Results
Supply under NPP is a possibility across all three scope markets. Below is a brief overview of NPPs in each market.
Germany
NPP is known as “Individueller Heilversuch”, which is appropriate when there are no alternative therapeutic options, and scientific evidence supports the assumption of benefit for the given patient.3 It is legally justified by the emergency regulation of §34 of the Criminal Code.3
There is neither an official application/approval for conduct of NPPs, nor are there any reporting requirements. Regarding cost assumption, there is no separate regulation; instead, this must be clarified individually for each case among the parties involved.3
Unless the treatment is globally unlicensed, charging under NPP is permitted, with the reimbursement provision usually via the respective Sick Fund.1
France
Following the reform in 2021, NPP, previously described by “Autorisation Temporaire d’Utilisation Nominative (ATUn)”, is covered by Compassionate Access, specifically the “Autorisation d’Accès Compassionnel (AAC)”, under the remit of “Agence Nationale de Sécurité du Médicament et des produits de santé (ANSM)”.4
AAC is primarily intended for treatments with no ongoing clinical trials in the specific indication, and it can be granted for up to one year, with the possibility to renew.4
With a medicine subject to research involving human subjects (RIHS), following its first AAC, the laboratory must submit a request for early access within 12 months, or 18 months in the case of rare diseases.5
The laboratory may make a justified request to extend this deadline.5 Upon grant of authorisation for compassionate access, there is automatically full coverage by Health Insurance, with the laboratory having the possibility to freely set the price.6 However, the laboratory is then subject to mandatory annual rebate payments.6
United Kingdom
NPP is also possible, described by “Specials” (unlicensed medicinal products) imported into the UK in response to an unsolicited order of a registered person for the treatment of individual patients.7
The importer of “Specials” must hold either a Wholesale Dealer’s License or a Manufacturer’s “Specials” License, if the product is being imported from a European Economic Area (EEA) country or a non-EEA country, respectively.7 An import notification is required, with the Medicines & Healthcare products Regulatory Agency (MHRA) having the option to object to the importation (e.g., due to patient safety concerns or licensed alternatives in the UK).8
However, an MHRA scientific assessment of the product is not required, hence receiving approval may be relatively quick (usually within 28 days).8 Charging is permitted, with the reimbursement provision usually via the hospital drug budget, or the National Cancer Fund, for example.1
Discussion & Conclusion
In many countries around the globe, including Germany, France and the UK, NPPs are a fundamental component of pre-approval access. NPPs may come with recognised benefits:
- From the patient’s viewpoint, NPPs can offer an access pathway to a pre-launch product, when all other therapeutic options have been exhausted. Thus, patient needs could be met in a more timely manner.
- From the manufacturer’s viewpoint, a level of control over product supply is possible and, in the case of charged NPPs, revenue can be created prior to launch. Moreover, knowledge and experience of key opinion leaders (KOLs) with the product is enhanced, which in turn helps build relationships early on and potentially enhances post-launch product uptake. Furthermore, any real-world data collected under NPPs could be leveraged towards the evidence base used for subsequent approval and/or reimbursement of the product.
NPPs are an option that should not be overlooked and warrant serious consideration by pharmaceutical companies when planning the commercial strategy of their product. This is particularly relevant to products developed for rare diseases, a therapeutic space with generally higher unmet needs, limited development pipelines, as well as greater pressure by physicians and patient advocacy groups.
Supply under NPP does not hinder the pharmaceutical company from pursuing commercialisation at a later timepoint. Indeed, in markets with greater uncertainty around demand, NPPs may represent an attractive access route, whether pharmaceutical companies opt to proceed with commercialisation in the specific market, or not.
The driving force behind NPPs
- Ultimately, decisions on NPP should be dictated by the company’s overall regulatory and go-to-market strategy, to see how NPPs could best serve this strategy.
- The objectives and desirable outcomes of NPPs should be explicit and clear.
- Decisions governing NPP implementation (for example, which countries to target, in what sequence etc.) should fit within the broader strategy.
Bringing NPPs to life
- Importantly, NPP implementation requires substantial understanding of their specificities and is often associated with considerable bureaucracy and administrative burden.
- Moreover, there is notable cross-country variance when it comes to the regulatory framework, pricing arrangements, logistics, practical considerations and guidance around NPPs.
- Therefore, to help manage the complex territory of NPPs, pharmaceutical companies tend to collaborate with NPP specialist organizations to plan and successfully implement these programs.
References:
- Lewis KD. Access to unlicensed medicines, who should pay when they are not provided for free? Medicine Access @ Point of Care. 2021;5:239920262110400. doi:https://doi.org/10.1177/23992026211040047
- DLRC. UK Early Access Programs: EAMS and “Specials.” DLRC. Published February 5, 2024. Accessed December 9, 2024. https://www.dlrcgroup.com/uk-early-access-programs-eams-and-specials/
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