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Strimvelis?

vocabularies

(Erica Voss) #1

Question, @PaolaSaroufim and I were wondering how a gene therapy treatment like Strimvelis might be represented in the OMOP Vocabulary. Would we consider Strimbelis a drug exposure correct?

The treatment is personalized for each patient; hematopoietic stem cell (HSCs) are extracted from the patient and purified so that only CD34-expressing cells remain. Those cells are cultured with cytokines and growth factors and then transduced with a gammaretrovirus containing the human adenosine deaminase gene and then reinfused into the patient. These cells take root in the person’s bone marrow, replicating and creating cells that mature and create normally functioning adenosine deaminase protein, resolving the problem.[1][2][3] As of April 2016, the transduced cells had a shelf life of about six hours.[4]

From Wiki

This came up because there is an AE listed for it in the European Consortium (PROTECT), Adverse Drug Reactions Database and we were trying to map it to RxNorm for CEM.


(Christian Reich) #2

@ericaVoss:

Right now it’s device, like any other tissue or transplant. Certainly not a drug, which is a chemical with an effect on the metabolism. If there is a use case that requires that to change bring it on.


(Erica Voss) #3

Okay, I see.

I was thinking about it like other human derived exposures (e.g. 535714-Rho(D) Immune Globulin) and thought we might need to create a drug concept.


(Christian Reich) #4

Yeah. The criterion is not how it is manufactured. It is about what it is and how it exerts its effect.


(Qi Yang) #5

@Christian_Reich

I see some inconsistency in the domain mapping of gene/cellular therapy. For example:

Luxturna currently belongs to the Drug domain

Two CAR-T therapy, Kymriah is in Drug domain and Yescarta is in both Drug and Device.


(Christian Reich) #6

Yeah. I am aware. We need to make a list of them all and fix the issue accordingly. Can you start?


(Qi Yang) #7

@Christian_Reich

Sure. I will start.


(Terrie Reed) #8

The definition of whether a medical product is a drug, device, biologic depends upon whether you are considering from a regulatory point of view.
Strimvelis was approved by the European Medicines Agency - see https://ec.europa.eu/health/sites/health/files/files/paediatrics/2016_annual_report.pdf p. 9
The consequences of how a product is regulated can affect requirements on health record documentation, reporting adverse events, identifying product, which database is authoritative etc.


(Christian Reich) #9

@trsmithin:

I understand all that. But the CDM follows the use cases, not how the data come about, or how governments regulate them. If the device doesn’t work we need a use case where you can derive that you need the typical drug-related data, like ingredient, dose form, strength, packaging, route of administration.

Do you?


(Terrie Reed) #10

This link to the EMA approval gives recommended dose, strength and method of administration for Strimvelis. https://www.ema.europa.eu/en/documents/product-information/strimvelis-epar-product-information_en.pdf


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