At the first highly attended ISPE Annual meeting since the pandemic hit, the themes that kept emerging from both sessions and attendees was convergence and collaboration. This concept reached its peak at the sessions that ended this conference, with a keynote from Peter Marks, MD/PhD, Director, CBER, FDA, the Regulatory Town Hall featuring representation from multiple authorities, and even the closing keynote delivered by Joydeep Ganguly, Sr. VP, Corporate Operations, Gilead Sciences, Inc.
Dr. Marks focused on gene therapy and its promise, showing a picture of one of the most obviously successful results for using gene therapy in treating a child with spinal muscular atrophy- a picture of the toddler running. (Children affected with SMA cannot even hold their heads up, so the image of the child with the gene corrected was quite affecting.) Dr. Marks spoke about how, for diseases with a single gene/allele as their etiology, it is possible to fix the genetic defect with current technologies, but there are several issues standing between gene therapy being a commercially viable treatment for a biotechnology company, amongst them are the lack of harmonization regarding compliance and regulatory requirements between different regulators. As a result, the promise of gene therapy is not being realized at a commercial scale.
The above conclusion was a perfect lead into the Regulatory Town Hall and the introduction of regulators from FDA, EMA, ANVISA, PMDA, the State Institute for Drug Control in the Czech Republic, and Health Canada. During this panel, discussion among panel members kept coming back to the pandemic and the regulatory convergence that happened as a result which facilitated the speed of brining needed medicines to market, but also touched on the challenges that still remain for complete harmonization. Variations in regulatory assessments for the same drug create challenges to maintain the dossier throughout the lifecycle of product and to implement post-approval changes that help prevent shortages. The panel concluded that a collaborative effort to understand the degree of divergence and impact is an important step toward improved global harmonization of control strategies.
The closing keynote presentation by Joydeep Ganguly illustrated the concept of collaboration in practice during the pandemic in developing medicines during an unprecedented public health emergency. Innovator companies that are usually competitors in “normal” times were collaborating to share manufacturing space among other assets, and even served as contract manufacturers for competitors’ products, a situation that can hardly be envisioned during “normal” times. However, the dire need for these medicines called for out-of-the-box thinking in order to speed medicines to market. In addition, the regulatory flexibilities and collaboration between regulatory authorities allowed for approval of these products, or the supplemental changes needed allowing them onto market in record time. In fact, a perfect illustration of this was shared by Joydeep with the timeline of remdesivir, a product that was in development for Ebola in January 2020 getting through safety readiness, through supply chain readiness, to engagement with regulators, to product approval for Covid in December 2020. It was a truly impressive feat and could not have occurred without herculean efforts on both the part of the regulators and industry.
There are many learnings that could be taken from the conference, and the sessions this single morning were the most impressive and informative at ISPE, highlighting the efforts made by all sectors of the industry and Agency. While it was undoubtably an incredibly challenging period for our industry (and society), we have the opportunity now to use this experience to learn from, collaborate with our peers, and harmonize international regulatory expectations.
