Conference organized by the Nordic Committee on Bioethics in collaboration
with Centre for Legal Studies in Welfare
and Market at the University of Copenhagen.
Venue: Alf Ross auditorium
(room 9A-3-01), Faculty of Law, University of Copenhagen
Below are abstracts for each of the talks - Click on the title of a talk to see the slides. Session 1 - How are clinical
innovations/unproven methods developed and introduced?
"Ethics and Policy Surrounding Patient Access to Experimental
Therapy",
Professor Jonathan
Kimmelman, Director of Biomedical Ethics Unit,
McGill
Debates about patient access to experimental
interventions are often framed as a zero sum gain between private preferences
of patients and public interest, or between innovation and patient protection.
After describing existing and proposed regulatory mechanisms for facilitating
patients access to experimental interventions, I will offer several reasons why
the above framings are inapt, and why oversight systems should restrict access
to experimental interventions. First, early in drug development, researchers
rarely know how to use an intervention, much less whether it is safe and
effective. Second, evidence suggests that patients do not benefit by accessing
unproven interventions; if anything they are more often harmed. Third, the
evidence standing behind novel interventions is often surprisingly weak.
Last, liberalizing patient access to experimental interventions can have
damaging effects on meaningful biomedical innovation. After considering some
rejoinders to my argument, I close by suggesting that debates about access are
best understood not in terms of private vs. public gain, but rather in terms of
who should bear the burdens and costs of medical uncertainty. I suggest that
any system committed to equitable distribution of such burdens ought to be very
restrictive about liberalizing access to experimental interventions.
“Experimental oncolytic immunotherapy for individualized treatment of
cancer patients incurable with routine approaches ”, Professor Akseli
Hemminki, University of Helsinki
Oncolytic viruses have been used for treatment of
cancer for more than a century, but the finding that a major part of their
anti-tumor activity is due to activation of the immune system, is a relatively
recent one. Thus, currently these agents are viewed more as immunotherapy than
sterile oncolytic drugs. Key to this realization was experience from human
cancer patients, since laboratory models often lack the key immunological
components. The central findings from our Advanced Therapy Access Program
(ATAP), ongoing between 2007-2012, will be explained. 290 cancer patients were
treated in an individual manner under the EU Advanced Therapy directive and its
Hospital Exemption (Hemminki A, Crossing the Valley of Death with Advanced
Cancer Therapy, Nomerta publishing 2015). While robust single agent activity is
seen only in a minority of patients with most oncolytic viruses currently in
development, they are emerging as key ingredients of combination immunotherapy.
Checkpoint inhibitors have gained much attention, due to their ability to
achieve lasting responses in immunologically “hot” tumors. However, they do not
work in “cold” tumors. Oncolytic viruses are able to turn cold tumors hot,
suggesting an attractive combination with some promisining early clinical data.
“ Developing a cancer vaccine, and challenges in performing clinical
trials of experimental therapies”, Professor Steinar Aamdal, University of Oslo
UV1 is a novel Therapeutic Cancer Vaccine (TCV) for
activation of T-cells against cancer cells and cancer stem cells.
UV1 is developed by comparing immune responses from
short term survivors with long term (10 years) survivors of several cancer
types. UV1 activates T-cells to mimic the immune responses of long term
survivors with broad immune responses against telomerase.
Phase I/IIa clinical trials in androgen sensitive
metastatic prostate cancer and non small cell lung cancer respectively showed excellent
safety profile and demonstrated ability to induce specific immune responses in
a large fraction of the patienst. In a small Phase I trial with UV1 in
combination with ipilimumab in melanoma it was shown that the combination was
well tolerated and gave rapid immunresponses in more than 90 % of the patients. Currently a Phase I
trial with UV1 in combination with pembrolizumab in melanoma patients is ongoing
in the US.
“We can do better - a patient
perspective”, Anne Sofie Boldsen Salicath,
MD, patient representative.
To be diagnosed with stage 4
cancer was by far the most scary thing that has ever happened to me. The
second, was to find out the lack of ambition and flexibility that characterize
the standard treatments offered to stage 4 patients today. To see the
healthcare system settle with safe standard treatments though they have been
proven to have only weak results. By repeating what has been shown decades ago,
not to work to improve overall survival significantly. Similarly it has been
overwhelming to see the interesting scientific evidence potentially available
for treatments, at or below phase 3 level, that never reach patients due to for
instance, technicalities, lack of ambition and a rigid health care system. And
to find out that the fate of the stage 4 patients is literally in their own
hands, because the healthcare systems have given up on them.
My talk argues we need to be more ambitious, bold and maybe just getting rid of the box, instead of trying to think outside it. It's about the right to try, because to me, the alternative would be unethical.
Session 2 - What is the legal and
regulatory environment concerning clinical innovations in medicine?
"The room for clinical innovation under Swedish law ", Senior
Lecturer Lena
Wahlberg, Lund University
In this talk, I will give an introduction to the
Swedish regulatory framework pertaining to the use of unproven methods in
medicine. Specifically, I will address the room for clinical innovation in
relation to the fundamental requirement that healthcare workers shall deliver
care in accordance with “science and proven experience”. I will also briefly
comment on the ethical dimensions of this requirement.
“The regulatory challenges in regards to clinical innovation”,
Professor Mette
Hartlev, University of Copenhagen
Development of new and better products and
methods in health care is generally welcomed by both patients, health care professionals,
and society. Consequently, there is a common interest among these actors and
innovators in clinical innovation. However, innovation always implies a certain
degree of uncertainty regarding benefits and risks – and this is particularly
true in regards to radical innovation. Consequently, development and testing of
new drugs, technologies and methods in health care takes place in a landscape
of uncertainties, which calls for regulatory considerations. This paper
explores the role of regulation in regards to clinical innovation. It explores
whether (and how) regulators can stimulate and promote innovations to the
benefit of society, while at the same time mitigating the potential risks
affiliated with clinical innovation. It also discusses the regulatory
challenges facing regulators when dealing with new technologies which does not
fit into the existing legal and ethical regulatory regime. Consequently,
innovation also calls for innovative regulation.
"Regulating Risk in Surgical Innovation? A UK perspective", Dr.
Jonathan
Ives, University of Bristol
This talk focusses
specifically on ethical and legal issues associated with regulating surgical
innovation. It will use, as a case study, a recent high profile UK case (2018),
where a 69 year old man died following complications during robotic assisted
heart surgery. A brief overview of the case will be used to highlight three key
problematic areas (1) defining innovation in surgery, (2) consenting for
unknown risk and (3) the learning curve, all of which highlight particular
challenges for would-be regulators of innovative surgical practice . The talk
will conclude by proposing an eliminativist approach to thinking about surgical
innovation, and asking whether a regulatory approach that focusses on risk
profiles could have prevented this tragic case
Session 3 - What
ethical principles should guide work on emerging treatments and experimentation
in hospitals?
"Novel medical treatments, innovation, hope and headlines", Catherine
Joynson, Nuffield Council on
Bioethics
There is a spectrum of treatments that might be
considered to be experimental, from those which have never been used in humans,
to those which are used routinely but are not licensed for the condition in
question. Experimental treatments include medicines, surgery, medical devices
and implants, stem cell and gene therapies, and fertility treatments. There
are several ways in which experimental treatments can be supplied and offered
to patients within the UK legal and regulatory framework, and crowdfunding has
emerged as a way to raise funds for costly medical treatments. The Nuffield
Council on Bioethics has identified some of the ethical issues that the use of
experimental treatments can raise. These include: difficulties in assessing
efficacy and safety; ensuring fairness of access; challenges around decision making
and consent in the context of online information; potential impacts on
knowledge generation; and ensuring healthcare professionals act responsibly.
These challenges can be particularly acute when parents or guardians seek to
access experimental treatment for a child or person who lacks capacity to
consent. A core challenge is balancing the interests of patients in accessing
experimental treatments and the need to support innovation, with ensuring there
are sufficient safeguards to protect patients from potential harm(s).
"Harms and benefits in clinical innovation", Dr. Gardar
Arnason, University of
Tübingen
The use of unproven methods in clinical practice, when
proven methods have been exhausted, is variously seen as either a matter of
clinical practice, a matter of both research and clinical practice, or as being
only defensible when provided in the context of clinical research. First, I
discuss the distinction between research and clinical practice and to what
extent it matters morally. I then consider the view that the use of unproven
methods outside of clinical trials is not research and that the dominating moral
principles in this context are respect for autonomy (of both patient and
clinician) and clinical freedom. Against this view, I argue that even if
clinical innovation is not research, it should be similarly regulated, and, in
particular where the unproven method is invasive or poses significant burden or
risk to the patient, its use must be reviewed by a research ethics committee or
a similar independent, institutional body. I conclude with some thoughts on the
importance of producing regulation that not only aims to protect patients while
promoting innovation, but that is also robustly implemented and that provides
careful and trustworthy oversight mechanisms.
"Ethical principles in unproven methods – is there a need for
regulations?", Professor Ingemar Engström, Örebro University/NCBio
In this talk, two Swedish documents on
ethical aspects on unproven methods (clinical innovation) are
presented. Fundamental ethical principles that have to be taken into
account if such methods are to be used in clinical medicine will be discussed.
The reports look at ethical value conflicts that can arise when unproven
methods are used in health care. Both reports conclude that it is ethically
acceptable to provide unproven methods outside research projects under some
strictly defined preconditions. In Sweden, this area is not satisfactorily
legally regulated. A better regulation should therefore be
prioritized.