Hot Topics in Biotech

In what Joy Cavagnaro, PhD, DABT, RAC, president of Access BIO, and moderator of this session, described as an “eclectic session,” Hot Topics in Biotechnology is a session held every year at the annual DIA Meeting to provide attendees with the most up-to-date critical issues taking place in the biotech industry. Topics of discussion this year include new methods to improving immunogenicity, pre-IND meetings, and practical experience with REMS for a biopharmaceutical agent.

The immunogenicity problem
In her session that addressed how to measure immunogenicity, Annie DeGroot, MD, hoped “to refine concepts, and show immunogenicity is measured by T-cell responses.”

She went on to warn, “If you don’t think of immunogenicity it can create roadblocks on the path to success.”

During her presentation, DeGroot addressed problems with the old methods of epitope mapping. In the age of advancing information technology, many software companies are focusing on the creation and development of prediction tools to aid in the measurement of immunogenicity.

Despite dated methods, immunogenicity risk assessment is possible, according to DeGroot. While FDA and EMEA are asking for this type of analysis, many companies are not integrating in immunogenicity screening into their pipeline.

Pre-IND meetings
Moving along the biotech theme into the realm of communication strategies, presenter A. Seth Pauker, PhD, MPH, focused on the importance of pre-IND meetings and when they are necessary.

Since FDA reviewers are not rewarded for pre-IND discussions under PDUFA and the FDAAA of 2007 imposes significant new FDA program burdens that have increased workload demands, Pauker insists, “put yourself in the reviewers shoes. If you can address issues upfront, you can take them off the table.”

“From my experience, I’ve seen people have a problem anticipating and defusing potential FDA questions,” he added.

When is it worth having a meeting? Pauker suggests sponsors should perform gap and risk–benefit analysis if:

• Developing a novel/emerging technology, product, target or indication

• There is deviation from guidance or precedents

• There are potential clinical hold issues

“My advice, don’t do show and tell, no one has time for it and regulatory agencies don’t care,” Pauker offered.

Risk evaluation and mitigation strategies
Rekha Garg, MD, executive director of risk intervention strategy and communication for Amgen, Inc., discussed the internal and external methods of communication when implementing a REMS.

Not all REMS are equal, they’re specific to product profiles, according to Garg. While they may not all be equal, they do all require a certain level of attention in terms of a communication strategy.

Among the steps in developing a communication plan for external stakeholders are letters to health care providers explaining the requirements of the REMS, encouraging providers to implement the REMS itself, and disseminating information to professional societies.

REMS also require certain elements to ensure safety, such as educating providers with any necessary training, experience, or specific certifications and dispensing drugs to patients only in certain health care settings.

The previously mentioned steps all require communication with external parties, but REMS also calls for strategic communication with internal stakeholders. As Garg shared with the audience in the lessons learned from Amgen’s N-plate REMS, ensuring internal stakeholder commitment-no matter how small the role-is a critical step. She provided manufacturing as an example, in which the plan may be to include an information packet with the drug upon distribution, but without this being communicated, there might not be space.

She went on to add that though there currently is no draft guidance from the FDA on REMS, there should be one available soon.