Benefit assessments (HTA) on pharmaceutical products and medical devices play an important role for refunds by the national health services. Whereas HTA has applied already for quite some time with regard to pharmaceutical products it reaches out these days increasingly to other health care technologies and medical devices in particular.
In regard to the statutory health insurance system, Germany is one of the few European countries where pharmaceutical enterprises are allowed to launch drugs without prior regulatory pricing. Regulatory pricing takes place separately from the product's market entry. In most other EU Member States, the prices of reimbursable drugs are fixed by the national authorities or determined through price negotiations before the products can enter the market as part of national health insurance schemes.
The majority of countries, including Spain, Bulgaria, Italy, the Netherlands, Austria and France, maintain positive lists of drugs covered by the national health insurance system. A pharmaceutical has to be admitted to the positive list, that is to say a reimbursable price has to be fixed for it, before it can be marketed under the national health insurance scheme.
HTA-based benefit evaluations are the basis for national pharmaceutical pricing already in many Member States. In other words, the decision on whether or not a drug offers an additional benefit determines its price.
In the current legal situation, pharmaceutical enterprises are required to carry out HTAs in each Member State in order to determine additional pharmaceutical benefits. The same applies increasingly to other Health Care Technology Products.
This leads to problems:
Through the external price referencing principle that is applied by most European countries with regard to pharmaceuticals, national regulatory pricing influences pricing in other Member States.
External price referencing means that the drug prices in the relevant countries are compared in order to fix a national price or establish a basis for price negotiations with the pharmaceutical undertaking.
In price negotiations in Germany, pharmaceutical enterprises are required to disclose their actual sales prices in other European countries.
In France, the Comité Economique des Produits de Santé evaluates the benefit of new drugs, determining their prices by reference to the prices in other important EU Member States (Germany, Italy, Spain and Great Britain).
A similar procedure is employed in the Netherlands, where the healthcare department fixes maximum wholesale prices for all prescription drugs, depending on the average price of similar products in Belgium, France, Germany and Great Britain.
However, the legal requirements for HTA-based benefit evaluations remain to be harmonised throughout the European Union.
The European authorities recognised the lack of harmonisation and the problems associated with it already in 2011. EUnetHTA, a European HTA network, has been established on the basis of Art. 15 of the Directive 2011/24/EU on the application of patients' rights in cross-border healthcare.
The aim is to support and facilitate cooperation and the exchange of scientific information among Member States within the voluntary EUnetHTA network, connecting the national authorities or bodies responsible for HTA designated by the Member States.
EUnetHTA was established to support Member States in providing objective, reliable, timely, transparent, comparable and transferable information on the relative efficacy as well as on the short- and long-term effectiveness of health technologies, and to enable an effective exchange of this information between the national authorities and bodies in order to avoid duplication of assessments.
Measures that are taken for purposes of this cooperation do not affect the Member States' responsibility for decisions on cost reimbursement by the national health services under Art. 168 (7) TFEU.
On 18 March 2013 the Commission published a proposal for a Directive relating to the transparency of measures regulating the prices of medicinal products for human use and their inclusion in the scope of public health insurance systems. The proposal offered first regulatory approaches but did not address or regulate any benefit assessment procedures. Due to controversial discussion it was finally withdrawn in March 2015.
HTA-based benefit evaluations as well as mechanisms for the recognition of and reference to such decisions on the same products in other EU Member States remained to be harmonised throughout the European Union.
On the 31 January the Commission published its draft of a new Health Technology Assessment Regulation which had been long awaited for. The laws governing the marketing authorisation and the marketing of medicinal products, medical devices, pharmacovigilance, and drug advertising are largely harmonised throughout the EU, including assessments in the context of a drug's marketing authorisation and the certification of medical devices respectively.
Member States that examine applications for a marketing authorisation have to exchange assessment reports and acknowledge each other's decisions. The same principles have been introduced with the MDR in respect to high risk Medical Devices following Art. 54 MDR.
This has been considered by the Commission in its Proposal for a Regulation of the European Parliament and of the Council on HTA and amending Directive 2011/24/EU. The new regulation promotes one of the European legislator's primary objectives – creating a single market for medicinal products and medical devices that is subject to harmonised legislation.
The Regulation aims to abolish obstacles to market access of medicinal products and medical devices which are subject to HTA in the various Member States, avoid double assessments and to facilitate cooperation and data exchange between the various bodies engaged in HTA in the Member States.
The scope of the regulation is to enhance and accelerate market access of innovative health care technology on the European market, to make better use of resources of medicinal products and medical devices and to harmonise HTA methodology and techniques. As Regulation the new legal framework, as proposed by the Commission, will, if adopted, be directly applicable in all Member States.
The Regulation is divided into five chapters which provide for further subsections. Chapter I sets the general rules, describing the scope of the regulation and defining the terms as used in the regulation, describing the role and function of a coordination group to be formed and its operation.
The coordination group to be formed by the Member States in accordance with Art. 3 of the draft will play a key role in achieving harmonisation in HTA-processes. The coordination shall be comprised by the national authorities and bodies and their subgroups competent for HTA in the respective Member States.
The Member States can nominate more than one authority or body as member of the coordination group, but each Member States has only one vote in the coordination group which will decide with simple majority. Sessions of the coordination group will be chaired by the Commission and a second chair person.
Members of the coordination group will be published by the Commission on a web-based platform to be developed following Art. 27 of the draft regulation.
Chapter II describes the scope and procedure as applied for HTA on an EU-level following the regulation. It distinguishes Joint Clinical Assessment (Section 1) and Joint Scientific Consultations (Section 2), providers for studies on emerging health technologies (Section 3) and the voluntary cooperation on HTA (Section 4).
The key element of the proposal is the joint clinical assessment. A joint clinical assessment involves applying a mandatory HTA to every medicinal product subject to the central marketing authorisation procedure and to every new therapeutic indication for which the medicinal product later undergoes the marketing authorisation procedure (Article 5 No. 1a of the Proposal for a Regulation).
The HTA procedures will be updated if the medicinal products were approved under conditions or if new, additional data becomes available that has the potential to increase the accuracy of the initial assessment. For class IIb and III medical devices according to the Regulation 2017/745 and for in vitro diagnostics under the Regulation (EU) 2017/746, that is to say products subject to the consultation procedure, the draft Regulation also provides for a joint European HTA to be carried out.
In addition, the proposal defines that they shall be selected on the basis of the criteria laid down in paragraph 2, obviously giving the Coordination Group a right of determination. Thus, according to the current draft proposal, not all medical devices of the relevant classes are subject to a joint European HTA.
Article 6 refers to the content and preparation of the joint clinical assessment report. Based on the assessed evidence and evaluation of the underlying patient-relevant health outcomes, the report analyses the relative therapeutic effects of the health technology being assessed and the degree of certainty of the therapeutic effects. The proposal does not include an official definition of the term "patient-relevant health outcomes".
Similar to the marketing authorisation procedure for a medicinal product, the content of the HTA report is prepared by assessors from the different Member States and coordinated by a Coordination Group.
Regarding the practical application of the findings of a joint clinical assessment report, Article 8 No. 1a of the proposal for a Regulation provides that, once the Commission has approved and published a draft HTA report, the Member States may no longer carry out a clinical assessment or equivalent HTA procedure on the assessed health technology (medicinal product or medical device) on the national level. Instead, the HTA report must be taken into consideration in the health technology assessments at Member State level.
Joint Scientific Consultations, following Chapter II Section 2 of the proposal are designated to help health technology developers through scientific advice to design their studies aiming to render the necessary evidence for the later use in a joint clinical assessment.
Already in the development phase of a health technology, the developer should by way of scientific advice get information on the data and evidence which most likely will be required as part of a future joint clinical assessment. The scientific consultation report addressed to the developer will have neither binding effect on him nor on any Member State at the time of joint clinical assessment, but will give guidance only.
However, without reason in a following clinical assessment it will be hard to argue that other data or evidence will be required than those as identified and recommended in the consultation procedure.
Chapter III sets out the rules for Clinical Assessment which will be developed in detail in tertiary legislation. The rules are to ensure a harmonised approach to clinical assessment across EU Member States and are designed to make use of tools already developed under the EUnetHTA Joint Actions. The rules will basically ensure that clinical assessment in the same way as clinical trials are done in an independent and transparent manner, free from conflicts of interest.
Chapters IV and V define the support network and in the formal provisions provide for an evaluation and monitoring of the new legal framework set out to the proposed regulation transitional provisions and the empowerment of the Commission to adopt the implemented and delegated acts referred to in the Art 32 of the proposed regulation.
Harmonisation in the area of HTA throughout the EU with regard to health care technology is a key element of ensuring efficient market access of innovative products and their free circulation on the European market. However, already now the proposal is criticised as inconsistent with Art. 168 (7) TFEU stipulating that the EU shall respect the Members States responsibility and freedom to organise their health care policy and the delivery of health care services.
It is argued that the binding effect of joint Healthcare Assessments does interfere with this principle. It can be debated whether this view stands a detailed legal analysis. Section 11 of the recitals of the proposal addresses these questions and clarifies that the scope of the rules is limited to the clinical assessment of a health technology. The outcome of this assessment, although binding, does not affect the Member States discretion with regard to subsequent decisions.
The envisaged harmonisation in this regard has the same effect as the Regulation on Clinical Trials and harmonised EU-law on the authorisation of medicinal products or the certification of medical devices. Their EU-wide market access does not prevent a Member State to exclude any product from its participation in the healthcare reimbursement system of the respective Member State unless this happens by way of discriminatory measures.
It will have to be seen with interest which way the debate on the proposal will take. The initiative of the Commission taken with the proposal is to be welcomed.