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Posted on November 23, 2014 at 10:11 PM

Paolo Bianco and colleague Douglas Sipp wrote a very
provocative and interesting piece recently published in the journal 
Nature discussing
a movement to permit stem cell medicines, among others, to be sold in the
market without the requirement to show much safety and efficacy data permitting
the market to determine safety and effectiveness of compounds. Here, patients
would basically pay to obtain products and also be research subjects. Certain
powerful groups are calling for the deregulation of clinical medicine as a
business model to bring innovative products to the marketplace. But before I
begin explaining what Bianco and Sipp discuss, we need to cover the current
system of regulatory oversight of medical products.

The Clinical
Translation Process

The current standard for market approval of drugs,
devices, biologics and other products is known as clinical translation. The
clinical translation process involves moving research from preclinical phases
to clinical phases. Preclinical research aims to provide evidence of
proof-of-principle and product safety through in vitro (e.g., cell-based assays) and in vivo (e.g., cell transplantation in disease animal model)
experiments. The idea here is that work using animals will help to ensure that
outcomes in humans will be clinically relevant and safe. However animal models
are not always the best indicators of effectiveness of interventions in humans
despite how genetically similar they are to humans. Demonstration of safety and
efficacy in preclinical research is required to persuade regulatory authorities
like the US Food and Drug Administration (FDA) to permit approval for
researchers to enter phase 1 clinical trials. The FDA classifies
investigational new drugs (INDs) into three general phases although additional
phases are also defined. Phase 1 trials are known as safety trials usually
involving a small number of healthy volunteers (~20-80 participants) in order
to assess toxicity of a compound, and its pharmacodynamics and
pharmacokinetics. In the case of stem cell research, participants will be
patients with a specific disease. While Phase 1 studies may show some signs of
improvement in patients, the goal of all phase 1 studies is to make sure the
products are safe. Phase 2 trials on the other hand aim to assess effectiveness
and associated short-term risks and side-effects and typically involve hundreds
to thousands of individuals depending on the trial. Phase 3 studies are
expanded controlled and uncontrolled studies to gain additional safety and
effectiveness data. After the successful demonstration of safety and efficacy
from phase 3 studies, the regulatory authority will grant market approval.
Phase 4 trials are post-market, which companies and researchers can perform to
gain additional information such as long-term adverse events, drug-related
morbidity and mortality, and to include previously excluded populations.

Deregulation &
the Free to Choose

Bianco and Sipp show that US think tanks are using stem
cells to promote deregulation of medical products. They point to several key
documents and actors that talk about the loosening of regulations. One such
document is the Free to Choose Medicine campaign put forward by the Heartland
Institute based in Chicago which puts forth that companies should be able to
sell drugs after small clinical trials. The Goldwater Institute in Phoenix,
Arizona calls for criminal penalties of public employees who seek to enforce
regulations on physicians who are selling investigational drugs, biological
products, and devices to terminally ill patients. Lastly, former FDA
commissioner Andrew von Eschenbach who currently heads Project FDA at the
Manhattan Institute for Policy Research in New York advocates for the selling
of regenerative medicine interventions in the market after proof-of-concept and
safety testing (Phase 1 studies).

These organizations and actors call for deregulation and
play on free market and the right to choose rhetoric, essentially making
patients pay for under-tested products while simultaneously having them as
research subjects. This is seen in the context of autologous stem cell products
where Colorado company Regenerative Sciences is battling the FDA arguing that
the agency does not have oversight over such products. Similar situations also
arose with Texas companyCelltex Therapeutics, which treated Governor Rick Perry
for back pain. While I understand that market approvals of new drugs is low – a
recent study showing that lead indications had a likelihood of approval of
15.3% (Hay et al., 2014

and that patients with serious debilitating conditions want to try novel
products even if insufficiently tested, loosening regulations is not the
answer. Bianco and Sipp call for improved business models for innovation and
increased collaboration between regulatory agencies, companies, patient
advocacy groups and other players in order to foster innovation and ensure that
safe and effective treatments are developed in a timely manner.

The Alden March Bioethics Institute offers a Master of Science in Bioethics, a Doctorate of Professional Studies in Bioethics, and Graduate Certificates in Clinical Ethics and Clinical Ethics Consultation. For more information on AMBI’s online graduate programs, please visit our website.

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