA experience – other attendees may have come away with different messages.

Here we go…

(1) The Official Theme: A Gathering of Global Perspectives

meetingThe organizers did a superb job of making sure this theme was evident throughout the conference. From the international representation at the Plenary Session to the inaugural DIAmond session comprised of members from 8 regulatory bodies worldwide (right) – this conference clearly represented a gathering of global perspectives.

The first panel in the DIAmond session entitled “International Regulatory Convergence, Collaboration, and Cooperation” consisted of heads of the FDA (US), PMDA (Japan), and EMA (Europe) and addressed some of the challenges that global regulatory agencies face, by their own assessment:

Among the FDA, PMDA, and EMA, there were 3 challenges mentioned by all:

  • How can the regulatory and clinical trial arenas keep up not only with the quickly evolving science, but also with technology and increasingly rapid methods of communication
  • How to innovate the regulatory process to get safe, effective products to patients who need them via faster mechanisms (eg, accelerated mechanisms, Sakigake, PRIME)
  • How to successfully rely on (and be a contributor to) global collaboration

These themes were then reaffirmed by the second panel, which included 5 representatives from ICMRA (International Council of Medicines Regulatory Authorities) – a global, voluntary organization consisting of 22 countries (with WHO as observer) whose goal is to provide strategic leadership on global regulatory topics and facilitate collaboration across regulatory authorities.

Members from regulatory authorities in Canada, Ireland, Australia, the United Kingdom, and Brazil spoke about ICMRA’s 3 strategic priorities:

  1. Maintaining the integrity of global supply chain management
  2. Establishing a process for crisis management globally (eg, Zika, Ebola)
  3. Sharing results of worldwide pharmacovigilance initiatives

This is a summary of just one of the global regulatory talks at this annual meeting (and the best one I attended) but the presentations given by many other regulators provided clear evidence that the conference stayed true to its theme. Global collaboration is a must.

(2) The Importance of Global Regulatory Strategy – Plan Globally from the Start

Not only was there a global regulatory presence throughout the conference, but not surprisingly, there was also a focus on planning a global regulatory strategy from the very start of a development program.

From the accelerated approval mechanisms in place in the US (eg, breakthrough designation) to the newer European (PRIME) and Japanese (Sakigake) initiatives, presenters made it clear that it is critical for companies to take the entire global landscape into consideration at the start of development.

One talk specifically focused on the importance of understanding the global landscape when creating a target product profile (TPP) early in development. For example:

Do you need a comparator? Where should you source your product (global or locally)? Are there any ethnic sensitivities to consider? Is a pediatric plan required? Are there any accelerated pathways to utilize (eg, the new Japanese Sakigake designation can only be considered if first submitted in Japan or simultaneous with another country)? Is shelf-life a consideration (eg, storage conditions differ greatly across regions)? And many more…

Interestingly, this talk also made a subtle but important point that the focus of drug development is shifting from approval to reimbursement. Success is now often defined by achieving return on investment rather than getting a product on the market. In a separate talk, the point was made to rethink the goal from success being approval to success being access. Moral being – approval is no longer a sufficient end goal, but rather an (important) step in the process.

Regardless of your perspective, understanding the always-evolving global landscape is critical during early development, when you are defining what “success” looks like for your product.

(3) Patient Engagement is Key – Incorporate the Patients’ Voice Early in Development

Patient involvement in the drug development process was a clear theme throughout the conference.

diaThe stage was set at the keynote address, when Parent Project Muscular Dystrophy (PPMD) won an award for their contribution to health for children with Duchenne Muscular Dystrophy (DMD). Parents of children with DMD were active during the conference. At the DIAmond session, one mom asked how to get disease-specific experts involved in early stages of development. Dr. Califf stressed the importance of patient advocacy groups being involved early in the process (more on this below) and working with KOLs and physicians to promote their cause.

PPMD was one of the advocacy groups at the conference; Sjögren’s Syndrome group was another. They made their voice heard during the presentation “Protocol is a Team Sport” –when multiple perspectives were provided on how to write an effective protocol. While statisticians, sites, and pharma perspectives were given, the one heard loudest was that of the patient.

Dr. Califf, representatives at the FDA Rare Disease Town Hall, and the Sjögrens’ spokesman strongly encouraged advocacy groups to help develop disease-specific outcomes and endpoints (including validation of patient-reported outcomes [PROs]), as well as to draft guidance(s) for the disease they support.

Informed patient input on endpoints, perceptions of risk/benefit, exclusion criteria, how to make a trial more user friendly, and how to best market a trial so patients don’t feel like “guinea pigs” were all seen as areas that patients could provide insight to assist protocol developers.

In addition to the advocacy group presence, there were many presentations dedicated to PROs, but unfortunately I could not attend them all!

(4) Rare Diseases were a Common Topic

Despite the lower number of patients affected in order to be labeled a “rare” disease, discussions of rare diseases played a prominent role during the conference.

cderThe most dedicated discussion was during the FDA Rare Disease Town Hall where the 3-person panel (2 CDER members and a law associate) spoke about the challenges with development of drugs for rare diseases as well as the resulting initiatives. The speakers reiterated the point that “patient involvement in rare diseases is critical,” perhaps even more so than for other fields.

Dr. Goldsmith (Associate Director, Rare Diseases Program/OND/CDER/FDA) showed that from January 2008 to September 2015 approximately 80% of rare disease development programs used “flexible development approaches” to regulatory approval, with the remainder following traditional development. These numbers are compelling.

Also, and as discussed above, patient advocacy groups were again strongly encouraged to be proactively involved in developing (and validating) well-defined outcome measures for rare diseases. The panel also encouraged groups to gather info from physicians (pro- or retrospectively) through electronic medical records, which is especially important for diseases with no natural history data to rely on. It was agreed that there is a place for real-world data in rare disease drug development, though this remains a challenge to date.

The panel indicated that the FDA is actively recruiting subject matter experts as reviewers for rare diseases, but stated that it is not always the specific disease they need to understand but rather the regulatory review. They stressed their reliance on academics, physicians, and patient groups to elucidate the clinical manifestations of the disease and to help develop outcome measures. One of their goals is to go from “boutique reviews” to finding commonalities across rare disease submissions.

One FDA initiative to encourage patient involvement includes public meetings to hear from patients living with a given disease, its impact on their daily life, and currently available therapies. These are a part of FDA’s Patient-Focused Drug Development Initiative and result in Voice of the Patient CDER reports (fibromyalgia example here). In addition, the Drug Development Tool Qualification Program was created to encourage reusing/modifying outcome measurements. (See our post on this program here).

Overall – the themes of patient involvement and rare diseases were intricately intertwined and evident throughout the conference.

Other Interesting Takeaway Messages

While the aforementioned themes permeated multiple presentations during the conference, there were other tidbits that I took from individual presentations. Not surprisingly, since I am a medical writer, each of these have a medical writing slant.

  • Specialized outsourcing is the current trend – not only for Medical Writing (as IMPACT’s own Tim Garver presented [read his posts here and here]) but also for Regulatory Affairs, as indicated in a Regulatory Outsourcing talk. Keys to successful partnerships (regardless of service type) include cultural fit, quality, and level of experience, among others.
  • Protocol development should involve multiple inputs – including simulation-guided trial design, feasibility assessments, and inputs from sites (eg, coordinators who can advise on whether it can be executed), to vendors (when possible), KOLs (those currently or recently seeing patients), and patients themselves (would they enroll?). Site Advocacy Groups are also available for protocol review. This extensive input is particularly helpful for trials in rare diseases.
  • Maximizing content reuse through software may (?) be the next medical writing frontier – While I’m not sure I’m a believer, it was interesting to hear that the future of medical writing might include the creation of “structured content” labeled with “conditional topic tags.” The structured content could be revised in one document and automatically updated throughout all other documents that use that text. Apparently the technical writing world has been using this technique for over a decade…is the pharmaceutical arena ready? I’m not sure…but if we’re ready, there is software out there to take us there.

This conference was a great success, with much to be learned on many, many topics. I hope you’ve enjoyed reading my takeaway messages. If you have any questions, don’t hesitate to contact me! And, as always, if you ever need help with regulatory consulting or medical writing services (among other things!), please contact us!

Category: Medical Writing Services, Regulatory Affairs, Drug Development Consulting
Keywords: DIA 2016 Annual Meeting

Talks referenced in this post are listed below. Many thanks to all of the knowledgeable presenters!!

Maximize the Value of Your Product by Beginning with the End in Mind
Take Advantage of Global Expedited Pathways: Breakthrough, Sakigake, PRIME!
Plenary Session and Keynote Address: Bad Bugs, Good People, and Big, Bold Ideas
DIAmond Session: International Regulatory Convergence, Collaboration, and Cooperation
Product Management Strategies: Maximizing Content Re-Use to Meet Diverse Customer Information Needs
Innovative Regulatory Solutions: Utilizing Flexible Outsourcing Strategies
FDA Rare Disease Town Hall
DIAmond Session: Protocol is a Team Sport
Navigating Partnerships and Submissions: Best Practices for Successful Medical Writing Deliverables Involving Multiple Stakeholders

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