APR March 2024 - 53

« DRUG DEVELOPMENT
estimated an appropriate price for Casgevy would be between $1.35
million and $2.05 million per patient.2
The prohibitive costs associated with these gene therapies pose
a formidable barrier to patient access. Recognizing the economic
ramifications of such high prices is crucial in understanding the
broader implications for individuals grappling with sickle cell disease.
The financial burden imposed by these treatments exacerbates
existing healthcare disparities, limiting access to those who stand to
benefit the most. It raises pertinent questions about the equitable
distribution of medical innovations and the imperative to navigate
economic barriers to ensure that advancements in healthcare are not
a privilege but a universal right.
In navigating this delicate intersection of medical innovation and
economic accessibility, the central thesis of our exploration comes
into focus - the indispensable role of global patient registries in
advancing research and development, especially for rare conditions
like sickle cell disease. As we delve deeper into this article, it becomes
evident that the establishment and meticulous curation of these
registries are not merely logistical necessities but ethical imperatives.
They hold the key to unraveling the complexities of gene therapies,
contributing to a better understanding of their efficacy, challenges,
and, critically, their impact on diverse patient populations. Global
patient registries emerge as catalysts for progress, bridging the gap
between groundbreaking therapies and the imperative of ensuring
they reach those who need them most.
Global Patient Registries in
Increasing Access to Gene Therapies
for Sickle Cell Disease
The landscape of gene therapies, particularly in the context of
addressing complex conditions like sickle cell disease, necessitates a
shift in how we approach research and development. Global patient
registries stand out as indispensable tools in this transformative
journey, playing a pivotal role in shaping the trajectory of gene
therapies and ensuring their efficacy on a broader scale.
At the heart of this necessity lies the fundamental requirement
for global patient registries in advancing gene therapies. These
comprehensive databases serve as reservoirs of invaluable information,
uniting patient demographics, treatment outcomes, and genetic
variations on an international scale. The global perspective they offer
is crucial, especially in the context of rare conditions like sickle cell
disease, where a more extensive and diverse dataset is imperative for
robust research and development.
The contribution
of
global patient
registries
to research and
development is profound. Their expansive datasets, comprising
information from diverse ethnicities, geographical locations, and
socio-economic backgrounds, facilitate a more comprehensive
understanding of the intricacies surrounding gene therapies. A diverse
and representative dataset is paramount in ensuring that the efficacy
and safety of these therapies are not limited to specific populations,
but rather universally applicable, thereby addressing health disparities
and ensuring equitable access.3
One of the remarkable attributes of global patient registries is their
capacity to explore disease heterogeneity. Sickle cell disease, for
instance, exhibits variations in its manifestations among different
populations. The staggering prevalence of sickle cell disease in subSaharan
Africa, where approximately 236,000 babies are born with the
condition each year, underscores the urgent need for a truly global
approach.4
This region, often associated with the highest prevalence
of the sickle cell trait, faces unique challenges in research and
development, exacerbated by linguistic diversity.
Despite the substantial burden of sickle cell disease in sub-Saharan
Africa, where the number of affected newborns exceeds that in
the United States by over 80 times, research efforts predominantly
emanate from more developed regions.
Notably, SCD has a significant presence in India, with the real incidence
and prevalence surpassing that of the United States. India has around
20 million patients living with SCD, which is approximately ten times
more SCD patients than the U.S., and yet, a disproportionate amount
of SCD research tends to concentrate in the U.S., reflecting a global
research gap that needs urgent attention.
By inclusively capturing data from a multitude of regions and
demographics, these registries enable researchers to delve into
disease heterogeneity. This exploration is invaluable in tailoring gene
therapies to be not only effective but also attuned to the diverse
genetic makeup of individuals affected by these conditions.
Global patient registries also bridge linguistic gaps, especially in
regions where English might not be the primary language. These
registries leverage software tools and artificial intelligence offering a
solution that transcends linguistic barriers and ensures accessibility for
non-English speakers.5
Illustrating the potential insights unlocked by a globally inclusive
approach, consider the scenario where a gene therapy demonstrates
high efficacy in a specific regional registry. Without a global
perspective, this success might be deemed universal, but global
patient registries might reveal that the therapy's effectiveness varies
in different populations due to genetic diversity. Such insights are
instrumental in refining and customizing gene therapies to ensure
their effectiveness across various genetic backgrounds.
Moreover, the cost-effectiveness and efficiency of global patient
registries starkly contrast to their regional counterparts. Building and
maintaining individual registries for each region can be resourceintensive
and may result in fragmented data. Global patient registries
streamline this process, providing a centralized, cost-effective solution
that avoids duplication of efforts.
The efficiency of these global databases is heightened by their ability
to facilitate collaboration and data-sharing among researchers and
institutions worldwide, fostering a collective approach to advancing
gene therapies.
www.americanpharmaceuticalreview.com |
| 53
»

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APR March 2024

Table of Contents for the Digital Edition of APR March 2024

Message from the Editor
Editorial Advisory Board
BIOPHARMACEUTICALS - Getting to GMP-Quality Biotherapeutics From Today’s Bench-Scale Continuous Manufacturing Systems: A Gap Analysis
MICROBIOLOGY - Critical Behavioral Attributes and the Application of COM-B Framework in Aseptic Processing
FORMULATION AND DEVELOPMENT - Use of AUC in AAV Analysis in a GMP Setting
FORMULATION AND DEVELOPMENT - Precision Medicine in Clinical Trials: A Statistical Perspective
An Interview with Dan Smithey, PhD President & CEO, Serán
FORMULATION AND DEVELOPMENT - Still Early Days for AI in Drug Discovery...Says Who?
QC Corner - Enhancing Material and Equipment Availability in Production Isolators
BIOPHARMACEUTICALS - Technologies for Aseptic Filling: The Choice is Clear
Vendor Viewpoint - Data Integrity and Rapid Micro Methods: Transforming to a Modern Microbiology Lab
MICROBIOLOGY - Bacterial Spore Formers in Disinfectant Efficacy Testing
Partner Perspective - Nanoparticle Technologies: Enablers for Ocular Drug Delivery
DRUG DELIVERY - Your Nails and You
DRUG DEVELOPMENT - Battling Exorbitance: High Costs in Sickle Cell Gene Therapies and the Imperative of Global Patient Registries for Equity
FORMULATION AND DEVELOPMENT - How Pharma Companies Are Solving Regulatory Challenges with AI-based Technology
Event Preview - CPHI North America
Event Preview - Excipient World 2024
P.I.N. Points
Advertiser's Index
APR March 2024 - CoverTip01
APR March 2024 - CoverTip02
APR March 2024 - Cover1
APR March 2024 - Cover2
APR March 2024 - 1
APR March 2024 - 2
APR March 2024 - 3
APR March 2024 - 4
APR March 2024 - 5
APR March 2024 - Message from the Editor
APR March 2024 - Editorial Advisory Board
APR March 2024 - BIOPHARMACEUTICALS - Getting to GMP-Quality Biotherapeutics From Today’s Bench-Scale Continuous Manufacturing Systems: A Gap Analysis
APR March 2024 - 9
APR March 2024 - 10
APR March 2024 - 11
APR March 2024 - 12
APR March 2024 - 13
APR March 2024 - MICROBIOLOGY - Critical Behavioral Attributes and the Application of COM-B Framework in Aseptic Processing
APR March 2024 - 15
APR March 2024 - 16
APR March 2024 - 17
APR March 2024 - 18
APR March 2024 - FORMULATION AND DEVELOPMENT - Use of AUC in AAV Analysis in a GMP Setting
APR March 2024 - 20
APR March 2024 - 21
APR March 2024 - 22
APR March 2024 - 23
APR March 2024 - FORMULATION AND DEVELOPMENT - Precision Medicine in Clinical Trials: A Statistical Perspective
APR March 2024 - 25
APR March 2024 - 26
APR March 2024 - 27
APR March 2024 - An Interview with Dan Smithey, PhD President & CEO, Serán
APR March 2024 - 29
APR March 2024 - 30
APR March 2024 - FORMULATION AND DEVELOPMENT - Still Early Days for AI in Drug Discovery...Says Who?
APR March 2024 - 32
APR March 2024 - 33
APR March 2024 - QC Corner - Enhancing Material and Equipment Availability in Production Isolators
APR March 2024 - 35
APR March 2024 - BIOPHARMACEUTICALS - Technologies for Aseptic Filling: The Choice is Clear
APR March 2024 - 37
APR March 2024 - 38
APR March 2024 - 39
APR March 2024 - Vendor Viewpoint - Data Integrity and Rapid Micro Methods: Transforming to a Modern Microbiology Lab
APR March 2024 - 41
APR March 2024 - MICROBIOLOGY - Bacterial Spore Formers in Disinfectant Efficacy Testing
APR March 2024 - 43
APR March 2024 - Partner Perspective - Nanoparticle Technologies: Enablers for Ocular Drug Delivery
APR March 2024 - 45
APR March 2024 - 46
APR March 2024 - 47
APR March 2024 - DRUG DELIVERY - Your Nails and You
APR March 2024 - 49
APR March 2024 - 50
APR March 2024 - 51
APR March 2024 - DRUG DEVELOPMENT - Battling Exorbitance: High Costs in Sickle Cell Gene Therapies and the Imperative of Global Patient Registries for Equity
APR March 2024 - 53
APR March 2024 - 54
APR March 2024 - 55
APR March 2024 - FORMULATION AND DEVELOPMENT - How Pharma Companies Are Solving Regulatory Challenges with AI-based Technology
APR March 2024 - 57
APR March 2024 - Event Preview - CPHI North America
APR March 2024 - 59
APR March 2024 - Event Preview - Excipient World 2024
APR March 2024 - 61
APR March 2024 - P.I.N. Points
APR March 2024 - 63
APR March 2024 - Advertiser's Index
APR March 2024 - Cover3
APR March 2024 - Cover4
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