THE 505(b)(2) PLATFORM 2021 ANNUAL RECAP

​We had a banner year in dealing with FDA/CDER.  Further, at the request of our members this year, we focused on teaching companies how to tell their stories to potential investors in multiple forums. 
 
FDA Interactivity
We had had multiple interactions with FDA and CDER.  Our principal meetings involved James Myers, Maarika Kimbrell, and Elizabeth Goldstein for discussions concerning the basics of (b)(2) filing and development issues. We also met with Dr. Kevin Bugin on the issue of decentralized clinical trials which is becoming a bigger issue as we move toward a post COVID world.  This issue can be of particular concern for 505(b)(2) development because these applications often deal with drug products intended for unique, but limited, patient populations. Further this approach provides access to a greater number of patients. At our 2022 annual meeting we will have more discussion on this issue.
 
Meetings and Symposia 
At the annual meeting, which was held virtually in July, we had invaluable presentations on telling your story.   The first was by the head of FDA’s Office of (b)(2)’s, James Myers, who discussed FDA’s applications and need for application comprehensiveness.  That was followed by presentations from very successful 505(b)(2) entrepreneurs, and investors, who discussed how to make money in the (b)(2) space.  That program was attended by over 200 high level pharma executives. 
The success of that event led us to the ambitious creation and launch of Value Added Medicines Week, late October which was a weeklong virtual event for people in this spaces.  It was co-sponsored with SAAMNow, the association of scientists and regulatory professionals in this space with whom we have worked closely for several years. We firmly established the event as the premier conference on all things current on 505(b)(2)’s and highlighted critical (b)(2) issues.
The weeklong event had the unique concept of independent commercial and regulatory tracks.  It was extremely well received. 
In our “Regulatory with a Commercial Mindset session” FDA panelist, Elizabeth Goldstein, provided a tutorial on the issues that have hindered FDA review and approval of (b)(2)’s in recent years.
Marketing approved (b)(2)s, and fund raising were the intertwined next commercial discussions. Comments found the candor of the sessions invaluable.
Investors in this space have certain basic assumptions of 505(b)(2)’s:

1. >70% of Annual NDA filings are (b)(2)’s and they have a >60% Approval Rate
2. Potential market exclusivity of 3, 5, or 7 years – the more the better
3. Strong IP Protection
4. Voracious Generic Competition will occur
5. Avaricious Payers

With these points as the foundational mindset, our panelists distilled the real issues facing companies as follows:

1. How do you make money for your investors?
   

• Marketing is the first order risk. You need to focus on this from day 1. You must Add Value by Design into your product pitch
  
• The market is a potentially Large Market Channel, but it is not a Glorified Generic Market

 2.  The importance of Payers and a Market Access Strategy was emphasized.

• Time Frames must include time for Addition to Formularies
• What is your product Distribution Strategy
• Who is providing you with support in the market, policy, political arenas - Supra -Regulatory Support

For Platform members, the presentations are available on the members' site.

Summary and 2022
We continued to build rapport with CDER as the prime voice for 505(b)(2)s applicants.

1. We add value to CDER by educating on the issues facing the evolving industry and developers of (b)(2)’s.  We are helping to minimize unreviewable files, which became an even greater issue during the early stages of the pandemic.

Over 95% of the preferred pandemic solutions were (b)(2) and were not geared to generate any meaningful data
FDA had to shift the unreviewable files to the side to avoid waste of reviewing resources
Known commodities were considered credible and received more comprehensive consideration
This area is not for novices.
Grew our membership by over 400% this year.
Finally we are beginning to generate cross-border interest, particularly from Indian generic drug companies who are looking to expand beyond the deflationary generic market.
We will be circulating a questionnaire to learn issues of concern for 2022. We hope that you had a great holiday season, and join the Platform by going to 505b2.org.