EMA and FDA collaborate to combat rare diseases

Drug production

Photo courtesy of the FDA

The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have set up a new working group, or “cluster,” on rare diseases.

This means the EMA and FDA will hold regular meetings via teleconference to share information on the regulation of medicines for rare diseases.

The cluster will provide a forum for the confidential exchange of draft documents, proposed policies, and detailed information supporting the scientific basis for decision-making on medicine development.

The agencies will exchange information on topics such as:

• The design of clinical trials in small populations and the use of statistical analysis methods
• The selection and validation of trial endpoints
• Preclinical evidence to support development programs
• The design of post-marketing studies—in particular, in the context of early access mechanisms such as the EMA’s conditional marketing authorization and the FDA’s accelerated approval
• Risk management strategies for long-term safety issues with medicines for rare diseases.

The first meeting of the rare diseases cluster took place on September 23, 2016. The cluster will initially meet once a month via teleconference and will be chaired jointly by the FDA and EMA.

The creation of this cluster is the latest step in the EMA’s and FDA’s wider objective to expand and reinforce international collaboration.

The clusters established by the agencies focus on areas where the parties involved could benefit from an intensified exchange of information and strengthened collaboration.

The existing EMA/FDA clusters address issues related to patient engagement, biosimilars, orphan medicines, cancer drugs, medicines for children, and pharmacovigilance, among other topics.

Drug production

Photo courtesy of the FDA

The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have set up a new working group, or “cluster,” on rare diseases.

This means the EMA and FDA will hold regular meetings via teleconference to share information on the regulation of medicines for rare diseases.

The cluster will provide a forum for the confidential exchange of draft documents, proposed policies, and detailed information supporting the scientific basis for decision-making on medicine development.

The agencies will exchange information on topics such as:

• The design of clinical trials in small populations and the use of statistical analysis methods
• The selection and validation of trial endpoints
• Preclinical evidence to support development programs
• The design of post-marketing studies—in particular, in the context of early access mechanisms such as the EMA’s conditional marketing authorization and the FDA’s accelerated approval
• Risk management strategies for long-term safety issues with medicines for rare diseases.

The first meeting of the rare diseases cluster took place on September 23, 2016. The cluster will initially meet once a month via teleconference and will be chaired jointly by the FDA and EMA.

The creation of this cluster is the latest step in the EMA’s and FDA’s wider objective to expand and reinforce international collaboration.

The clusters established by the agencies focus on areas where the parties involved could benefit from an intensified exchange of information and strengthened collaboration.

The existing EMA/FDA clusters address issues related to patient engagement, biosimilars, orphan medicines, cancer drugs, medicines for children, and pharmacovigilance, among other topics.

Drug production

Photo courtesy of the FDA

The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have set up a new working group, or “cluster,” on rare diseases.

This means the EMA and FDA will hold regular meetings via teleconference to share information on the regulation of medicines for rare diseases.

The cluster will provide a forum for the confidential exchange of draft documents, proposed policies, and detailed information supporting the scientific basis for decision-making on medicine development.

The agencies will exchange information on topics such as:

• The design of clinical trials in small populations and the use of statistical analysis methods
• The selection and validation of trial endpoints
• Preclinical evidence to support development programs
• The design of post-marketing studies—in particular, in the context of early access mechanisms such as the EMA’s conditional marketing authorization and the FDA’s accelerated approval
• Risk management strategies for long-term safety issues with medicines for rare diseases.

The first meeting of the rare diseases cluster took place on September 23, 2016. The cluster will initially meet once a month via teleconference and will be chaired jointly by the FDA and EMA.

The creation of this cluster is the latest step in the EMA’s and FDA’s wider objective to expand and reinforce international collaboration.

The clusters established by the agencies focus on areas where the parties involved could benefit from an intensified exchange of information and strengthened collaboration.

The existing EMA/FDA clusters address issues related to patient engagement, biosimilars, orphan medicines, cancer drugs, medicines for children, and pharmacovigilance, among other topics.