Addressing Gaps in Pharmacy Standards for Cell and Gene Therapy Management

Abstract

Background and Aims

The rapid growth of FDA-approved cell and gene therapies (CGTs) has elevated the role of pharmacies in the receipt, storage, handling, and dispensing of these complex and high-value treatments. While pharmacies play a critical role in safeguarding CGT integrity and ensuring patient safety, current pharmacy quality standards and accreditations are less suited to the specialized requirements for managing CGTs. This gap underscores the urgent need for CGT-specific standards to ensure product potency and integrity, operational consistency, and patient safety.

To address this gap, a multidisciplinary committee of pharmacy stakeholders convened to develop the 1st edition of the Cell and Gene Therapy Standards for Pharmacy (see Figure 1 for committee composition and expertise). These Standards, grounded in established quality system essentials, aim to guide the safe and consistent management of CGTs across various pharmacy settings.

Methodology

To further identify existing gaps and future needs, a targeted survey of pharmacy stakeholders (n = 10) was conducted. Although the sample size was limited, the qualitative feedback provided valuable insights into current practices and areas requiring improvement (see Figure 2 for full survey results).

Results

1. Growing Role of Pharmacies:

Nearly all respondents (90% strongly agreed, 10% agreed) anticipate that pharmacies will assume an increasingly critical role in dispensing FDA-approved CGTs over the next 3–5 years. This expectation underscores the importance of readiness, training, and infrastructure support.

2. Need for Enhanced Standards:

Only half of the respondents felt that existing standards are adequate for managing CGTs, while 40% disagreed or strongly disagreed. This finding affirms the urgent need to establish and implement updated, specialized guidelines that address the unique handling, receipt, storage, and dispensing requirements of CGTs.

Conclusion

These findings reinforce the necessity of developing robust, CGT-specific standards for pharmacy practice. By proactively establishing these standards, pharmacies can better mitigate risks, uphold product integrity, and ensure patient safety—ultimately equipping themselves with tools to safeguard quality, and enhance patient outcomes.