Targeted import as a mechanism for accessing innovative therapies

In this article, you will learn:

• What is targeted import, and in what circumstances can it provide access to treatments that are not available in Poland?
• What conditions must be met in order to import a medicinal product under the destination import scheme?
• How does the import process work – from the doctor issuing a prescription to the delivery of the product?
• what role do the doctor, the consultant, the Ministry of Health, the pharmacy and the pharmaceutical wholesaler play in the whole process?
• Under what conditions can one apply for reimbursement of a medicinal product imported under the destination import scheme?
• When does the Agency for Health Technology Assessment and Terapies (AOTMiT) become involved in the reimbursement process, and what is the significance of the recommendation it issues?

Abstract

The rapid development of medicine is leading to increasingly personalised treatments and the development of pharmaceutical technologies designed to address previously unmet medical needs. Although the situation for Polish patients regarding access to these innovative medicines is steadily improving, the pace of some changes still leaves much to be desired. As a result, early access programmes are beginning to play an increasingly important role. In the case of pharmaceutical technologies that have not yet been granted marketing authorisation in Poland but are registered in another country, targeted importation may be a solution.

Keywords

targeted imports, early-access programmes, innovative medicines, orphan medicines

The rapid development of molecular biology in recent years, including genomics and proteomics, has enabled the identification and classification of further subtypes of diseases, as well as the development of molecularly targeted therapeutic technologies that support the ongoing individualisation of treatment.

However, the processes for authorising these innovative medicines for marketing are becoming increasingly demanding and time-consuming, whilst the challenges they pose for healthcare systems are growing. The world’s two largest regulatory bodies, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), have for years been taking steps to accelerate the marketing authorisation of the highest-rated innovative medical technologies.

Recently, however, the US regulator has shown a clear lead, both in the number of applications received for the authorisation of new pharmaceutical technologies and in the speed at which they are processed. This applies to both standard and conditional procedures [1, 2]. It appears that this issue has been recognised in Europe, which has contributed to the growing popularity of early access programmes. In order to keep public funds under control, in most cases they are restricted to medicines offering the highest added value.

In the case of promising medicines that have not yet been authorised for marketing in the European Union, including in Poland, but have been registered in another country, early access may be provided through the targeted import mechanism.

Article 4 of the Act of 6 September 2001 – Pharmaceutical Law, which defines the procedure for targeted imports within the national context, also introduces certain restrictions [3]. Firstly, the use of the imported product must be essential to save the patient’s life or protect their health. Secondly, the medicinal product being imported must not have an equivalent registered in Poland containing the same active substance.

The primary aim of targeted imports is to ensure that patients have access to medicines that are not available on the domestic market but are essential for safeguarding their health and lives. This is particularly important in the case of rare diseases and cancer, where treatment options are usually very limited.

It is worth noting that, under the final import procedure, it is also possible to import from abroad foodstuffs for special nutritional purposes that are essential for saving life or health, as regulated by the Regulation of the Minister of Health of 14 March 2012. [4].

The final import process begins when a doctor issues a request for the import of a specific medicinal product. This applies both to medicines used exclusively in healthcare facilities and to those which patients may take themselves at home. This is carried out electronically via the Targeted Import Service System, which, in addition to issuing the requests themselves, also enables them to be processed, collected by pharmacies and pharmaceutical wholesalers, and facilitates communication with the Minister responsible for health.

In the second step, the request is forwarded to the national or regional consultant responsible for the relevant medical condition to confirm that it is justified. This is done via SOID within a maximum of 7 days.

The Minister of Health then considers the submitted request, usually within 21 days, and notifies the applicant of the outcome via the SOID system. A positive decision therefore constitutes authorisation to import and bring the medicine in from abroad [3].

Once authorisation for import has been granted, a request must be submitted to the pharmaceutical wholesaler via a pharmacy within 60 days, so that the wholesaler can begin processing it. Importantly, if this deadline is not met, the request becomes invalid and the procedure must be restarted from the beginning [3].

Once a medicinal product has been imported into Poland, it is usually subject to customs clearance and, upon arrival at the wholesaler’s premises, to a check of the documentation and the transport and storage conditions. Once the administrative procedures have been completed, it is delivered to healthcare facilities and pharmacies, where it becomes available to patients.

Although targeted imports can be an effective tool for ensuring access to innovative therapies, they also present a number of challenges. One of these is the issue of payment for imported medicinal products. Unfortunately, the prices of new, innovative medicinal technologies used to treat rare diseases or cancer are usually beyond the means of the average patient. It is worth noting, however, that the legislator has allowed for the possibility of applying for reimbursement for a medicine imported under the targeted import scheme.

An application for reimbursement is submitted directly to the Ministry of Health and is considered within a period not exceeding 30 days. It should be noted, however, that in order to assess the merits of granting reimbursement for a given medicinal product, the Minister of Health may request the President of the Agency for Health Technology Assessment and Tariff System (AOTMiT) to issue a recommendation on the matter. In such cases, the waiting time for approval is automatically extended by the time required for the President of AOTMiT to analyse the matter and prepare a recommendation [5].

The primary task assigned to the agency in the context of issuing recommendations is to provide a transparent and reliable compilation of analyses summarising the health, social, economic and ethical data and information relating to a given pharmaceutical technology.

The result of this work is a report, which usually includes:

• a description of the medical technology covered by the order, with particular regard to the availability of alternative technologies for the relevant indication or medical condition,
• a description of the medical condition and the specific indications for which the medicinal technology covered by the prescription is intended,
• the presentation of scientific evidence demonstrating the efficacy of a medicinal technology for a given indication, and clinical practice guidelines,
• data on the costs of the technology covered by the contract and its constituent parts.

Once the report has been drawn up, it is forwarded immediately to the Transparency Council, which uses it as a basis for preparing a position paper for the agency’s president. It should be emphasised that the recommendation of the AOTMiT president remains valid for three years and may be consistent with or contrary to the Council’s position [5].

Similarly, a negative recommendation from the President of the Agency for Medicines and Medical Devices (AOTMiT) may, but need not, constitute grounds for the Minister of Health to refuse to grant reimbursement for a given medicinal product.

It is mandatory to obtain a recommendation from the President of the Agency for Health Technology Assessment and Tariff Setting where the Minister of Health receives more than 10 applications for authorisation to reimburse a medicinal product containing a specific active substance for a given indication.

Targeted imports are a key element in shaping medicines policy, particularly in relation to conditions with clearly unmet medical needs. In practice, this applies to patients with chronic or life-threatening conditions carrying a high risk of progression, such as rare diseases or cancers, where any delay in commencing treatment results in irreversible consequences and is ethically reprehensible.

However, its effective implementation in the national context requires the commitment of all parties involved – doctors, pharmacists, pharmaceutical wholesalers and patients. After all, any measure that reduces the time patients have to wait for previously unavailable yet effective pharmaceutical technologies is in line with a patient-centred approach to health policy.

1. Joppi R, Bertele V, Vannini T, Garattini S, Banzi R. Food and Drug Administration vs European Medicines Agency: Review times and clinical evidence on novel drugs at the time of approval. Br J Clin Pharmacol. 2020, pp. 86:170–174.
2. Downing NS, Zhang AD, Ross JS. Regulatory Review of New Therapeutic Agents – FDA versus EMA, 2011–2015. N Engl J Med. 2017, 376(14): 1386–7.
3. Act of 6 September 2001 – Pharmaceutical Law. 2001. Journal of Laws 2001 No. 126, item 1381.
4. Regulation of the Minister of Health of 14 March 2012 on the importation from abroad of foodstuffs for special nutritional purposes necessary to save life or health. 2012. Journal of Laws 2012, item 348.
5. The Act of 12 May 2011 on the reimbursement of medicines, foodstuffs intended for particular nutritional uses and medical devices. 2011. Journal of Laws 2011 No. 122, item 696.