Regulation of Gene Therapy in Canada
Gene therapy is still a research study that is being done. In Canada, there are no regulations that are specifically directed to gene therapy but there are still general regulations for products regarding biotechnology which includes gene therapy [21]. Based on the Canadian Food and Drugs Act, the Food and Drug Regulations, and the Medical Devices Regulations, the information that is required for the product to be reviewed by Health Canada before being marketed includes:
- The pre-clinical studies that are done in test tubes (in vitro), and in animals (in vivo), that are done to assess the performance of the drug, including the toxicity and the extent of negative effects [21].
- How the product is manufactured or produced, including quality control and evidence showing consistency in the manufacturing process [21].
- The package and labelling of the product [21].
- Therapeutic or diagnostic claims [21].
- Conditions for use [21].
- A list of Potential side effects [21].
They would also need to provide at least three lots of samples of the product so that they can be tested [21]. If the review shows that the benefits of the product outweigh the risks, authorization to sell the product would be given to the manufacturer [21]. Also, in Canada, before gene therapy can be used to treat a certain genetic disorder, there are certain requirements that are needed to be met:
- The faulty gene that needs to be cured must be identified and how it results in the condition must be known so that the vector can be genetically altered for use and the appropriate cell or tissue can be targeted [8].
- The gene has to be able to be cloned so that it can be inserted into the vector [8].
- When the gene is transferred into the new cell, whether it is turned on or off needs to be controlled [8].
- There must be sufficient value in treating a disorder with gene therapy, it must be known if there is a simpler way to treat that disorder [8].
- The benefits and risks of using gene therapy for that condition must be considered favourable compared to other types of therapies [8].
- There must be enough data collected from cell and animal experiments to show that the procedure works and is safe to be used [8].
When these conditions are met, researchers may begin to start clinical trials that are closely monitored by institutional review boards and governmental agencies for safety [8].
- The pre-clinical studies that are done in test tubes (in vitro), and in animals (in vivo), that are done to assess the performance of the drug, including the toxicity and the extent of negative effects [21].
- How the product is manufactured or produced, including quality control and evidence showing consistency in the manufacturing process [21].
- The package and labelling of the product [21].
- Therapeutic or diagnostic claims [21].
- Conditions for use [21].
- A list of Potential side effects [21].
They would also need to provide at least three lots of samples of the product so that they can be tested [21]. If the review shows that the benefits of the product outweigh the risks, authorization to sell the product would be given to the manufacturer [21]. Also, in Canada, before gene therapy can be used to treat a certain genetic disorder, there are certain requirements that are needed to be met:
- The faulty gene that needs to be cured must be identified and how it results in the condition must be known so that the vector can be genetically altered for use and the appropriate cell or tissue can be targeted [8].
- The gene has to be able to be cloned so that it can be inserted into the vector [8].
- When the gene is transferred into the new cell, whether it is turned on or off needs to be controlled [8].
- There must be sufficient value in treating a disorder with gene therapy, it must be known if there is a simpler way to treat that disorder [8].
- The benefits and risks of using gene therapy for that condition must be considered favourable compared to other types of therapies [8].
- There must be enough data collected from cell and animal experiments to show that the procedure works and is safe to be used [8].
When these conditions are met, researchers may begin to start clinical trials that are closely monitored by institutional review boards and governmental agencies for safety [8].
Regulation of Gene Therapy in the U.S
In the U.S. gene therapy products have not been approved by the FDA (Food and Drug Administration) for sale yet [3]. But, there is a growing amount of research and development of gene-related therapies and the FDA has taken responsibility to oversee the activity of gene-related studies [3]. There have also been many manufacturing companies in the U.S. that are requesting to study gene therapy and develop products related to this study [3].
According to the FDA’s center for Biologics Evaluation and Research (CBER) which regulates the research for gene therapies the flowing requirements must be met: Manufacturers must test their products extensively [13]. They must first tell the FDA of why they are considering selling a gene therapy product [13]. They must test the product in a laboratory and in research animals [13]. They must obtain a permission exemption from the FDP before studying the gene therapy product in humans [13]. To obtain the permission exemption, the manufacturer must fill out an application stating how they will conduct the study, what possible risks may be involved with the study, what steps will be taken to protect the patients from these risks, they also need to provide data in favour of the study [13]. Researchers must have approval from an Institutional Review Board which protects the people who might participate in the study [13]. They also need to inform the patients participating in the study of the potential risks and benefits before obtaining their consent [13].
According to the FDA’s center for Biologics Evaluation and Research (CBER) which regulates the research for gene therapies the flowing requirements must be met: Manufacturers must test their products extensively [13]. They must first tell the FDA of why they are considering selling a gene therapy product [13]. They must test the product in a laboratory and in research animals [13]. They must obtain a permission exemption from the FDP before studying the gene therapy product in humans [13]. To obtain the permission exemption, the manufacturer must fill out an application stating how they will conduct the study, what possible risks may be involved with the study, what steps will be taken to protect the patients from these risks, they also need to provide data in favour of the study [13]. Researchers must have approval from an Institutional Review Board which protects the people who might participate in the study [13]. They also need to inform the patients participating in the study of the potential risks and benefits before obtaining their consent [13].
Similarity and Differences
The regulation of gene therapy products both in Canada and in the U.S. is very similar. There are many tests and conditions that must be met before a gene therapy product can even be clinically researched. And currently both in Canada and the United States, there are no gene therapy products that are approved for sale [13, 21].
In most countries, the regulation for gene therapy is very similar, there is a similar standard of rules that a manufacturer needs to follow before gene therapy can even go through a clinical trial. But in some countries, ethical views are more valued, especially in European countries such as Germany and Italy [12]. In these countries, the authorization of selling gene therapy products may be harder to obtain the in other countries because of the negative ethical impacts of gene therapy. This means that researchers from that country may have to join another manufacturing company in another country in order to do research on gene therapy. These researchers will then have to subject to the disease that is being researched in that manufacturing company and not the disease they would like to research. Also because of strict rules, patients in that country may feel that they should not get treatment because it is not allowed in that country, other patients may need to travel to other countries to get the treatment they need, adding to their list of expenses. Another problem caused by stricter regulations in another country would be that the therapies that are allowed to be tested and are authorized in one country may not be allowed in another country. This makes it seem that the country that allows the treatment is being less careful than another country and the government may be penalized for not imposing stricter rules. Also if this happens, there may be debate between the two governments between whether or not this product is safe, with criticisms told between the two countries, this may hurt the relationship between the two countries and impact other acts such as trade between the two countries.
The implications of having to go through a similar process for approval of clinical trials for a gene therapy products is unfortunate for the researcher, because if one country does not approve of the therapy, this means that other countries will probably also reject the product. There is no other way the researcher would be able test and manufacture the product which may or may not be helpful in terms of finding a cure.
In most countries, the regulation for gene therapy is very similar, there is a similar standard of rules that a manufacturer needs to follow before gene therapy can even go through a clinical trial. But in some countries, ethical views are more valued, especially in European countries such as Germany and Italy [12]. In these countries, the authorization of selling gene therapy products may be harder to obtain the in other countries because of the negative ethical impacts of gene therapy. This means that researchers from that country may have to join another manufacturing company in another country in order to do research on gene therapy. These researchers will then have to subject to the disease that is being researched in that manufacturing company and not the disease they would like to research. Also because of strict rules, patients in that country may feel that they should not get treatment because it is not allowed in that country, other patients may need to travel to other countries to get the treatment they need, adding to their list of expenses. Another problem caused by stricter regulations in another country would be that the therapies that are allowed to be tested and are authorized in one country may not be allowed in another country. This makes it seem that the country that allows the treatment is being less careful than another country and the government may be penalized for not imposing stricter rules. Also if this happens, there may be debate between the two governments between whether or not this product is safe, with criticisms told between the two countries, this may hurt the relationship between the two countries and impact other acts such as trade between the two countries.
The implications of having to go through a similar process for approval of clinical trials for a gene therapy products is unfortunate for the researcher, because if one country does not approve of the therapy, this means that other countries will probably also reject the product. There is no other way the researcher would be able test and manufacture the product which may or may not be helpful in terms of finding a cure.