The market for medicines in Europe is highly regulated, diverse and complex. The European Commission establishes common procedures for marketing authorization, and broad principles for pricing and reimbursement (P&R) (such as the need for criteria to be clear and objective),1 but national authorities set the criteria they consider appropriate.

This article compares P&R in Spain with four other countries: England, Sweden, France, and Germany. The process in Spain has recently been criticized by public auditors,2,3 particularly concerning the vagueness of the criteria for P&R decisions and the high degree of discretion allowed to decision making bodies and there have been several proposals for reform4–7 A substantial literature has compared health technology assessment (HTA) procedures across countries8–16 but to our knowledge this is the first study that makes recommendations specifically for the P&R process for new medicines for Spain. The four comparator countries were chosen because they each have mature HTA systems (and hence have had time to learn from experience), offer universal public healthcare (as does Spain), and have formal, well-respected HTA processes. Nevertheless, HTA takes place within a particular institutional, legal and cultural setting, and hence comparisons must be cautious.

We searched the official documentation for HTA procedures for supporting P&R available from public websites of the relevant country agencies during July 2018. This was supplemented by country profiles from the World Health Organization or the Organization for Economic Collaboration and Development. We cross-checked and filled in gaps from two recent reviews.8,10 For the comparator countries we included only documents in English. This was not an important handicap as these countries include extensive English documentation on their websites. For Spain, we included documents in English or Spanish. We examine how the health service is financed and organized, the purpose and timing of the P&R decision, the HTA procedures supporting that decision, and the criteria used. Results are presented as summary tables and narrative synthesis.

Table 1 gives a short description of the financing and organization of healthcare services in each country.17–21Table 2 summarises the timing and purpose of HTA in each country, and Table 3 describes the criteria used to make P&R decisions. Spain and France use HTA to support decisions for P&R before any medicine can be sold in the country. Sweden only uses HTA to support P&R for outpatient medicines. England allows free-pricing (with profit control), and conducts HTA to support reimbursement decisions, while Germany operates free-pricing with full reimbursement for up to 12 months, with HTA supporting subsequent P&R decisions.

Historically, Spain has been slow to implement the legal and institutional measures to formalise HTA, with the result that, in comparison with other countries, the P&R process lacks clear criteria and allows excessive discretionality.2,3 Spain was one of the first countries to set methods guidelines for economic evaluation within HTA, but these were never officially adopted.41 Nevertheless, considerable progress has been made in recent years since the establishment of the clinical HTA reports (IPT) in 2013.42 We make specific recommendations for further development with the objective of improving transparency, governance, economic evaluation and efficiency (summarized in Table 4). In each case, the recommendation is justified by comparison with other countries and the expected benefit arising from implementing the change. We have aimed to be objective and constructive in our criticisms, though of course the recommendations are grounded in the knowledge and experience of the authors.

Transparency allows public scrutiny and democratic oversight of technocratic decisions. Drummond et al. recommend that the HTA process is conducted independently of the body that will ultimately be responsible for adopting, paying and implementing the decision (the health insurance funds or national health service).43 The HTA bodies in England, Sweden and Germany have greater legal autonomy than France and Spain, where the institutions are agencies of the MoH. Spain is the only country among those reviewed in this paper where the P&R decision is made by a body that includes appointees from outside Health, that is, the ministries of Finance, Industry and Economy. Reference is made in the documentation to the “contribution of the manufacturer to gross domestic product”,22 but the meaning of this concept is vague. As there is little transparency about how P&R decisions are actually made, it cannot be ruled out that favourable terms are being given to companies with a manufacturing or research base in Spain. This practice may contravene European competition law, as well as being against the interests of Spanish consumers and tax-payers.3 Governance could be improved by strengthening the independence of the bodies charged with producing supporting HTA data, freeing decision-making bodies from political influence,44 and separating healthcare decisions from industrial policy.

Transparency is far from perfect in other countries. HAS, NICE and IQWIG/G-BA publish most of the technical reports used to make decisions. Nevertheless, “commercially confidential” information is censored. The TLV only publish a “summary of decision”, and gives companies the opportunity to withdraw rather than publish a negative decision. AEMPS in Spain publishes the HTA report (IPT) but do not publish other documentation used by the CIPM to make the decision. After the decision is made, the IPT is amended to state whether the medicine is financed or not in the NHS. However, specific reasons are not usually elaborated. A more transparent process would relate the decision more closely with the evidence.

While all countries publish the official price, they often negotiate confidential discounts. Pharmaceutical firms ask for transparent P&R criteria in order to correctly align their long term investment decisions with payers’ priorities.45 But at the same time, manufacturers ask for discounts to remain confidential, because many countries set prices for medicines by comparing against the official prices paid in other countries (international reference pricing). Therefore Spain is not an “outlier” in withholding this information. The lack of transparency makes it difficult to assess whether value for money is being achieved in public procurement.

Other countries are more transparent than Spain in procedures, decision making criteria and the involvement of stakeholders. Procedures and documentation could be made clearer. The reasons for decisions and areas of uncertainty should be published. Stakeholders, patients and the public should be encouraged to participate and be consulted on draft versions of the decisions. NICE has made considerable progress in involving stakeholders and might be considered a reference.7

There are currently no formal criteria for linking price to therapeutic value in Spain. Other countries use formal scales for defining health gain relative to a comparator (ASMR in France, Added Benefit in Germany, QALY gain in Sweden and England), and to a greater or lesser extent, the price of the medicine is related to this measure of health gain. The clinical HTA reports (IPT) produced by the agencies in Spain are of a high methodological quality, but the health gain is not mapped to a generic scale as elsewhere. The use of such a measure would improve efficiency and transparency of pricing, by relating the price to health benefit in a more consistent way across different pathologies and indications.

The law in Spain requires cost-effectiveness analysis to be taken into account, but does not say how this should be applied. This ambiguity about the role of economic evaluation is not unique to Spain; France requests cost-effectiveness analysis, but it is unclear how this impacts on the decision. German decision makers appear reluctant to use it, apparently based on ethical and legal concerns about “monetizing” health.27 Guidelines for hospital formularies in Spain recommend a fairly simple cost-effectiveness and budget impact methodology.25 Academic papers propose more sophisticated methods,46 some recommending an explicit cost-per-QALY threshold.5,47–49 It is recommended in health economic analyses to use a long time horizon, and incorporate real-world effectiveness data, rather than only short-term efficacy. However, there may be a trade-off between ensuring rapid access to new medicines and having the time to undertake sophisticated modeling.

All countries aim to make prompt decisions to ensure timely access to new medicines. Nevertheless, there are important differences between countries in the purpose of HTA, and this influences what type of HTA is most appropriate. In France and Sweden, as in Spain, the price at which any medicine can be sold must be determined by the relevant national authority before the medicine can be marketed. The 180 day timetable set by the EC directive aims to ensure prompt access, but may also put practical limits on the extent of analysis and modelling that can be undertaken (Fig. 1). To some extent, the free-pricing model followed by England and Germany allows immediate access by patients to new medicines in these countries, while allowing time for HTA agencies to collect more data and conduct a thorough set of analyses. However, in practice providers in England are reluctant to prescribe before NICE has issued guidance, and NICE has been under pressure to speed up decision making.50

Figure 1.

Purpose and timing of health technology appraisal. EMA: European Medicines Agency (or equivalent national body); HTA: health technology appraisal; RCT: randomized controlled trial; RWE: real world evidence.

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All the countries in this review ask the manufacturer to submit a dossier of evidence. In Sweden and Germany the HTA agencies review and comment on the manufacturer's dossier rather than conduct new analyses in-house, and this is increasingly the case for P&R in England as well. The advantage of this approach might be quicker P&R decisions and less cost for the HTA agency, while the limitations are that decisions are made in an incremental, piecewise manner as each new product comes on to the market (rather than compare all options head-to-head), and perhaps allows the manufacturer greater discretion about what to include in the dossier or how to interpret this data. The risk of conflict of interest is particularly acute in the case of economic evaluation which requires assumptions and judgments about what to include in the model. If manufacturers are charged with preparing the clinical and economic evidence, then the decision maker must be prepared to reject a manufacturer's submission (and hence, possibly, reject the application for P&R) if it falls short of the required methodological rigour. A high level of transparency is needed to ensure proper democratic oversight. In any event, Spain needs to invest in sufficient researchers with the necessary skills to review these clinical and economic evaluations and to strengthen substantially the technical staff supporting the national P&R committee. It is noteworthy that currently the regional HTA agencies (AETS) produce and publish evaluations of a wide range of healthcare programs (mainly devices and diagnostic technologies),51 but play almost no part in the P&R process for new medicines. Agencies in other countries conduct HTA for all types of therapies and technologies, in accordance with recommendations for best practice.43

AEMPS conducts clinical HTA, but does not evaluate costs or cost-effectiveness. One reason given for this is that the price is unknown, as the clinical HTA report precedes the P&R decision. However, this does not have to be an obstacle. HTA agencies in Sweden, France and England conduct or review cost-effectiveness analyses using the price the manufacturer is asking for. Another reason given is that discussion of cost is outside the remit of AEMPS. We recommend that full HTA of costs and benefits of new medicines should be coordinated within the regional Network, supported by AEMPS.

HTA needs to be appropriate for the decentralized Spanish health system. An important element of P&R process is whether the decisions are advisory or binding on the health service. In Spain, although the CIPM makes a central P&R decision, the 17 regions and hospitals exercise some discretion, particularly for inpatient medicines. Hence the nationally set price in Spain is the “maximum price”, and a positive reimbursement decision at national level is necessary but not sufficient to ensure access for all patients.

If regions and local hospitals are making independent decisions, then this should avoid duplicating technical tasks (for example, systematic review or model construction). With this in mind, coordination among the different HTA agencies in Spain (including AEMPS and AETS) might be strengthened. More research is needed about the impact of decentralized decision making on inequality in access.52

Clara Bermúdez-Tamayo.

The corresponding author on behalf of the other authors guarantee the accuracy, transparency and honesty of the data and information contained in the study, that no relevant information has been omitted and that all discrepancies between authors have been adequately resolved and described.

D.E. conceived and designed the study. Both authors reviewed the literature, analysed the data, drafted the manuscript and authorised the final version.