S6-2_Maria Minerva Calimag

The Emergency use authorization,
Compassionate Use and
Research ethics during health emergencies
Prof. Dr. Maria Minerva P. Calimag, M.D., M.Sc., Ph.D.
Philippine Medical Association
UST Faculty of Medicine and Surgery
Introduction
The Declaration of Helsinki, a cornerstone
document in research ethics, provides valuable
principles and guidelines for the conduct of
medical research involving human subjects.
2
Introduction
When considering emergency use authorization
(EUA) and compassionate use during health
emergencies in the context of the Declaration of
Helsinki, several ethical considerations come to
the forefront. 3
Introduction
The Declaration of Helsinki emphasizes the
protection of research participants and the
importance of balance between the welfare of
individuals and the advancement of knowledge
for the common good.
4
Emergency Use Authorization
(EUA) and Compassionate Use
The Declaration of Helsinki emphasizes the
protection of research participants and the
importance of balance between the welfare of
individuals and the advancement of knowledge
for the common good.
5
Specifically, the following principles are relevant
to EUA and compassionate use:
6
Emergency Use Authorization
(EUA) and Compassionate Use
1. Informed Consent:
1. Informed Consent: The Declaration stresses the need for
voluntary informed consent from participants. In the
context of EUA and compassionate use, where urgency may
be heightened due to health emergencies, mechanisms for
rapidly conveying essential information to patients and
families are crucial to allow for informed decision-making.
7
However, such processes must maintain ethical
rigor and provide patients with the necessary
information about risks, benefits, and available
alternatives.
8
1. Informed Consent:
2. Risks and Benefits:
2. Risks and Benefits: An essential ethical
consideration is the careful assessment of risks
and benefits associated with investigational
interventions.
9
2. Risks and Benefits:
This includes the need for continuous monitoring
and evaluation of the outcomes and potential
harms of the interventions authorized for use.
10
2. Risks and Benefits:
Transparency in communicating the available
evidence and any emerging concerns is vital to
uphold ethical standards and maintain public
trust.
11
3. Equitable Access:
3. Equitable Access: The ethical frameworks
outlined in the Declaration of Helsinki stress the
importance of fair and equitable access to
investigational therapies.
12
This principle emphasizes that vulnerable
populations should not be further marginalized
and should have the opportunity to benefit from
potentially life-saving treatments during health
emergencies.
13
3. Equitable Access:
The Declaration of Helsinki offers comprehensive
ethical principles for the conduct of research,
particularly in the context of health emergencies.
These principles are relevant to emergency use
authorization and compassionate use.
14
RESEARCH Ethics in the
Declaration of Helsinki
The primary focus on protecting the welfare of
research participants is particularly crucial in the
context of expedited pathways such as EUA and
compassionate use.
15
1. Welfare of Participants:
Prioritizing participant safety and well-being
while balancing the need for timely access to
interventions is in line with the Declaration’s
emphasis on the welfare of individuals involved
in research.
16
1. Welfare of Participants:
The Declaration highlights the importance of
independent ethical review throughout the
research process.
17
2. Independent Oversight:
This oversight becomes particularly critical
during health emergencies when the urgency to
provide access to potentially beneficial
treatments must be balanced with ethical rigor
to protect vulnerable populations.
18
2. Independent Oversight:
The Declaration’s principles emphasize the need
for continuous evaluation of ongoing studies and
interventions.
19
3. Continuous Evaluation:
This is especially relevant when considering the
dynamic nature of health emergencies and the
rapid dissemination of new information about
investigational interventions.
20
3. Continuous Evaluation:
21
22
Health technology assessment (HTA) is a
multidisciplinary process that uses systematic
and explicit methods to evaluate the
properties and effects of a health technology.
23
The Health Technology
Assessment
A health technology is conceived as any
intervention (test, device, medicine, vaccine,
procedure, program) at any point in its
lifecycle (premarket, regulatory approval,
post-market, disinvestment). 24
The Health Technology
Assessment
The HTA aim is to inform “decision-making in
order to promote an equitable, efficient, and
high-quality health system”.
25
The Health Technology
Assessment
HTA plays a crucial role in informing decision-
making, resource allocation, and policy
development during health emergencies when
expedited pathways for drug approval are
utilized.
26
The Health Technology
Assessment
Several key considerations arise in conducting
HTA for drugs under EUA and Compassionate
Use:
27
The Health Technology
Assessment
HTA aims to assess the clinical efficacy and
safety profile of drugs authorized for emergency
use.
28
1. Efficacy and Safety
Evaluation:
This includes understanding the strength of
evidence supporting the interventions, potential
risks and benefits, and the comparability of
these interventions with standard treatments or
alternatives.
29
1. Efficacy and Safety
Evaluation:
Given the urgent nature of health emergencies
and the need to provide timely access to
potentially life-saving treatments, HTA processes
for drugs under EUA and Compassionate Use
must be expedited.
30
2. Rapid Assessment:
This requires the adaptation of HTA methods to
enable rapid yet evidence-based assessments
that can inform critical healthcare decisions in a
timely manner.
31
2. Rapid Assessment:
HTA involves the collection of real-world data to
complement clinical trial results for drugs under
EUA and Compassionate Use.
32
3. Real-world Data and Post-
authorization Monitoring:
Ongoing monitoring and evaluation of patient
outcomes post-authorization are essential to
ensure the continued safety and effectiveness of
these interventions.
33
3. Real-world Data and Post-
authorization Monitoring:
The economic impact of drugs under EUA and
Compassionate Use is a vital aspect of HTA.
34
4. Cost-Effectiveness
Considerations:
Assessing the cost-effectiveness of these
interventions within the context of health
emergencies, as well as their potential long-term
economic implications, is crucial for informed
decision-making and resource allocation.
35
4. Cost-Effectiveness
Considerations:
In the HTA process for drugs under EUA and
Compassionate Use, involving a wide range of
stakeholders, including patients, healthcare providers,
policymakers, and experts, is essential to ensure that
diverse perspectives and needs are considered.
36
5. Stakeholder Engagement:
HTA should also address ethical and equity
considerations, ensuring that vulnerable populations
have access to these interventions while the
societal implications, such as potential disparities in
access and outcomes, are carefully examined.
37
6. Ethical and Equity
Considerations:
By integrating these considerations into the HTA
process, decision-makers can make informed and
ethical decisions about the authorization, use, and
allocation of drugs during health emergencies.
38
Proposal For inclusion in the
Declaration of helsinki
This proactive approach can help maximize the
clinical benefits of these interventions, minimize
potential risks, and ensure equitable access, all in
alignment with ethical principles, patient safety,
and public health objectives.
39
Proposal For inclusion in the
Declaration of helsinki
18th WMA General Assembly, Helsinki, Finland, June 1964.
29th WMA – Tokya, Japan, Oct-1975.
35th WMA – Venice, Italy, Oct-1983.
41st WMA – Hong Kong, Sep-1989
48th WMA – Somerset West, RSA, Oct-1996.
40
Development of the
Declaration of helsinki
52nd WMA – Edinburgh, Scotland, Oct-2000.
• Clarifications of Articles 29 & 30 in 2002 & 2004,
53rd & 55th WMA General Assembly.
59th WMA – Seoul, Oct-2008.
41
Development of the
Declaration of helsinki