Some words have the ability to induce instant and unexplained anxiety. In developing industries, such as cell therapy, one such example is regulation – of any sort.
Crudely, our negative perception of regulation can be attributed to two factors: an aversion to both ‘red tape’ and uncertainty. In reality, regulation (when appropriately devised and implemented) should represent a framework that apportions risk and benefit fairly amongst all members of society. In the case of the health care sector, regulation must equitably balance the potential benefits of product access to patients and product developers against the risk to public health of a potential adverse event. The effective management of the range of opportunities and risks conferred by increasing regulatory scope is a mission critical step in realizing the potential of the cell therapy industry 2027.
Drivers of increasing regulation in the cell therapy industry
In its short history, the cell therapy industry has had its fair share of high profile regulatory challenges, including the Bush ban on human embryonic stem cell research, the Brustle vs Greenpeace case in the EU, uncertainty concerning FDA attitudes towards what constitutes a cellular transplant versus a cell-based medicinal product and, most recently, patient deaths leading to the closure of the Xcell Centre in Germany. It’s been a rollercoaster regulatory path to industry growth.
Increased regulation is an unavoidable feature of industry growth; a consequence of the perceived risk to society posed by the industry’s operation. The position of any such threshold is also influenced by historic events, both positive and negative. Arguably, two such events that ‘bookend’ the cell therapy industry are the death of Jesse Gelsinger in the late ‘90s during a gene therapy trial, and slightly over a decade later, the landmark approval of Provenge, a cellular therapy for prostate cancer.
Industry landmarks are facilitated primarily by advances in basic science. And the greater our knowledge of fundamental science, the more there is to regulate! However, such expansions in the purview of regulation also form the basis for ‘enabling technologies’ that facilitate the implementation of and adherence to new regulations. For example, assays to elucidate therapeutic mechanisms of action.
An additional consequence of industry growth is increased collaboration with and potential encroachment on the work of established fields, within which there is an expectation to adhere to their minimum standards.
So, if we are to view regulation as inevitably simultaneous during industry growth, what aspects of regulation contribute the most to this?
Benefits of increased regulation
Foremost, increased regulation is intended to ensure patient safety, thereby enhancing the public perception of the cell therapy industry and reducing opposition to what, at times, can be a controversial pursuit. Further, an increase in the availability and quality of public records of industry activities, including resources such as clinicaltrials.gov, contributes to increased industry transparency and provides insight into the cell therapy landscape. This has the added benefit of allowing necessary resources for industry development to be readily accessible – ensuring robust and sustainable industry growth.
For example, regulatory innovations, including adaptive licensing and value based pricing, are likely to enable novel business models that are vital to the long-term commercial viability of the sector, as well as increasing its attractiveness to pharma as a partner and/or acquisition target. Finally, while industry spends a huge amount of time lobbying for clearer regulation, astute regulatory compliance also represents a key mechanism for securing competitive advantage – particularly given the continued uncertainty surrounding IP strategies in the cell therapy industry.
If you don’t like something, change it. If you can’t change it, change your attitude.” — Maya Angelou
Increasing regulatory expectations are here to stay. We are doing something that could have widespread impact on society, and regulation is a vital part of ensuring its ultimate success. One need only consider the threat of stem cell tourism to imagine what unnecessary risks a ‘regulatory vacuum’ would incur. Only if an unreasonable and unjustified regulatory burden emerges should we petition regulators for change, particularly through industry organizations such as ARM and ISCT.
We should remember that being a regulator can be an ostensibly thankless task. Like a goalkeeper in soccer, it is a job where success is measured more by negative outcomes, than rare positive ones. Let’s try to be supportive teammates to our regulators: after all, we are all playing for the same outcome.
D.A.B. is a stockholder in Translation Ventures Ltd. a company that amongst other services provides regulatory advice to clients in the cell therapy sector.
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