TMC Pharma Services Ltd

Marcelo Vaz

Rare Disease Advocate

Marcelo Vaz is the Vice President of Medical Services at TMC Pharma, bringing extensive experience in pulmonology, internal medicine and pharmaceutical research. He earned his medical degree from São Paulo University Medical School in 1992 and completed a doctorate in pulmonology, conducting part of his research at Vanderbilt University and St. Thomas Hospital. His career spans clinical practice, academic roles, and leadership positions in the pharmaceutical industry, including serving as Senior Medical Manager at Biosintetica/Aché, Medical Director at ICON Clinical Research, and Director of Medical Affairs at PRA Health Sciences.

Through this article, Vaz highlights the complex challenges pharmaceutical and biotechnology companies face in ensuring compliance with varying regulations for product communication, particularly within the rare disease space in the UK and EU.

UK/EU product communication compliance is a complex challenge for pharmaceutical and biotechnology companies. The complexity is multiplied when it comes to rare diseases.

Inside a rare disease environment, even before commercial launch, access to the product may be achieved through an earlyaccess program (for example, a compassionate-use or a cohortuse program), potentially requiring non-promotional or educational activities and materials. These conditions are often surrounded by limited sources of information and a lack of clarity on how the patient (or healthcare provider or healthcare organisation) must navigate this intricate route.

Even after commercialisation, when a patient-support program or a non-interventional post-authorisation safety study is required, additional non-promotional or educational activities and materials must be compliant and have their content, dissemination, and message adequately aligned.

Failure to align with compliance standards and advertisement regulations can have severe repercussions. In addition to corrective actions, potential regulatory fines, and administrative charges, the consequences include reputational damage and litigation risks, with possible legal action arising from misrepresentations or regulatory breaches. In extreme cases, violations could revoke marketing authorisation or even criminal charges in key markets.

Given the risks and complexity of these tasks, pharmaceutical and biotech companies operating in the rare disease space face mounting pressure to achieve compliance right from the outset.

Variability in compliance: country-by-country challenges

The European Federation of Pharmaceutical Industries Association’s (EFPIA) code of practice covers how interactions with healthcare professionals (HCPs), healthcare organisations (HCOs), and patient organisations (POs) should be conducted. However, each country will have its own specificities and local regulations modulating the application of the code in each country inside the EU.

What is permissible in one jurisdiction may require significant modifications—or result in outright rejection—in another.

Even with non-promotional materials, differences between countries are often subtle but significant. For example, in Germany, educational materials require prior vetting by educational bodies before they can be used. In Denmark, materials cannot include patient images, while in Norway, all promotional content must be uploaded to a central archive.

Even with non-promotional materials, differences between countries are often subtle but significant. For example, in Germany, educational materials require prior vetting by educational bodies before they can be used. In Denmark, materials cannot include patient images, while in Norway, all promotional content must be uploaded to a central archive.

A common misconception is that English-language materials approved for one market, such as the UK or US, can automatically be used across the EU. This is not the case. Compliance reviews must be country-specific in English-speaking markets, as regulatory interpretations differ. UK-approved materials must still undergo local adaptation, verification, certification, and approval before being used in other European markets.

Compliance also extends beyond materials to include activities that involve interactions with POs, HCPs, HCOs, and members of the public. These activities should also be verified and certified or approved before use, ensuring the contractual agreements guarantee compliance with code requirements. This includes mandatory financial disclosures, clear identification of all involved parties, and strict adherence to transparency obligations. Despite not being part of the EFPIA code of practice, GDPR requirements should also be evaluated and applied to the regions and countries where such activity is meant to be developed.

On top of the regulatory dimension, ethical sensitivity is crucial (as covered in the EFPIA’s code of conduct). Rare disease populations are often vulnerable, with limited treatment options or available information about the disease and treatment, resulting in a heightened reliance on patient advocacy groups for treating physicians. When working collaboratively with these patient organisations, companies must remain compliant—balancing the urgency of delivering vital information with ensuring their communications are not misleading, inappropriately promotional, or lacking clarity.

However, one of the primary distinctions in rare disease compliance is the scale of operations. Large pharmaceutical companies may have a footprint spanning several countries—with extensive portfolios of established in-country offices staffed with dedicated medical affairs and regulatory teams. With this localised approach, promotional and nonpromotional materials are reviewed and adapted per national regulations.

This model is simply unfeasible for small biotech firms developing therapies for rare diseases, where there may be just a handful of patients scattered across multiple countries.

Proactive compliance planning when entering European markets

Companies without in-country capabilities in all countries where the product is commercialised must adopt a different strategy to navigate compliance in rare diseases successfully.

One approach to succeed in compliance is to use the most stringent compliance codes to develop a master document that can be adapted for various markets. This 'master' material must then be reviewed by a network of expert ‘approvers’ across each country in which the material is intended to be used, ensuring local adaptations align with national requirements and maintain regulatory integrity. While interacting with country-specific approvers may be the more straightforward solution, working across several countries can also prove challenging. The process must be meticulously managed, beginning with English-language adjustments before translating, approving, and revalidating the material for each market.

Another effective strategy is appointing a dedicated service provider that offers marketing authorisation holder services or a centralised pan-European solution. This arrangement establishes a co-accountability model where the product owner delegates critical compliance oversight to a specialised entity, using the service provider’s established networks of medical and regulatory experts and local signatories to flag potential issues early and streamline market access. The provider shoulders the regulatory responsibilities associated with the product, ensuring every promotional and non-promotional material is thoroughly reviewed and approved in line with local and regional regulations.

The key takeaway for pharmaceutical or biotech companies new to the rare disease space or the European region is that product communication compliance cannot be an afterthought. It must be embedded into the commercialisation and distribution strategy from day one. Engaging with medical and regulatory experts, implementing a network of local compliance professionals, and planning for country-specific adaptations are critical to avoiding costly delays and regulatory pitfalls.

By taking a structured, proactive approach, pharmaceutical and biotech companies can navigate the complexities of UK/EU compliance while continuing to make a meaningful impact in the rare disease community—even without a physical presence in the region.