S4-2 Costa DoH Vatican
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23/01/2024
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Enrico Costa
Head of International Affairs Department – AIFA
Member of the Committee for Orphan Medicinal Products – EMA
Session 4: Experiences of conducting
medical research in low-resourced
settings under the DoH (B)
….through the lens of Sickle cell disease and the
regulatory perspective…
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Public Declaration of transparency/interests*
The view and opinions expressed are those of the individual presenter and should not be attributed
to AIFA
Interests in pharmaceutical industry NO Current
From 0 to 3
previous years
Over 3 preavious years
DIRECT INTERESTS:
1.1 Employment with a company: pharmaceutical
company in an executive role
X mandatory
1.2 Employment with a company: in a lead role in the
development of a medicinal product
X
mandatory
1.3 Employment with a company: other activities X optional
2. Consultancy for a company
X
optional
3. Strategic advisory role for a company X optional
4. Financial interests
X
optional
5. Ownership of a patent X optional
INDIRECT INTERESTS:
6. Principal investigator X optional
7. Investigator X optional
8. Grant or other funding X optional
9. Family members interests
X
optional
*Enrico Costa, in accordance with the Conflict of Interest Regulations approved by AIFA Board of Directors (Resolution n. 37 dated
13/10/2020).
N.B. I am not receiving any compensation
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Sickle cell disease, a brief introduction
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Lancet Glob Health 2014; 2: e80–89
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N Engl J Med 2011
Foetal Haemoglobin (HbF) and Hydroxyurea
Charache et al. N Engl J Med 1995
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Costa et al. (under revision)
Ø 20 years of sickle cell orphan drug development has ~12% success
rate, with highest rates when targeting early pathophysiological
steps.
Ø Failure rates for lack of efficacy were highest in late stages of drug
development when vaso-occlusive crisis was the key clinical
endpoint.
Successes and pitfalls in orphan drug development for SCD
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BMJ 2014;349:g4254
Revision of Helsinki declaration aims to prevent exploitation of study participants (BMJ 2013;347:f6401)
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Gene therapy
Provisions
participants continuing
crizanlizumab upon
completion of their EOT
visit via commercial
supply or post-trial
access
*
*
Pivotal clinical trials of medicines approved for SCD in the US and the EU
Costa et al. in submission
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The objective of this study was to investigate the views of adults with SCD, parents, and healthcare
providers on what information is required to sufficiently understand the risks and benefits of
consenting to participate in a genome editing clinical trial.
The general goal of informed consent for genome editing trials is the same as for other clinical trials:
ensuring the participant understands the aims of the trial; procedures, risks and benefits;
Understanding genome editing trials is especially complex, however, due to the nature of the
treatment and potential for misunderstanding the treatment, its goal, and its process
Ø Desired information about CRISPR genome editing
Ø Concerns about side effects of treatment
Ø Mechanism of action: How does it work?
Ø Inclusion criteria for being a study participant
Ø Impact on quality of life
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The aim is to improve access to safe, effective, affordable, and quality medical products across African
countries by creating and enabling regulatory environment.
Developing common standards and regulations, coordinating reviews of clinical trial applications,
coordinating the evaluation of medical products and pharmaceutical ingredient manufacturing sites,
and sharing information about products authorised for marketing
The entry of new products into the healthcare system can be accelerated, the costs of medicines can be
decreased, access to medicines will be increased, and that fake, substandard, and harmful products
will be crowded out.
BMJ 2023;380:p386 | doi: 10.1136/bmj.p386
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Conclusions
Globalization and complexity of clinical research in the regulatory space have
been increasing
Global movements of people: north-to-south and south-to-north trajectories
The implementation of the AMA can improve the reliability of the whole
pharmaceutical ecosystem including clinical trials, so protecting the most
vulnerable communities
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