Euro Convergence: How orphan drug suppliers can win labelling exemptions

Regulatory NewsRegulatory News | 11 May 2021 |  By 

Thomas Liebers

A strong local presence in EU markets is needed when seeking labelling exemptions that help reduce the cost of providing orphan products, according to experts who spoke at RAPS Euro Convergence 2021.
Manufacturers of orphan drugs can gain exemptions from some labelling requirements at an EU level and from individual member states, a potentially valuable flexibility for companies that make orphan drugs targeting very small numbers of patients. Such products face specific market access barriers stemming from the application of regulations that are a better fit for mass-market products.
“It is of course much more expensive for a company to produce a small number of packaging components, especially if those materials that need to be prepared with several local languages. This is just one example of a barrier that has its root cause in a regulatory requirement,” Thomas Liebers, senior director of regulatory labelling at PRA Health Sciences, said at the event.
With his colleague Betti Mozes-Stoddart, the regulatory labelling manager at PRA, Liebers discussed the ways orphan drug manufacturers can address those barriers and shared case studies about what has worked and not worked in the past.
As the experts explained, the EU and national-level exemptions differ. EU regulations allow parts of an orphan drug label to be in the official language of another member state. Authorities can grant full or partial exemptions from the obligation of translating the label and package leaflet, enabling, for example, a medicine to be supplied in Germany with an English leaflet. The exemption is limited to products supplied to healthcare professionals for administration to patients.
Manufacturers can seek EU exemptions from the initial marketing authorization application onward, including after a drug comes to market. There is no application fee and the exemption is permanent.
National-level exemptions are different. Such exemptions typically cover the entire pack, rather than the specific components addressed by the EU flexibilities, and are limited to a specific period of time or batch. Typically, manufacturers still need to provide the package leaflet in the local language, for example by attaching it to the outer carton.
Mozes-Stoddart presented case studies that described specific efforts to obtain exemptions; the examples pointed to the value of a local presence. “Having that local presence really speeds up the procedure. You can quite easily have a response to your request to a local exemption application within a few days, if it's made in the right way,” Mozes-Stoddart said.
Liebers spoke more broadly, describing the types of requests that are likely to be accepted or rejected by the authorities. “Exemptions are not supposed to fix poorly designed labelling strategies. Patient safety comes first, so exemptions for translation for leaflets containing critical information on the safe administration are rarely approved,” Liebers said. 
Health authorities expect companies to have explored the packaging options available to them, such as shortening the company address and shrinking the logo, before requesting an exemption, Liebers said.
RAPS Euro Convergence 2021


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