Global Health Community (GHC)

Hello GHC Community! We're excited to announce "Wondering Wednesdays for World Health" - a new discussion board initiative to engage our community in topics and musings related to global health. Every Wednesday we'll post an article, question, or thought for consideration and we encourage you to respond with your own perspectives, questions, and content. This is a great opportunity for us to take a moment out of our week to think about these topics, but also to connect and build our network of like-minded individuals to promote engagement and drive innovation in global health.

For our very FIRST Wondering Wednesday, we wanted to share this article on The Translational Gap for Gene Therapies in Low and Middle-Income Countries: DOI: 10.1126/scitranslmed.adn1902

There's been such recent excitement in the industry, and in the news, about progress in gene therapy and the potential that these technologies have for changing the course of human health. But have we thought about what it will take to make these therapies accessible to the broader, global, population? Are we doing enough even in these early stages of development to find solutions to the barriers that the complexity and instability of these systems present for global distribution and access? Are these barriers keeping us from applying these new technologies to unmet medical needs with disease burden that predominately impacts low and middle-income countries? What do you think?

We've heard recently that there are some challenges with the AAPS Communities website - stay tuned for step-by-step instructions on how you can directly engage with the message board through your email!

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Erica Schlesinger
VP, Technical Development
Seran Biosciences, LLC
Bend OR
[email protected]

Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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This paper on Gene Therapy in LMICs is really interesting - covering so many challenges we face in making Gene Therapy available globally. My thoughts:

1. Based on costs only, I cannot justify the Gene Therapy cost per dose ($400K to $4MM) in LMIC patient populations that are already struggling with access to cheaper, well-proven, medicines. Even as the cost of Gene Therapy is expected to decrease in the next decade, I think that budgets need to be spent to get the greatest 'bang for your buck'
2. The country-specific examples helped me realize there is some potential that the cost-based argument (bullet 1) does not need to prevail. For example, if LMIC patients are enrolled in pivotal clinical trials, then those patients are an essential part of getting a new therapy approved. The example of hemophilia patients in Brazil stands-out. In this case the developer has a greater obligation to 'pay-back' to the patient population that supported the development. I have heard of some R&D programs where the enrollment of patients from LMICs is especially critical to understand the new therapy because they are treatment naive. In these cases, developers need to make the new therapies available in LMICs, regardless of the cost.  

I am interested to hear other opinions on this

Kieran Crowley

Consultant @ Pharma FastRx LLC

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Kieran Crowley Ph.D.
Owner
Pharma FasTrx LLC
Highland Park NJ
[email protected]

Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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Original Message:
Sent: 05-15-2024 11:26
From: Erica Schlesinger
Subject: Announcing: Wondering Wednesdays for World Health!

Hello GHC Community! We're excited to announce "Wondering Wednesdays for World Health" - a new discussion board initiative to engage our community in topics and musings related to global health. Every Wednesday we'll post an article, question, or thought for consideration and we encourage you to respond with your own perspectives, questions, and content. This is a great opportunity for us to take a moment out of our week to think about these topics, but also to connect and build our network of like-minded individuals to promote engagement and drive innovation in global health.

For our very FIRST Wondering Wednesday, we wanted to share this article on The Translational Gap for Gene Therapies in Low and Middle-Income Countries: DOI: 10.1126/scitranslmed.adn1902

There's been such recent excitement in the industry, and in the news, about progress in gene therapy and the potential that these technologies have for changing the course of human health. But have we thought about what it will take to make these therapies accessible to the broader, global, population? Are we doing enough even in these early stages of development to find solutions to the barriers that the complexity and instability of these systems present for global distribution and access? Are these barriers keeping us from applying these new technologies to unmet medical needs with disease burden that predominately impacts low and middle-income countries? What do you think?

We've heard recently that there are some challenges with the AAPS Communities website - stay tuned for step-by-step instructions on how you can directly engage with the message board through your email!

------------------------------
Erica Schlesinger
VP, Technical Development
Seran Biosciences, LLC
Bend OR
[email protected]

Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
------------------------------

Cost is definitely a challenge, however I think that advances in technologies and cost of goods may overcome these barriers in the future. Monoclonal antibodies used to be in the too expensive category, but with the advances made over decades of investment and more recent biosimilars, costs are coming down and they may soon be available.

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Joleen White Ph.D.
AAPS 2024 Global Health Community Chair
Bioanalytical 101 Course Development
Senior Bioassay Development Lead
Gates Medical Research Institute
Cambridge MA
[email protected]

Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
------------------------------

Original Message:
Sent: 05-15-2024 11:26
From: Erica Schlesinger
Subject: Announcing: Wondering Wednesdays for World Health!

Hello GHC Community! We're excited to announce "Wondering Wednesdays for World Health" - a new discussion board initiative to engage our community in topics and musings related to global health. Every Wednesday we'll post an article, question, or thought for consideration and we encourage you to respond with your own perspectives, questions, and content. This is a great opportunity for us to take a moment out of our week to think about these topics, but also to connect and build our network of like-minded individuals to promote engagement and drive innovation in global health.

For our very FIRST Wondering Wednesday, we wanted to share this article on The Translational Gap for Gene Therapies in Low and Middle-Income Countries: DOI: 10.1126/scitranslmed.adn1902

There's been such recent excitement in the industry, and in the news, about progress in gene therapy and the potential that these technologies have for changing the course of human health. But have we thought about what it will take to make these therapies accessible to the broader, global, population? Are we doing enough even in these early stages of development to find solutions to the barriers that the complexity and instability of these systems present for global distribution and access? Are these barriers keeping us from applying these new technologies to unmet medical needs with disease burden that predominately impacts low and middle-income countries? What do you think?

We've heard recently that there are some challenges with the AAPS Communities website - stay tuned for step-by-step instructions on how you can directly engage with the message board through your email!

------------------------------
Erica Schlesinger
VP, Technical Development
Seran Biosciences, LLC
Bend OR
[email protected]

Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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