3 ms·

Spinraza—an incredibly expensive treatment for a deadly genetic condition in infants—was available to US patients for two and a half years before it was availab

Spinraza—an incredibly expensive treatment for a deadly genetic condition in infants—was available to US patients for two and a half years before it was available to treat infants in the English NHS: https://www.reuters.com/article/idUSKCN1SK2QM/ https://www.reuters.com/article/idUSKCN1SK2QM/ Many such cases!

sofixa 3y ago
There is a difference between something being available later (probably due to regulatory reasons, and with good reason, the FDA sucks at it's job - look at Oxycontin and sunscreens for two egregious examples on the two ends of the spectrum so of course other countries' regulators wouldn't just blindly trust them), and something not being available at all because it's too expensive as was the original claim. So again, sources for medical devices or medicine not available in single payer healthcare systems because they're too expensive, but available in the US via an insurance? To expand on this, the FAA also showed itself to be criminally incompetent to the point of sacrificing human lives in service of Boeing. European (and other countries if they wish to, but as a European they're the only ones I can directly advocate for) regulators should stop trusting American regulators, and only pay attention to them to get inspiration and see what to do/what not to do. European regulators are far from perfect, but they're doing a better job and more trustworthy than their underfunded, hated by 1/3 of the country, revolving door corrupt American counterparts.
- twoodfin 3y ago
  Spinraza was specifically not covered for years because the English NHS and Biogen could not reach financial terms based on the NHS’ assessment of the value of the drug over other treatments. Look, if you don’t want to engage with this specific example, there are many more. Cerdelga to treat Gaucher’s disease was approved by the EU in 2015, but the English NHS didn’t agree to pay for it for another two years: https://pharmatimes.com/news/nice_u-turn_backs_sanofis_gaucher_disease_drug_1194651/ https://pharmatimes.com/news/nice_u-turn_backs_sanofis_gauch... Canada as well, not approved for coverage until 2017: https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00359 https://hpr-rps.hres.ca/reg-content/summary-basis-decision-d...
  - sofixa 3y ago
    Again, not being approved for 2 years, for whatever reason, doesn't match the original claim of not being approved at all because it's too expensive.
    - twoodfin 3y ago
      If you’re a parent of a child suffering from a terrible genetic condition with what is effectively a miracle cure available, does it really matter to you if perhaps, hypothetically, someday—years later—a drug your government already deems safe is made available to you? Prices aren’t static. Competitors enter the market. Governments are willing to offer more or less. Eventually, generics or biosimilars become available. It’s 100% accurate that English and Canadian citizens were denied access to these new drugs until they ceased being “too expensive” (at which point they were no longer “new”).
refurb 3y ago
Another good example is the cystic fibrosis drugs like Kalydeco. Game changing therapy that corrects the gene defect and restores near normal lung function. Available shortly after launch in the US in 2012. Even Medicaid patients (insurance for poor Americans) had access. The UK didn’t pay for it until 2020. Eight years later. And in Canada only 6 of 12 provinces/territories pay for it and only for children. If you’re an adult, well, you’re out of luck.

sofixa 3y ago

There is a difference between something being available later (probably due to regulatory reasons, and with good reason, the FDA sucks at it's job - look at Oxycontin and sunscreens for two egregious examples on the two ends of the spectrum so of course other countries' regulators wouldn't just blindly trust them), and something not being available at all because it's too expensive as was the original claim. So again, sources for medical devices or medicine not available in single payer healthcare systems because they're too expensive, but available in the US via an insurance? To expand on this, the FAA also showed itself to be criminally incompetent to the point of sacrificing human lives in service of Boeing. European (and other countries if they wish to, but as a European they're the only ones I can directly advocate for) regulators should stop trusting American regulators, and only pay attention to them to get inspiration and see what to do/what not to do. European regulators are far from perfect, but they're doing a better job and more trustworthy than their underfunded, hated by 1/3 of the country, revolving door corrupt American counterparts.

twoodfin 3y ago
Spinraza was specifically not covered for years because the English NHS and Biogen could not reach financial terms based on the NHS’ assessment of the value of the drug over other treatments. Look, if you don’t want to engage with this specific example, there are many more. Cerdelga to treat Gaucher’s disease was approved by the EU in 2015, but the English NHS didn’t agree to pay for it for another two years: https://pharmatimes.com/news/nice_u-turn_backs_sanofis_gaucher_disease_drug_1194651/ https://pharmatimes.com/news/nice_u-turn_backs_sanofis_gauch... Canada as well, not approved for coverage until 2017: https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00359 https://hpr-rps.hres.ca/reg-content/summary-basis-decision-d...
- sofixa 3y ago
  Again, not being approved for 2 years, for whatever reason, doesn't match the original claim of not being approved at all because it's too expensive.
  - twoodfin 3y ago
    If you’re a parent of a child suffering from a terrible genetic condition with what is effectively a miracle cure available, does it really matter to you if perhaps, hypothetically, someday—years later—a drug your government already deems safe is made available to you? Prices aren’t static. Competitors enter the market. Governments are willing to offer more or less. Eventually, generics or biosimilars become available. It’s 100% accurate that English and Canadian citizens were denied access to these new drugs until they ceased being “too expensive” (at which point they were no longer “new”).

refurb 3y ago

Another good example is the cystic fibrosis drugs like Kalydeco. Game changing therapy that corrects the gene defect and restores near normal lung function. Available shortly after launch in the US in 2012. Even Medicaid patients (insurance for poor Americans) had access. The UK didn’t pay for it until 2020. Eight years later. And in Canada only 6 of 12 provinces/territories pay for it and only for children. If you’re an adult, well, you’re out of luck.