EMA ODD (recap)
Hi
Currently on vacation but managed to pull this one out :)
This is related to ODD from EMA, link below go to page 27
Committee for Orphan Medicinal Products (COMP)
Minutes for the meeting on 11-13 July 2016
http://www.ema.europa.eu/docs/en_GB/document_library/Minutes/2016/09/WC500213115.pdf
I am just trying to do the math here: read this in page 27
“
2.2.17. Fimaporfin (in combination with gemcitabine) - EMA/OD/111/16
PCI Biotech AS;
Treatment of cholangiocarcinoma
COMP coordinator: Katerina Kopečková/Dinko Vitezic
The Committee agreed that the condition, cholangiocarcinoma, is a distinct medical entity and meets the criteria for orphan designation.
The intention to treat the condition with the medicinal product containing fimaporfin was considered justified based on clinical data in patients who achieved stable disease, partial responses and improved survival.
The condition is life-threatening and chronically debilitating due to biliary obstruction, late diagnosis and a median survival of less than 24 months.
The condition was estimated to be affecting less than 1.3 in 10,000 persons in the European Union, at the time the application was made.
The sponsor has also established that there exists no satisfactory method of treatment that has been authorised in the European Union for patients affected by the condition.
A positive opinion for fimaporfin, for treatment of cholangiocarcinoma, was adopted by consensus”
Assuming population in European Union is > 500 million, this gives approx 65000 people affected per year??? Why did they use 1.3 where they could have just said less than 5 per 10000 (which is a condition for ODD)
Is the market for fimachem bigger than we think in Europe, remember this is EMA, so who know better than them ???
Currently on vacation but managed to pull this one out :)
This is related to ODD from EMA, link below go to page 27
Committee for Orphan Medicinal Products (COMP)
Minutes for the meeting on 11-13 July 2016
http://www.ema.europa.eu/docs/en_GB/document_library/Minutes/2016/09/WC500213115.pdf
I am just trying to do the math here: read this in page 27
“
2.2.17. Fimaporfin (in combination with gemcitabine) - EMA/OD/111/16
PCI Biotech AS;
Treatment of cholangiocarcinoma
COMP coordinator: Katerina Kopečková/Dinko Vitezic
The Committee agreed that the condition, cholangiocarcinoma, is a distinct medical entity and meets the criteria for orphan designation.
The intention to treat the condition with the medicinal product containing fimaporfin was considered justified based on clinical data in patients who achieved stable disease, partial responses and improved survival.
The condition is life-threatening and chronically debilitating due to biliary obstruction, late diagnosis and a median survival of less than 24 months.
The condition was estimated to be affecting less than 1.3 in 10,000 persons in the European Union, at the time the application was made.
The sponsor has also established that there exists no satisfactory method of treatment that has been authorised in the European Union for patients affected by the condition.
A positive opinion for fimaporfin, for treatment of cholangiocarcinoma, was adopted by consensus”
Assuming population in European Union is > 500 million, this gives approx 65000 people affected per year??? Why did they use 1.3 where they could have just said less than 5 per 10000 (which is a condition for ODD)
Is the market for fimachem bigger than we think in Europe, remember this is EMA, so who know better than them ???
Redigert 14.08.2018 kl 00:31
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Stock DZ
13.08.2018 kl 23:32
1951
If you read in the same report about other ODD applications you can see that some are less than 0,01 or 0,1 or 1 or 3,3 ... etc per 10000 in our case is less than 1,3 per 10000. So this number must have a background behind not just arbitrary number
Redigert 13.08.2018 kl 23:33
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Stock DZ
14.08.2018 kl 00:19
1871
Seems that EMA (COMP) uses prevalence rate in ODD applications rather than incidence rate, then the numbers make sense and we should stick to pci biotech guiding. In the link below page 2 you ll find how EMA calculate/define the prevalence rate
http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2009/09/WC500003773.pdf
Prevalence is traditionally defined as the number of persons with a disease or condition at a specified instant in time in a given population. It is sometimes referred to as ‘point prevalence’ and expressed as a proportion.
The ‘prevalence criterion’, which is described in article 3 (1) (a) of Regulation (EC) No 141/2000, requires the demonstration through authoritative references that the disease or condition for which the medicinal product is intended, affects not more than 5 in 10,000 persons in the Community, when the application is made. Therefore, in the context of the orphan legislation the prevalence refers to the number of persons with the condition at the time the application is made, divided by the population of the Community at that time. In the application for designation, prevalence should be expressed as the proportion of persons affected by the condition, per 10,000. For instance, with an estimated population in the Community of 377.6 million (as of 1 January 2001) a total of 188,800 persons correspond to a prevalence of 5 in 10,000.
For the purpose of establishing the ‘prevalence criterion’, prevalence is expressed as a proportion, and the population at risk (the denominator) should always refer to the entire population of the Community even if the population at risk of the condition is just a subset of the entire general population (e.g., ovarian cancer in women, idiopathic respiratory distress syndrome in premature newborns).
For conditions of average duration of less than one year, prevalence data should be complemented with yearly incidence data (relevant to the year of submission of the application) and the sponsor should establish that the condition affected less than 5 per 10,000 persons during the year when the application was submitted.
In many situations, the true prevalence at the time of application will not be known and the demonstration of the ‘prevalence criterion’ will be based on the estimated prevalence of the condition at a certain point in time. Where this is the case, there should be reasonable evidence that the estimate provided is a good approximation of the true prevalence of the claimed orphan condition in the European Union, at the time of application.
http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2009/09/WC500003773.pdf
Prevalence is traditionally defined as the number of persons with a disease or condition at a specified instant in time in a given population. It is sometimes referred to as ‘point prevalence’ and expressed as a proportion.
The ‘prevalence criterion’, which is described in article 3 (1) (a) of Regulation (EC) No 141/2000, requires the demonstration through authoritative references that the disease or condition for which the medicinal product is intended, affects not more than 5 in 10,000 persons in the Community, when the application is made. Therefore, in the context of the orphan legislation the prevalence refers to the number of persons with the condition at the time the application is made, divided by the population of the Community at that time. In the application for designation, prevalence should be expressed as the proportion of persons affected by the condition, per 10,000. For instance, with an estimated population in the Community of 377.6 million (as of 1 January 2001) a total of 188,800 persons correspond to a prevalence of 5 in 10,000.
For the purpose of establishing the ‘prevalence criterion’, prevalence is expressed as a proportion, and the population at risk (the denominator) should always refer to the entire population of the Community even if the population at risk of the condition is just a subset of the entire general population (e.g., ovarian cancer in women, idiopathic respiratory distress syndrome in premature newborns).
For conditions of average duration of less than one year, prevalence data should be complemented with yearly incidence data (relevant to the year of submission of the application) and the sponsor should establish that the condition affected less than 5 per 10,000 persons during the year when the application was submitted.
In many situations, the true prevalence at the time of application will not be known and the demonstration of the ‘prevalence criterion’ will be based on the estimated prevalence of the condition at a certain point in time. Where this is the case, there should be reasonable evidence that the estimate provided is a good approximation of the true prevalence of the claimed orphan condition in the European Union, at the time of application.
Stock DZ
14.08.2018 kl 00:59
1824
One of the best papers about CCA with incidence rates...etc
Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA)
https://www.nature.com/articles/nrgastro.2016.51.pdf
If you don’t like pdf you can find it in this link
https://www.nature.com/articles/nrgastro.2016.51
Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA)
https://www.nature.com/articles/nrgastro.2016.51.pdf
If you don’t like pdf you can find it in this link
https://www.nature.com/articles/nrgastro.2016.51