APR March 2024 - 55
« DRUG DEVELOPMENT
Global patient data privacy regulations must align with collaborative
efforts, with local institutional review boards (IRBs) or ethics
committees ensuring ethical and legal data exchange. Effective
policies and regulatory measures are necessary, and governments
play a crucial role in implementing changes and bringing novel
drugs to the market.11
In the context of gene therapies, streamlined
commercialization, and global availability rely on supportive policies
and regulatory measures.
Governments
can institute policies
Pricing controls or negotiation of reimbursement
that prioritize affordability
and accessibility, particularly for therapies targeting underserved
populations.12
agreements can be implemented to prevent excessive costs
associated with innovative treatments. By assessing the value of a
therapy in terms of its effectiveness, patient benefits, and societal
impact, governments can contribute to keeping the cost of innovative
treatments reasonable.
Moreover, transparency in pricing structures and promoting fair
competition within the pharmaceutical industry are essential
components of effective policymaking. These measures not only
prevent monopolies but also create a competitive market environment
that contributes to affordability. Governments can implement
mechanisms to ensure that the pricing of gene therapies aligns with
the broader goal of making healthcare accessible to all.
Incentives for treatments addressing unmet medical needs can also
be incorporated into pricing policies. Extended exclusivity periods
or research grants can encourage the development of therapies that
specifically target conditions affecting marginalized or economically
disadvantaged populations. This approach fosters innovation in areas
that align with public health priorities and ensures that breakthrough
treatments reach those who need them without imposing
insurmountable financial burdens.
Global collaboration and government cooperation play a pivotal role
in the broader strategy of balancing innovation with accessibility in
the realm of gene therapies. By creating an environment that supports
both research and equitable healthcare outcomes, governments
contribute significantly to the realization of groundbreaking
treatments that positively impact the lives of individuals in need.
Conclusion
The recent FDA approvals signify a breakthrough in sickle cell
gene therapies, yet their high costs present formidable barriers to
accessibility. This article highlights the vital role of global patient
registries, stressing their importance in fostering inclusivity and
efficiently organizing diverse data. Software platforms enhance this
inclusiveness, ensuring representation across various demographics.
The call for collaboration and government cooperation is
paramount, exemplified by successful models balancing innovation
References
1.
2.
3.
4.
with affordability. Urgency surrounds the need for an accessible
healthcare landscape, urging stakeholders to unite in prioritizing
equity. Navigating
this
complex
terrain demands a collective
commitment to transformative and accessible gene therapies for all
affected communities.
FDA News Release, December 8, 2023, FDA Approves First Gene Therapies to Treat Patients
with Sickle Cell Disease
Ned Pagliarulo, BioPharma Dive, 31 Jan 2024, New CMS pilot to test payment scheme for
pricey sickle cell gene therapies.
Oktawia Borecka, et al, Future Rare Diseases, VOL. 2, NO. 4, Future Medicine, Sickle cell
disease: a global patient registry review
Albert E. Zhou, M.D., Ph.D., et al, The New England Journal of Medicine, August 11,
2022, Perspective Bringing Sickle-Cell Treatments to Children in Sub-Saharan Africa, DOI:
10.1056/NEJMp2201763
5.
Scott Askin, et al, Springer Nature - PMC COVID-19 Collection, PMID: 36923325, Artificial
Intelligence Applied to clinical trials: opportunities and challenges, Published online 2023
Feb 28. doi: 10.1007/s12553-023-00738-2
6.
Jong-Lyul Ghim, et al, Translational and Clinical Pharmacology, Transforming clinical
trials: the emerging roles of large language models, Published online 2023 Sep 19. doi:
10.12793/tcp.2023.31.e16
7.
Joseph A. DiMasi, Springer Open Choice, Therapeutic Innovation and Regulatory Science,
Assessing the Financial Value of Decentralized Clinical Trials, Published online 2022 Sep 14.
doi: 10.1007/s43441-022-00454-5
8.
Ojasav Sehrawat MBBS, Mayo Clinic Proceedings, Volume 98, Issue 9, September 2023,
Data-Driven and Technology-Enabled Trial Innovations Toward Decentralization of Clinical
Trials: Opportunities and Considerations, https://doi.org/10.1016/j.mayocp.2023.02.003
9.
10.
Monique L. Anderson, M.D, The New England Journal of Medicine, Compliance with Results
Reporting at ClinicalTrials.gov, DOI: 10.1056/NEJMsa1409364
Indo US Bridging RARE Summit 2023, Connecting the Global Rare Disease Community
11. Geoffrey Kabue Kiriiri, Future Journal of Pharmaceutical Sciences, Exploring different
approaches to improve the success of drug discovery and development projects: a review,
Published: 23 June 2020, Article number: 27 (2020)
12. Henry G. Grabowski, Medicare and Medicaid Research Review, Public Policy and
Pharmaceutical Innovation,
Author Biography
Harsha K Rajasimha, Ph.D., is the Founder and CEO of Jeeva Clinical
Trials (https://jeevatrials.com), a venture-backed startup with the mission
to modernize clinical trials, dramatically improve efficiency and their
universal accessibility with a unified software with digital clinical research
capabilities for biopharmaceutical and medical device sponsors. Harsha
is a fierce advocate for patient-focused drug development for rare and
common diseases including oncology. He chairs the annual Indo-US
bridging RARE Summit (https://summit.indousrare.org to bring the
stakeholders together to address grand challenges.
www.americanpharmaceuticalreview.com |
| 55
»
https://www.jeevatrials.com
https://summit.indousrare.org
http://www.americanpharmaceuticalreview.com
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
https://www.nxtbookmedia.com