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Challenges in the translation and commercialization of cell therapies.

Dodson BP, Levine AD - BMC Biotechnol. (2015)

Bottom Line: Cell therapies are an emerging form of healthcare that offer significant potential to improve the practice of medicine and provide benefits to patients who currently have limited or no treatment options.Ideally, these innovative therapies can complement existing small molecule, biologic and device approaches, forming a so-called fourth pillar of medicine and allowing clinicians to identify the best treatment approach for each patient.Key pre-market challenges included identifying and maintaining stable funding to see firms through lengthy developmental timelines and uncertain regulatory processes.

View Article: PubMed Central - PubMed

Affiliation: School of Public Policy, Georgia Institute of Technology, Atlanta, GA, USA. bdodson3@gatech.edu.

ABSTRACT

Background: Cell therapies are an emerging form of healthcare that offer significant potential to improve the practice of medicine and provide benefits to patients who currently have limited or no treatment options. Ideally, these innovative therapies can complement existing small molecule, biologic and device approaches, forming a so-called fourth pillar of medicine and allowing clinicians to identify the best treatment approach for each patient. Despite this potential, cell therapies are substantially more complex than small molecule or biologic interventions. This complexity poses challenges for scientists and firms developing cell therapies and regulators seeking to oversee this growing area of medicine.

Results: In this project, we retrospectively examined the development of seven cell therapies - including three autologous interventions and four allogeneic interventions - with the aim of identifying common challenges hindering attempts to bring new cell therapies to market. We complemented this analysis with a series of qualitative interviews with experts in various aspects of cell therapy. Through our analysis, which included review of extant literature collected from company documents, newspapers, journals, analyst reports and similar sources, and analysis of the qualitative interviews, we identified several common challenges that cell therapy firms must address in both the pre- and post-market stages. Key pre-market challenges included identifying and maintaining stable funding to see firms through lengthy developmental timelines and uncertain regulatory processes. These challenges are not unique to cell therapies, of course, but the novelty of cell-based interventions complicates these efforts compared to small molecule or biologic approaches. The atypical nature of cell therapies also led to post-market difficulties, including challenges navigating the reimbursement process and convincing providers to change their treatment approaches. In addition, scaling up production, distributing cell therapies and managing the costs of production were challenges that started pre-market and continued into the post-market phase.

Conclusions: Our analysis highlights several interrelated challenges hindering the development of cell therapies. Identifying strategies to address these challenges may accelerate the development and increase the impact of novel cell therapies.

No MeSH data available.


Classifications of key challenges in the development of cell therapies
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Fig2: Classifications of key challenges in the development of cell therapies

Mentions: In our analysis we found it useful to group these challenges into three broader categories (see Fig. 2). These three categories are “Pre-Market Challenges,” which covers pre-clinical and clinical research from the initial conception of the product idea up to the point that it gains market access, “Post-Market Challenges,” which covers the time from market access to the present (or the point when a product was no longer available), and “Manufacturing Challenges,” which covers issues associated with producing cells for therapeutic use and cuts across both pre- and post-market time periods. Some of the individual challenges that fall within each classification are shown in Fig. 2. We discuss each classification below and illustrate the challenges with key details from the product histories and quotes from the interviews.Fig. 2


Challenges in the translation and commercialization of cell therapies.

Dodson BP, Levine AD - BMC Biotechnol. (2015)

Classifications of key challenges in the development of cell therapies
© Copyright Policy - open-access
Related In: Results  -  Collection

License 1 - License 2
Show All Figures
getmorefigures.php?uid=PMC4528687&req=5

Fig2: Classifications of key challenges in the development of cell therapies
Mentions: In our analysis we found it useful to group these challenges into three broader categories (see Fig. 2). These three categories are “Pre-Market Challenges,” which covers pre-clinical and clinical research from the initial conception of the product idea up to the point that it gains market access, “Post-Market Challenges,” which covers the time from market access to the present (or the point when a product was no longer available), and “Manufacturing Challenges,” which covers issues associated with producing cells for therapeutic use and cuts across both pre- and post-market time periods. Some of the individual challenges that fall within each classification are shown in Fig. 2. We discuss each classification below and illustrate the challenges with key details from the product histories and quotes from the interviews.Fig. 2

Bottom Line: Cell therapies are an emerging form of healthcare that offer significant potential to improve the practice of medicine and provide benefits to patients who currently have limited or no treatment options.Ideally, these innovative therapies can complement existing small molecule, biologic and device approaches, forming a so-called fourth pillar of medicine and allowing clinicians to identify the best treatment approach for each patient.Key pre-market challenges included identifying and maintaining stable funding to see firms through lengthy developmental timelines and uncertain regulatory processes.

View Article: PubMed Central - PubMed

Affiliation: School of Public Policy, Georgia Institute of Technology, Atlanta, GA, USA. bdodson3@gatech.edu.

ABSTRACT

Background: Cell therapies are an emerging form of healthcare that offer significant potential to improve the practice of medicine and provide benefits to patients who currently have limited or no treatment options. Ideally, these innovative therapies can complement existing small molecule, biologic and device approaches, forming a so-called fourth pillar of medicine and allowing clinicians to identify the best treatment approach for each patient. Despite this potential, cell therapies are substantially more complex than small molecule or biologic interventions. This complexity poses challenges for scientists and firms developing cell therapies and regulators seeking to oversee this growing area of medicine.

Results: In this project, we retrospectively examined the development of seven cell therapies - including three autologous interventions and four allogeneic interventions - with the aim of identifying common challenges hindering attempts to bring new cell therapies to market. We complemented this analysis with a series of qualitative interviews with experts in various aspects of cell therapy. Through our analysis, which included review of extant literature collected from company documents, newspapers, journals, analyst reports and similar sources, and analysis of the qualitative interviews, we identified several common challenges that cell therapy firms must address in both the pre- and post-market stages. Key pre-market challenges included identifying and maintaining stable funding to see firms through lengthy developmental timelines and uncertain regulatory processes. These challenges are not unique to cell therapies, of course, but the novelty of cell-based interventions complicates these efforts compared to small molecule or biologic approaches. The atypical nature of cell therapies also led to post-market difficulties, including challenges navigating the reimbursement process and convincing providers to change their treatment approaches. In addition, scaling up production, distributing cell therapies and managing the costs of production were challenges that started pre-market and continued into the post-market phase.

Conclusions: Our analysis highlights several interrelated challenges hindering the development of cell therapies. Identifying strategies to address these challenges may accelerate the development and increase the impact of novel cell therapies.

No MeSH data available.