Крайне любопытный текст про текущие сложности коммерциализации генной терапии на кейсе Glybera, геннотерапевтическом препарате для лечения дефицита липопротеиновой липазы, одобренном еще в 2012 году в Европе.
В двух словах: почти никто не готов платить $1 млн. даже за лечение неизлечимого другими способами и крайне редкого заболевания. Также весьма сложно проводить клинические исследования подобных терапий и сложно квантифицировать пользу от них.
Удивительно, что вообще нашелся врач, сумевший уговорить страховую компанию оплатить такое лечение - пришлось собрать целый том документов.
На подходе еще 68 генных терапий, поэтому очень важно будет разобраться в ближайшее время с финансовыми вопросами и вопросами регуляции, а компаниям - придумать более реалистичные модели коммерциализации.
The company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which weren’t geared to consider a new technology like gene therapy. Initially, for instance, regulators said they expected a clinical trial of 342 patients. Executives wryly noted that there were only 250 people with the disease in all of Europe. “The filing was a nightmare experience,” says Sander van Deventer, a biotech investor who was once the company’s chief medical officer. “They didn’t have the know-how to approve an advanced therapy.”
https://www.technologyreview.com/s/601165/the-worlds-most-expensive-medicine-is-a-bust/
P.S. Если заинтересовало - вот еще продолжение про Strimvelis от GSK https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/
В двух словах: почти никто не готов платить $1 млн. даже за лечение неизлечимого другими способами и крайне редкого заболевания. Также весьма сложно проводить клинические исследования подобных терапий и сложно квантифицировать пользу от них.
Удивительно, что вообще нашелся врач, сумевший уговорить страховую компанию оплатить такое лечение - пришлось собрать целый том документов.
На подходе еще 68 генных терапий, поэтому очень важно будет разобраться в ближайшее время с финансовыми вопросами и вопросами регуляции, а компаниям - придумать более реалистичные модели коммерциализации.
The company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which weren’t geared to consider a new technology like gene therapy. Initially, for instance, regulators said they expected a clinical trial of 342 patients. Executives wryly noted that there were only 250 people with the disease in all of Europe. “The filing was a nightmare experience,” says Sander van Deventer, a biotech investor who was once the company’s chief medical officer. “They didn’t have the know-how to approve an advanced therapy.”
https://www.technologyreview.com/s/601165/the-worlds-most-expensive-medicine-is-a-bust/
P.S. Если заинтересовало - вот еще продолжение про Strimvelis от GSK https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/
An extremely curious text about the current difficulties of commercializing gene therapy on the Glybera case, a gene therapy drug for lipoprotein lipase deficiency, approved back in 2012 in Europe.
In a nutshell: almost no one is willing to pay $ 1 million, even for the treatment of an incurable in other ways and an extremely rare disease. It is also very difficult to conduct clinical trials of such therapies and it is difficult to quantify the benefits of them.
It is surprising that in general a doctor was found who managed to persuade the insurance company to pay for such treatment - I had to collect a whole volume of documents.
Another 68 gene therapies are on the way, so it will be very important to deal with financial and regulatory issues in the near future, and companies will come up with more realistic models of commercialization.
The company spent more than $ 100 million testing the drug and carving a path through Europe’s medical rules and regulations, which weren’t geared to consider a new technology like gene therapy. Initially, for instance, regulators said they expected a clinical trial of 342 patients. Executives wryly noted that there were only 250 people with the disease in all of Europe. “The filing was a nightmare experience,” says Sander van Deventer, a biotech investor who was once the company’s chief medical officer. “They didn’t have the know-how to approve an advanced therapy.”
https://www.technologyreview.com/s/601165/the-worlds-most-expensive-medicine-is-a-bust/
P.S. If interested - here's another continuation about GSK's Strimvelis https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/
In a nutshell: almost no one is willing to pay $ 1 million, even for the treatment of an incurable in other ways and an extremely rare disease. It is also very difficult to conduct clinical trials of such therapies and it is difficult to quantify the benefits of them.
It is surprising that in general a doctor was found who managed to persuade the insurance company to pay for such treatment - I had to collect a whole volume of documents.
Another 68 gene therapies are on the way, so it will be very important to deal with financial and regulatory issues in the near future, and companies will come up with more realistic models of commercialization.
The company spent more than $ 100 million testing the drug and carving a path through Europe’s medical rules and regulations, which weren’t geared to consider a new technology like gene therapy. Initially, for instance, regulators said they expected a clinical trial of 342 patients. Executives wryly noted that there were only 250 people with the disease in all of Europe. “The filing was a nightmare experience,” says Sander van Deventer, a biotech investor who was once the company’s chief medical officer. “They didn’t have the know-how to approve an advanced therapy.”
https://www.technologyreview.com/s/601165/the-worlds-most-expensive-medicine-is-a-bust/
P.S. If interested - here's another continuation about GSK's Strimvelis https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/
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