141
ISBT Science Series (2007) 2, 141–146
ORIGINAL PAPER 3B-S12-2
© 2007 The Author.
Journal compilation © 2007 Blackwell Publishing Ltd.
Blackwell Publishing Ltd
Regulatory oversight of cellular therapies
A. Farrugia
Office of Devices, Blood and Tissues, Therapeutic Goods Administration, PO Box100, Woden, ACT 2603, Australia
The agencies charged with the oversight of the mainstream therapeutics industry are
challenged by the need to develop appropriate models for the regulation of emerging
biological therapies. While banked tissue is subject to some level of alignment to
manufacturing standards such as Good Manufacturing Practices, many of the
traditional concepts for assuring safety, quality and efficacy have not been applied to
most of the products derived from human cells and tissues. With the products of
higher level manipulation such as tissue engineering and cell vaccines, a fit into the
paradigm for regulating devices and medicines has presented difficulties for the
regulator and regulated alike. These products have been viewed as being part of
medical practice rather than as the result of mainstream pharmaceutical manufacture.
Furthermore, their unique source makes them difficult to assess in traditional regulatory
systems based on the tenets of pharmaceutical quality control. These considerations
have led the established regulatory agencies of the developed world to develop new
regulatory paradigms for the products of transplantation practice. While a number of
concerns have driven these developments, the minimization of infectious disease risk
remains the paramount driver for introducing these regulatory systems. More than
the regulation of medicines and medical devices manufactured in traditional
pharmaceutical modes, the regulation of cell and tissue products is intimately linked
to areas of public health policy and funding. This places regulators in a challenging
position as they attempt to reconcile their roles as independent assessors with the
needs of the overall public health framework.
The role of therapeutic product regulation in
healthcare decision-making
Government regulation of the therapeutics industry remains
a surprisingly established feature of public healthcare policy.
In the face of the predominant deregulatory philosophy
underpinning much of Western governmental agendas of
the past 30 years, the oversight of the therapeutics sector
has been proven to be remarkably resilient and even the
occasional thrusts from review processes appear half-
hearted and more questioning of detail rather than broad
principles [1]. Why this should be is beyond the scope of
this review; it is likely due to a combination of various
converging factors. Although Western governments have
been deregulatory in their approach in recent times,
they have also not hesitated to utilize the supposedly
independent role of product regulators in managing other
pressures such as costs and industry protection. While in
most instances regulatory agencies are statutorily separate
from the policy and funding areas of government, the
distinction between what constitutes ‘policy’ and ‘regulation’
generates a perpetual tension within government. Regulators
are government officials, charged, like all their fellow
bureaucrats, with the core task of implementing the policy
of the government of the day and getting it re-elected. Thus,
the evidence-based decision-making that is supposed to
underpin the regulator’s brief of assuring the safety, quality
and efficacy of therapeutic products has necessarily to be
colored by the societal – and hence political – imperatives
that manifest when products of significant public interest are
under review. That this can occasionally lead to developments
that draw public ire on the regulator is generally ignored by
the public [2], and now accepted by regulatory agencies as
an occupational hazard.
Correspondence: Albert Farrugia, Office of Devices, Blood and Tissues,
Therapeutic Goods Administration, PO Box 100, Woden, ACT 2603, Australia
E-mail: albert.farrugia@health.gov.au