This is why federal funding is vital. Scientists and researchers need access to funding to try out different theories without the fear of failure. Many believe with federal funding cut that companies can step in, but most companies are more cautious about spending bc they want to make sure investments are worth it.
The first thing that came to mind after reading this article is that corporations have tremendous R&D (research and development) costs that need to be charged initially to keep their doors open and , although the cure may be administered to a patient with a rare disease , the corporation must be compensated accordingly for all the reasons aforementioned. That's the cold hard facts of the matter as I see it and this is where the government or philanthropy comes into play to cover these costs. To know that there is a cure but only for the super rich, is heartbreaking ,especially when a child of poor parents is involved, thus if someone can't afford to pay for the cure, the child dies of a disease that could have been cured of the ailment after the drug had been administered. In a single payer health care system , this would not be a problem as the government would pay for the procedure off of taxpayer's dollars after establishing a more equitable price for the treatment through negotiations between the producer and the government to the benefit of all concerned.
I would heartily approve of such a system in the USA.
Thanks for such a thoughtful comment! You are correct. It’s hard because the companies themselves have the R&D costs and research costs are only increasing with tariffs and inflation. Many treatments fail in trials, so the companies need to stay afloat through costs of products that do make it to market. When the drug or product has a large pool of patients the costs can be lower per patient. But for rare diseases it’s much harder because there are fewer possible users.
Typically, the US federal government and foundations are crucial in providing some of the support for the early research that is needed before it goes to an actual company for drug development. With canceled grants, and proposed changes in funding research, many of us are worried about what that means for this whole ecosystem. Companies cannot take on these early research costs on top of what they already do.
Then, once a drug is approved, your points of how we make it accessible to those who need it is so important. Some US health insurance companies DO cover zolgensma for example, but not all. And we know in the US health insurance access is not a guarantee. In the UK though, the National Health Service does cover zolgensma for all eligible children. So there are examples of systems that work for more equitable access.
This is why federal funding is vital. Scientists and researchers need access to funding to try out different theories without the fear of failure. Many believe with federal funding cut that companies can step in, but most companies are more cautious about spending bc they want to make sure investments are worth it.
Yes! Absolutely true. I worry deeply about the changes happening for this reason. Industry can’t fill this gap.
The first thing that came to mind after reading this article is that corporations have tremendous R&D (research and development) costs that need to be charged initially to keep their doors open and , although the cure may be administered to a patient with a rare disease , the corporation must be compensated accordingly for all the reasons aforementioned. That's the cold hard facts of the matter as I see it and this is where the government or philanthropy comes into play to cover these costs. To know that there is a cure but only for the super rich, is heartbreaking ,especially when a child of poor parents is involved, thus if someone can't afford to pay for the cure, the child dies of a disease that could have been cured of the ailment after the drug had been administered. In a single payer health care system , this would not be a problem as the government would pay for the procedure off of taxpayer's dollars after establishing a more equitable price for the treatment through negotiations between the producer and the government to the benefit of all concerned.
I would heartily approve of such a system in the USA.
Thanks for such a thoughtful comment! You are correct. It’s hard because the companies themselves have the R&D costs and research costs are only increasing with tariffs and inflation. Many treatments fail in trials, so the companies need to stay afloat through costs of products that do make it to market. When the drug or product has a large pool of patients the costs can be lower per patient. But for rare diseases it’s much harder because there are fewer possible users.
Typically, the US federal government and foundations are crucial in providing some of the support for the early research that is needed before it goes to an actual company for drug development. With canceled grants, and proposed changes in funding research, many of us are worried about what that means for this whole ecosystem. Companies cannot take on these early research costs on top of what they already do.
Then, once a drug is approved, your points of how we make it accessible to those who need it is so important. Some US health insurance companies DO cover zolgensma for example, but not all. And we know in the US health insurance access is not a guarantee. In the UK though, the National Health Service does cover zolgensma for all eligible children. So there are examples of systems that work for more equitable access.