The International Gaucher Alliance (IGA) has issued a position statement on the use of non-comparable medicines for Gaucher disease. The statement outlines the IGA’s stance on the use of ‘intended copies’ of enzyme replacement therapy (ERT) medicines as a treatment for Gaucher disease. These ‘copies’ are also known as non-comparables because they have not been directly compared to the original medicine.

Non-comparables are copies of licensed biological medicines which have not followed the rigorous regulatory pathways required for biosimilar medicines, which are strictly regulated by the by European Medicines Agency (EMA), the United States Food and Drug Administration (FDA) and the Japanese Pharmaceuticals and Medical Devices Agency (PDMA), to ensure they are as safe and effective as the reference product. Therefore, it is difficult to know how a non-comparable medicine will perform in terms of efficacy, safety, and quality.

The IGA welcomes the availability of new medicines, as this offers better access and greater treatment choice for patients. However, we are aware of the challenges in the way non-comparables could be used or interchanged with other approved medicines. In particular, we have concerns about:

• the lack of safety and efficacy data.
• the lack of accurate product information.
• the lack of clarity and around how local approvals have been made.
• how patients are informed about these treatment options, as this has the potential

to cause anxiety for the patient community as well as create false expectations.

Non-comparables being used for the treatment of Gaucher disease is the reality in many countries. The current situation is unsatisfactory, but the IGA believes that many of the current issues can be addressed by taking targeted action.

“We have made several recommendations to help make the use of non-comparables safer,” said IGA CEO Tanya Collin-Histed. “These include medicine naming and labelling that clearly indicates if it is a non-comparable, accurate product information being included in all packaging, robust safety and efficacy data and improved education about non-comparables.

“Community groups should engage directly with non-comparable manufacturers,” Collin-Histed continued. “We should seek to establish a positive dialogue with the manufacturers of non-comparables in Gaucher disease and explore opportunities for mutual collaboration that will benefit all parties.”

Collin-Histed added: ”The IGA aims to engage with medicines manufacturers to explore the possibility of pricing biological medicines more competitively and transparently, in order to improve patient choice and increase access to medicines, while limiting the risk of compromising patient due to economic considerations. The IGA will also seek to engage with all stakeholders to raise awareness of the issues surrounding non-comparables in Gaucher disease and generate a positive discussion about how to proceed.”

View the full text of the Position Statement.

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