Biopharma Excellence Launches Groundbreaking ‘Science Huddle’ Series With First Panel Debate on Statistical Significance in a ‘Small Data’ World

• The first panel discussion, on November 2nd, will tackle the theme Small data, big outcomes: strategies for achieving statistical significance when global patient populations are limited.
• Participants include the Deputy Director for Innovation Accelerator & Regulatory Science at MHRA; the CEO/Chair of Trustees at AKU Society; & the President/ CEO of Akari Therapeutics.
• They will discuss the scope of Bayesian statistics, predictive approaches, and pre-hoc analyses to overcome population challenges as biopharma becomes more narrowly targeted.
• Christian K. Schneider, Head of Biopharma Excellence at PharmaLex, and a world expert in adapting existing regulatory frameworks for biopharmaceuticals, will moderate the event.

Frankfurt, Germany - October 12th , 2022. Biopharma Excellence has launched a groundbreaking new Science Huddle panel debate series, with a high-impact first session delving into Small data, big outcomes: strategies for achieving statistical significance when global patient populations are limited.

Confirmed participants include experts in the field:

• Daniel O’Connor, Deputy Director, Innovation Accelerator and Regulatory Science, MHRA
• Nick Sireau, CEO and Chair of Trustees for the AKU Society
• Rachelle Jacques, President and CEO, Akari Therapeutics

Dr. Christian K. Schneider, Head of Biopharma Excellence and a world expert in adapting existing regulatory frameworks for biopharmaceuticals, will moderate the event.

The Science Huddle, Sparked by Biopharma Excellence, is a groundbreaking new series of quarterly thought leadership panel discussions, featuring key stakeholders from across the life sciences ecosystem, focusing on the complex challenges in the race to bring critical, cutting-edge treatments to patients.

The powerful forum will bring together senior representatives such as regulators, investors, product developers, scientists and patient advocacy groups, for frank exchanges that give rise to actionable ideas.

Commenting on the launch of the Science Huddle, and the announcement of the first panel, Head of Biopharma Excellence, Dr. Christian K. Schneider, said: “Advanced/Cell & Gene Therapies are a huge and growing focus for life science product developers, investors, regulators, patients with rare conditions and the clinicians treating them. Yet, despite huge efforts to expedite new innovation, some significant and complex challenges remain.

“It has never been more important for stakeholders from across the life sciences ecosystem to join forces and find answers to the issues threatening to delay market access to these critical treatments.

From achieving statistical significance with small clinical trial populations, to the criticality of an integrated product development strategy, to tackling the risks of empty capsids in trials of gene therapies, each Science Huddle will bring together some of the sharpest minds in their field to air the emerging issues and identify practical ways forward.”

From medical science to data science, product commercialisation and reimbursement, the Science Huddle panel series will examine challenges and devise viable and sustainable solutions across the full lifecycle of Advanced/Cell & Gene Therapy development and delivery.

Future Science Huddles, due to take place quarterly, will address:

• Proposed approaches to the practical problem and associated risks of empty capsids in gene therapy trials.
• The fallout of poor global product development planning, and the advantages of starting a dialogue with regulators much earlier in the cycle.
• The difference a more aggressive strategy can make in biosimilar delivery and market success.

Biopharma Excellence is a fusion of three scientific powerhouses, PharmaLex, ERA Consulting and Biopharma Excellence, under the PharmaLex brand. Established as a distinct entity in October 2021, the service line combines 35+ years of empirical experience and respected regulator relationships. Its global team of 70+ scientific, regulatory and commercial professionals offer strategic product development and proactive regulatory services geared specifically to developers of advanced therapy medicinal products (ATMPs).

Biopharma Excellence’s growing prominence comes as the maturation of the biopharma industry accelerates, giving commercial form to important new innovation in fields including cell and gene therapies, monoclonal antibodies (MABs), vaccines and biosimilars.