Pharmaceutical Technology - May 2019

34 Pharmaceutical Technology BIOLOGICS AND STERILE DRUG MANUFACTURING 2019 P h a r mTe c h . c o m Manufacturing tion process that can be adapted for use in differ- ent therapeutic areas. While production decisions often focus on basic factors including geographic location, some gene and cell therapies present op- portunities for a diversified development platform with a unifying focus. The Human Genome Project and the Interna- tional HapMap Project are examples of initiatives that aim to better understand the genetic factors that are associated with many diseases. Research can lead to the development of more gene and cell therapies with the potential to expand treatment to additional indications, potentially including dis- ease states that affect large patient populations. It can be advantageous for manufacturers to expand their focus on production beyond efficiency and to include methods and technologies that may be adaptable and expandable in the future, for use in additional indications. Collaboration with stakeholder groups, especially patient communities, can help make sure that manufacturing decisions are in line, [not only] with commercialization goals [but with] factors that affect patient access and management of care. Addressing long-term safety and efficacy Limitations on data and the potential for curative efficacy requires manufacturers to put systems into place for long-term safety and efficacy moni- toring. These current limitations can have a pro- found impact on costs and commercial viability. When an FDA Advisory Committee unanimously recommended approval of Spark Therapeutics' Luxturna for treatment of inherited retinal dis- ease in October 2017 (5), they cautioned that a lack of long-term follow-up data makes it unclear whether efficacy could diminish over time. They also raised questions about the potential for future adverse events that had not been demonstrated in clinical research (6). Limitations on data can also fuel the perception that some gene and cell therapies do not provide incremental clinical value over existing therapies, making it difficult to justify their high prices. Here again, companies must plan for technologies and procedures that can meet target goals in long-term patient monitoring to avoid costs and cumbersome record keeping and other requirements that can affect commercial potential of new drugs. Engaging with stakeholder groups In part to support the collection of real-world data, manufacturers should also consider new levels of engagement with key stakeholders, potentially including healthcare providers (HCPs), payers, and clinicians. Alliance with a wide network of stakeholders spanning different geographies could provide valuable resources and facilitate long-term post-marketing surveillance efforts as well as sup- port broader understanding of the benefits and risks of gene and cell therapies. Collaboration with stakeholder groups, espe- cially patient communities, can also help make sure that manufacturing decisions are in line with both commercialization goals and factors that can affect patient access and management of

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