Publication date: November 07, 2024
The role of Polish Agency for Health Technology Assessment and related reimbursement of medicinal products
1. Introduction
In December 2021, Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (OJ EU L 458, 2021, p. 1; hereinafter referred to as the Joint Clinical Assessment Regulation or the Regulation) was adopted. In turn, less than half a year ago, Commission Implementing Regulation (EU) 2024/1381 of 23 May 2024 was adopted, establishing, in accordance with Regulation (EU) 2021/2282 on health technology assessment, procedural rules on interactions during the preparation and updating of joint clinical assessments, the exchange of information on the preparation and updating of joint clinical assessments and the participation in the preparation and updating at Union level of joint clinical assessments for medicinal products for human use, and establishing document templates for those joint clinical assessments (OJ EU L 2024; hereinafter referred to as the Implementing Regulation).
Both of these regulations will soon become legally binding, which will lead to fundamental changes in the pharmaceutical market of the European Union Member States, including Poland. A joint clinical assessment will be introduced, as a result of which the clinical assessment of medicines and medical devices introduced to the Polish market will no longer be carried out at the national administration level by the Agency for Health Technology Assessment and Tariff System.
2. Assumptions of a joint clinical assessment
What is a joint clinical assessment? In short, under the Regulation, the clinical assessment necessary for marketing authorization will be carried out at the level of EU institutions. At first glance, this seems like a good solution. A pharmaceutical company that wants to introduce a drug to the European market will be able to introduce the drug to the market in the European Union (27 Member States) and the European Economic Area (Norway, Iceland and Liechtenstein) based on a single clinical assessment and a report prepared on its basis. This will significantly facilitate the circulation of drugs within the common market. However, experts see far-reaching consequences of this legal change for applications for the establishment of reimbursement for a drug, which is still done at the level of the national administration of individual Member States. However, before this problem is discussed, the basic assumptions of the reform will be presented.
Under Article 2(6) of the Regulation on joint clinical assessment, a joint clinical assessment of a health technology is defined as: “a scientific compilation and description of the comparative analysis of available clinical evidence on a health technology in comparison with one or more other health technologies or existing procedures, in accordance with the scope of assessment agreed in accordance with this Regulation and based on the scientific aspects of clinical HTA domains including a description of the health problem addressed by the health technology and the current use of other health technologies addressing that health problem, a description and technical characterisation of the health technology, the relative clinical effectiveness and the relative safety of the health technology”.
Importantly, this does not apply to every product that meets the EU definition of a health technology. According to Article 7(1) of the Joint Clinical Assessment Regulation, such an analysis will be carried out on:
1. medicinal products for which marketing authorisation is applied for;
2. medicinal products for which a change to the issued marketing authorisation is requested;
3. Class IIb or III medical devices placed on the market under the terms of Regulation (EU) 2017/745 of the European Parliament and of the Council of 5 April 2017 on medical devices, amending Directive 2001/83/EC, Regulation (EC) No 178/2002 and Regulation (EC) No 1223/2009 and repealing Council Directives 90/385/EEC and 93/42/EEC (OJ EU L. of 2017, No. 117, p. 1, as amended) – including:
– non-invasive products coming into contact with damaged skin or mucous membrane are intended for use primarily in the case of skin damage with damage to the dermis or mucous membrane, which can only heal by granulation,
– invasive products intended for long-term use,
– surgically invasive devices specifically intended to control, diagnose, monitor or correct defects of the heart or central circulatory system through direct contact with these parts of the body,
surgically invasive devices specifically intended for use in direct contact with the heart or central circulatory system or central nervous system;
in vitro diagnostic medical devices , which are placed on the market under the terms of Regulation (EU) 2017/746 of the European Parliament and of the Council of 5 April 2017 on in vitro diagnostic medical devices and repealing Directive 98/79/EC and Commission Decision 2010/227/EU (OJ EU L. of 2017, No. 117, p. 176, as amended) – i.e. in vitro diagnostic medical devices intended for use for the following purposes:
– detecting the presence of, or exposure to, a transmissible agent in blood, blood components, cells, tissues or organs or any of their derivatives in order to assess their suitability for transfusion, transplantation or cell administration,
– detecting the presence of a transmissible agent causing a life-threatening disease with a high or suspected high risk of spreading or detecting exposure to such an agent,
– determining the concentration of an infectious agent of a life-threatening disease, where monitoring is of fundamental importance in the process of patient care,
– marking of the following markers as part of blood grouping or tissue compatibility grouping to ensure immunological compatibility of blood, blood components, cells, tissues or organs intended for transfusion, transplantation or cell administration: ABO system [A (AB01), B (AB02), AB (AB03)], markers of the Rh system [RH1 (D), RHW1, RH2 (C), RH3 (E), RH4 (c), RH5 (e)], markers of the Kell system [Kel1 (K)], markers of the Kidd system [JK1 (Jka), JK2 (Jkb)], markers of the Duffy system [FY1 (Fya), FY2 (Fyb)].
The joint clinical assessment under Article 8 of the Joint Clinical Analysis Regulation is carried out by two persons representing the Member State Coordination Group for Health Technology Assessment, referred to in the Regulation as the ‘assessor’ and the ‘co-assessor’. These persons are to be experts from different Member States. The result of this procedure is a joint clinical assessment report with an attached summary report, which becomes legally binding after approval by the Member State Coordination Group for Health Technology Assessment under Article 12(2) of the Joint Clinical Assessment Regulation. This document is one of the required annexes to the application for placing the medicinal product on the market.
Importantly, pursuant to Article 14 of the Joint Clinical Assessment Regulation, it is possible to update a joint clinical assessment if the original report states that: “it will be necessary to update it when additional evidence for further assessment becomes available”. This is of great importance not only from the perspective of progress in health sciences and updating the state of pharmaceutical knowledge, but also of possible side effects of the use of medicines, which will only be detected after obtaining a joint clinical assessment.
What has been controversial in relation to the joint clinical assessment is the issue of using documents developed through the analysis conducted at the EU level in the procedure for granting the status of a reimbursed medicine conducted at the national administration level. In the current legal situation, the entry on the list of reimbursed medicines is decided by the Member States of the European Union through the competent authorities. In Poland, such an authority is the minister responsible for health, which results from Article 11 paragraph 1 Act of 12 May 2011 on the reimbursement of medicines, foodstuffs for particular nutritional uses and medical devices (consolidated text: Journal of Laws of 2024, item 930, as amended; hereinafter referred to as the Reimbursement Act). In the period before the regulation on joint clinical assessment became legally binding, voices were raised in the public debate that there might be a discrepancy between the information disclosed in the report on the joint clinical assessment developed at the level of EU institutions and the information from the clinical assessment required to be presented in the procedure for granting reimbursement of the drug. One of the proposals to solve this problem was the so-called bis clinical assessment – an additional document submitted alongside the joint clinical analysis by the applicant for entry on the list of reimbursed medicines[1].
However, everything indicates that such a “bis-clinical assessment” will not be necessary. An amendment to the Reimbursement Act is currently being prepared to adapt it to the regulation on joint clinical assessment, which is intended to solve the problem of discrepancies between the requirements of the EU joint clinical assessment and the national entry on the list of reimbursed drugs[2]. According to the position of the Ministry of Health: “it is necessary to adapt the provisions of national law by appropriately reducing the requirements for applications for reimbursement and establishing the net sales price and adding new tasks for the Agency for Health Technology Assessment and Tariff System, hereinafter referred to as the <Agency> and the President of the Agency”. In light of the data from the Government Legislation Center, the draft bill is in the phase of consultations and public consultations[3]. The bill is planned to be adopted by the Council of Ministers in the fourth quarter of 2024[4] and then submitted to the Sejm for consideration.
As part of the amendment, Article 26, point 2) letter h) of the Reimbursement Act will receive a new wording. The new provision will mean that in certain cases, the entity applying for reimbursement of a drug will not be obliged to submit a clinical assessment to the Polish body at all, provided that this data has already been included in the joint clinical assessment, and the application for reimbursement of the drug:
– concerns medical technology in the same indication
– applies to the same target population
– meets the requirements of the Reimbursement Act[5].
Therefore, the discrepancies between the joint clinical assessment and the requirements for the drug to be reimbursed in Poland will most likely be removed. Entrepreneurs from the pharmaceutical sector should not have any concerns about this.
It is also worth mentioning that under Section 2 of the Joint Clinical Assessment Regulation there is also the possibility of joint scientific consultations. These are arrangements between the Member States Coordination Group for Health Technology Assessment and entities developing a medical technology (e.g. pharmaceutical companies). They are intended to facilitate: “the generation of evidence that meets the likely evidence requirements for a future joint clinical assessment of that health technology”, which results from Article 16(1) of the Joint Clinical Assessment Regulation. Therefore, it will be possible to determine, even before the joint clinical assessment procedure begins, issues that are important for the manufacturer interested in obtaining a marketing authorisation.
The procedures for joint scientific consultations are similar to those for joint clinical assessment. The joint scientific consultation results in a final document developed by the assessor and the co-assessor under the terms of Article 18 of the Regulation on joint clinical assessment. Then, under Article 19 of that Regulation, the Member States Coordination Group on Health Technology Assessment will approve the final document of the joint scientific consultation and give it legal force.
The regulation on joint clinical assessment will be implemented in several stages. First, on 12 January 2025, it will start to apply to medicines intended for the treatment of cancer and advanced therapy medicines. Then, on 13 January 2028, it will gain legal force for the so-called orphan medicines, the list of which is developed on the basis of Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ EU. L. of 2000, No. 18, p. 1, as amended). Then, from 13 January 2030, it will apply to all medicinal products placed on the market in the European Union and the European Economic Area.
In turn, the implementing regulation, which establishes procedural rules for the joint clinical assessment carried out before the European Medicines Agency, will become legally binding on 12 January 2025. This will ensure that appropriate procedures are in place already at the first stage of implementation of the regulation on the joint clinical assessment, which will ensure their effectiveness.
The new regulation of clinical assessment in the European Union is a step forward in the harmonization of the common market. It is of key importance for a highly promising sector of the economy, which is the pharmaceutical industry. Thanks to the joint clinical assessment and possible joint scientific consultation, manufacturers of medicines and medical devices will be able to enter the markets of other EU and EEA countries more easily, which will allow, on the one hand, to increase their profits, but on the other hand, to increase competition in individual national markets.
The new rules for clinical assessment in the European Union member states have raised controversy related to the adjustment of the EU system of joint clinical assessment with national drug reimbursement systems. This is nothing extraordinary – this happens very often when implementing new regulations, especially such complex ones. However, everything indicates that during the legislative work on the amendment of the reimbursement act, these problems will be removed, and national law will be adjusted to EU law.
Ultimately, a joint clinical analysis facilitates the introduction of medicines and medical devices to the common market of the European Union and the European Economic Area (30 countries in total), which is of great importance for the growing pharmaceutical sector.
Bibliography:
a) legal acts:
– Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ EU L 18, 2000, p. 1, as amended);
– Regulation (EU) 2017/745 of the European Parliament and of the Council of 5 April 2017 on medical devices, amending Directive 2001/83/EC, Regulation (EC) No 178/2002 and Regulation (EC) No 1223/2009 and repealing Council Directives 90/385/EEC and 93/42/EEC (OJ EU L 117, 2017, No. 117, p. 1, as amended);
– Regulation (EU) 2017/746 of the European Parliament and of the Council of 5 April 2017 on in vitro diagnostic medical devices and repealing Directive 98/79/EC and Commission Decision 2010/227/EU (OJ EU L. of 2017, No. 117, p. 176, as amended);
– Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (OJ EU L 458, 2021, p. 1);
– Commission Implementing Regulation (EU) 2024/1381 of 23 May 2024 establishing, in accordance with Regulation (EU) 2021/2282 on health technology assessment, procedural rules for interactions during the preparation and updating of joint clinical assessments, for the exchange of information on the preparation and updating of joint clinical assessments and for participation in the preparation and updating at Union level of joint clinical assessments for medicinal products for human use, and for establishing templates for the documents for those joint clinical assessments (OJ EU L 2024);
– Act of 12 May 2011 on the reimbursement of medicines, foodstuffs for particular nutritional uses and medical devices (consolidated text: Journal of Laws of 2024, item 930, as amended);
– Draft of 22 September 2024 (UC 69) – Act amending the Act on the reimbursement of medicines, foodstuffs for particular nutritional uses and medical devices and the Act on health care services financed from public funds.
b) press materials:
– Luiza Jakubiak, Clinical analysis bis. This is AOTMiT’s idea of how to connect two worlds, Rynekzdrowia.pl press material from October 15, 2024, https://www.rynekzdrowia.pl/farmacja/analiza-kliniczna-bis-to-pomysl-aotmit-jak-polaczyc-ze-soba-dwa-swiaty,263876,6.html?utm_source=newsletter&utm_medium=email&utm_campaign=rynekzdrowia.pl15-10-2024 (accessed: November 4, 2024);
– Katarzyna Redmerska, Changes in the principles of health technology assessment, MedicalPress.pl press material from September 25, 2024, https://medicalpress.pl/system/zmiany-w-zasadach-oceny-technologii-medycznych_XgR7dW9yZO/ (accessed: November 4, 2024).
c) Internet materials:
– https://legislacja.rcl.gov.pl/projekt/12389801/katalog/13082795#13082795 (accessed: 04/11/2024);
[1] Luiza Jakubiak, Clinical assessment bis. This is AOTMiT’s idea of how to connect two worlds, Rynekzdrowia.pl press material from October 15, 2024, https://www.rynekzdrowia.pl/farmacja/analiza-kliniczna-bis-to-pomysl-aotmit-jak-polaczyc-ze-soba-dwa-swiaty,263876,6.html?utm_source=newsletter&utm_medium=email&utm_campaign=rynekzdrowia.pl15-10-2024 (accessed: November 4, 2024).
[2] Katarzyna Redmerska, Changes in the principles of health technology assessment, MedicalPress.pl press release of September 25, 2024, https://medicalpress.pl/system/zmiany-w-zasadach-oceny-technologii-medycznych_XgR7dW9yZO/ (access: November 4, 2024).
[3] https://legislacja.rcl.gov.pl/projekt/12389801/katalog/13082795#13082795 (accessed: 04/11/2024).
[4] https://www.gov.pl/web/premier/projekt-ustawy-o-zmianie-ustawy-o-refundacji-lekow-srodkow-spozywcze-specjalnego-przeznaczenia-zywieniowego-oraz-wyrobow-medycznych-oraz-ustawy-o-swiadczeniach-opieki-zdrowiej-finansowanych-ze-srodkow-publicznych (accessed: 04/11/2024).
[5] Draft of 22 September 2024 (UC 69) – Act amending the Act on the reimbursement of medicines, foodstuffs for particular nutritional uses and medical devices and the Act on health care services financed from public funds.