Drawing increasing attention in the global industry/regulator dialogue is the need for excipients that are fit-for-purpose in meeting the complex challenges of formulating, manufacturing, and delivering the new generation of medicines and making them as safe, efficacious, and patient- and distribution-friendly as possible.
In focus are: ● the limitations in the current excipient portfolio ● what is needed in excipient development to fill the shortcomings, and ● how the current regulatory processes could be altered to better foster that development.
Responding to strong stakeholder input in recent years that an independent regulatory process for the submission and review of toxicological and quality data on novel excipients would spur their development, FDA released a proposal for a pilot review program in the Federal Register in early December 2019.
The agency requested that stakeholders provide information and comments that would assist in determining whether the pilot program should be established. The informative FDA notice included an explanation of the background, what a pilot program might look like, and the questions on which stakeholder input would be particularly helpful. By the end of the comment period in early February, twenty-six different companies and organizations had responded.
The story is divided into five parts, which are being released serially:
PART I focuses on FDA’s excipient review proposal and how the respondents addressed the pressing questions for which the agency sought answers. [CLICK HERE.]
PART II explores the relevant discussions that took place at a workshop at Health Canada (HC) in Ottawa in late September, cosponsored by the International Pharmaceutical Excipients Council of the Americas (IPEC-Americas) and the Canadian Society for Pharmaceutical Sciences (CSPS). In focus are presentations given by two industry experts who have been heavily engaged on behalf of IPEC and the IQ Consortium in the dialogue with regulators on the novel excipient issues.[CLICK HERE.]
PART III continues with a review of the insights provided by an HC expert at the workshop about the formulation challenges and potential patient benefits of bringing more biologics into subcutaneous systems. [CLICK HERE.]
PART IV reviews the efforts that USP is now making to support novel/fit-for-purpose excipient development and usage involving excipient performance, naming, quality, biological safety, and monograph development. Reviewed are the discussions that took place at USP’s excipient stakeholders forum in November around the results of an influential survey USP conducted during 2019 to help assess the need for improved excipients and the impact of the current regulatory constraints on their development. [CLICK HERE.]
PART V shifts attention onto how to better understand the risks that excipients pose across the selection, manufacturing, supply chain, and product formulation/processing continuum. In focus is a collaboration between PDA and the IPEC Federation, which resulted at the end of 2019 in a report on the “formalized risk assessment of excipients.” The report draws on and fleshes out key aspects of the FDA and international regulatory guidance on the issues. Understanding better the risks is another strong catalyst for having more fit-for-purpose excipient options available for managing them. [CLICK HERE.]
[Editor’s Note: IPQ has closely tracked the global industry/regulator dialogue on novel excipients as it evolved over the past decade, and a chronological listing of the related stories is provided immediately below.]