Abstract
BackgroundCell 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.ResultsIn 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.ConclusionsOur 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.
Highlights
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
Many medical conditions that are not well treated today occur as a result of cellular dysfunction and cell therapies, which rely on the use of cells rather than small molecules or biologics, appear to offer potential to address many of these conditions
The use of cell therapies has been identified as a potential strategy to address a wide range of disease targets, including heart disease, diabetes, neurodegenerative diseases, musculoskeletal disorders and spinal cord injury, among others [1, 3]. While this specific list of targets may be open to Dodson and Levine BMC Biotechnology (2015) 15:70 debate, it is clear that scientists and clinical researchers have identified cell therapy as a promising therapeutic strategy to help patients with a broad range of medical conditions. This potential is observed in the growth of cell therapy clinical trials with a recent study identifying 1,925 ongoing cell therapy clinical trials as of June 2010 [4]
Summary
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 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. The use of cell therapies has been identified as a potential strategy to address a wide range of disease targets, including heart disease, diabetes, neurodegenerative diseases, musculoskeletal disorders and spinal cord injury, among others [1, 3] While this specific list of targets may be open to Dodson and Levine BMC Biotechnology (2015) 15:70 debate, it is clear that scientists and clinical researchers have identified cell therapy as a promising therapeutic strategy to help patients with a broad range of medical conditions. Another slightly more recent analysis of clinical trials, focusing on stem cell therapies, identified trials of novel cell therapies targeting a range of different conditions and concluded that cardiovascular disease, neurological disease, cancer, liver disease and bone conditions were the most common [5]
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