2.1.1. Effectiveness in the SBG V

The Fifth Book of the German Social Code (SGB V) regulates the Statutory Health Insurance (SHI) system, with the legal provisions governing the provision of SHI benefits evincing a certain terminological confusion. Specifically, there is an overlap between the notions of effectiveness, cost-effectiveness, quality, medical benefit and medical necessity.

Section 12(1) sentence 1 SGB V sets out general requirements that benefits must be ‘sufficient, appropriate and cost-effective; they must not exceed what is necessary’. Here, appropriateness can be understood as a distinct effectiveness requirement (Wagner, Reference Wagner2024; Roters, Reference Roters2010). However, Section 2 SGB V establishes another general provision for mentioning cost-effectiveness, quality and effectiveness separately – without referring to appropriateness – stating that “the quality and effectiveness of benefits must correspond to the generally recognised state of medical knowledge” (para. 1). Paragraph 4 adds that all actors must ensure that “the services are provided effectively and cost-effectively and are only utilised to the extent necessary.” In view of these overlaps, one must separate the concepts of effectiveness, cost-effectiveness, and quality in the German legal terminology (Huster, Reference Huster2013):

1. – Effectiveness means the capacity of a therapeutic intervention to bring about a defined clinical outcome.

2. – Cost-effectiveness refers to the relationship between costs and benefits (Meesen et al., Reference Meesen, Becker, Schön, Schlegelmilch, Vilaclara and Neueder2011; Bockholdt, Reference Bockholdt2024). In SHI law, this usually (only) means that, if two treatments have the same outcome, the cheaper one should be chosen. More general assessments relating to rationing are not yet relevant.

3. – Quality refers to the characteristics of a healthcare intervention. Insofar as it is not only related to structures and processes but also to outcomes (Donabedian, Reference Donabedian1966), it could require a minimum level of effectiveness. A connection to cost-effectiveness is likewise plausible (Bockholdt, Reference Bockholdt2024) though not conceptually needed. In lieu of a detailed definition, the legislator relies on the “generally recognised state of medical knowledge”.

While cost-effectiveness and quality are requirements concerning how a therapeutic intervention is implemented, effectiveness determines whether it should be delivered at all.

Among the criteria to be used in the assessment of diagnostic and treatment methods, Section 135(1) SGB V also uses the terms “medical benefit”, alongside “medical necessity” and “cost-effectiveness”. According to the explanatory information of the Federal Joint Committee (GBA), “effectiveness” constitutes the first sub-criterion of “medical benefit” (GBA, 2013). The concept of “medical benefit” would place two specific qualitative requirements on effectiveness (Roters, Reference Roters2010), one related to the specific patient-oriented output (i.e. the benefit for the quality or duration of individual patients’ lives, see Buyx et al., Reference Buyx, Friedrich and Schöne-Seifert2009; Köbberling, Reference Köbberling2022) and one concerning the overall care situation. Comparable questions surround the use of the term “medical necessity”, which is mentioned in Section 92(1) SGB V (Schöne-Seifert et al., Reference Schöne-Seifert, Huster, Harney and Friedrich2022). This similarly presupposes effectiveness, and may relate to the individual or to society more widely, but concerns the scope of the benefits that are to be granted.

2.1.2. Constitutional framework

Notwithstanding this terminological ambiguity, it is ultimately beyond doubt that a requirement of effectiveness exists. This result is supported by the constitutional framework that shapes the German SHI: by Art. 2(2) Basic Law (Grundgesetz - GG) for health protection (the right to physical integrity), by Art. 20(1) GG, framing the functioning of the SHI (the social state principle), and by Art. 12(1) GG, framing the activities of service providers (occupational freedom). These principles must be balanced, with effectiveness going most directly towards patient safety (and the right to physical integrity) and with cost-effectiveness going towards financial viability (the functioning of the SHI). However, effectiveness also protects against inappropriate expenditure and thus a misuse of the SHI’s financial resources. In addition, a certain openness of healthcare systems to innovation requires efficacy assessments, as will be discussed below.

Finally, as laid down by the Federal Constitutional Court, effectiveness constitutes the overarching requirement of a contribution-funded, mandatory insurance system which must, overall, be effective (Federal Constitutional Court decision of 6 December 2005, 1 BvR 347/98; Becker, Reference Becker2010). The right to obtain certain healthcare services was placed on two constitutional foundations – with the state’s protective obligations (Art. 2(2) GG) being significantly reinforced by the state compelling insured persons to join the SHI (Art. 2(1) in conjunction with 20(1) GG).

2.2.1. Effectiveness in the French code of social security

Effectiveness is a central criterion in determining the eligibility of drugs, medical devices and healthcare procedures for reimbursement by the French national health insurance. However, the precise definition, scope and impact of this criterion vary depending on the category of intervention being assessed. This differentiation stems from the structure of the benefit reimbursement system, whereby health technologies are included in different reimbursable lists under conditions stated in the Social Security Code (CSS). The assessment of effectiveness plays a key role in determining whether a technology can be included in any of the lists. Effectiveness is typically measured as one component of the actual or expected ‘clinical benefit’ (service rendu or service attendu respectively), referred to as ‘medical benefit’ (service médical rendu) in the case of medicines. This concept broadly reflects the potential of technologies to improve patients’ health status. For all health technologies, this assessment contains three components:

1. – Benefit-risks balance

2. – Comparative evaluation

3. – Value to public health system

Each component is specified differently depending on the technology in question. For medicines, Art. R163-3 CSS stipulates that the assessment of the medical benefit shall take into account: (i) effectiveness (efficacité) and adverse effects, (ii) the severity of the condition, (iii) its place in the therapeutic strategy in relation to other available therapies, and (iv) its public health interest. For medical devices and aids used directly by the patient, Art. R165-2 stipulates that expected benefit shall be evaluated in terms of: (i) therapeutic, diagnostic, or disability-compensating effect and adverse effects or risks associated with its use, (ii) taking into account other available therapies or means of diagnosis or compensation and (iii) its expected public health value, considering the severity of the condition and its impact on the healthcare system and on public health policies more widely.

Particularly the first component, that evaluates the benefit-risks balance, depends on clinical effectiveness. However, effectiveness is also relevant to the comparative evaluation, which measures the added effectiveness in relation to existing technologies. The second and third components (comparative evaluation and public health value, respectively) link the assessment of the technology’s quality and effectiveness to the principles of cost-effectiveness and rationing.

A distinctive feature of the French system is that the effectiveness assessment influences not only the reimbursement eligibility decision, but also the economic conditions under which the technology is covered. While a sufficient level of effectiveness is necessary for listing on a reimbursement list, comparative effectiveness impacts the technology’s reimbursement rates and pricing.

2.2.2. Constitutional framework

The integration of effectiveness assessments into reimbursement decisions is supported by French constitutional law. A constitutional right to health protection can be derived from Paragraph 11 of the Preamble of the 1946 Constitution. This provision declares that ‘the nation guarantees to all […] the protection of health’. This right is specified as a fundamental right to health protection in Art. L1110-1 of the Public Health Code (CSP), requiring that the state guarantees ‘equal access for everyone to the care required by their state of health, continuity of care and the best possible health security’. Moreover, effectiveness is linked with the protection of health in Art. L1110-5 CSP, insofar as it is recognised that ‘everyone has the right […] to receive the most appropriate treatment and care […] and to benefit from recognised effective therapies that guarantee the best possible health […] in view of established medical knowledge’ and that ‘preventive, investigative, treatment and care measures must not, given the current state of medical knowledge, expose them to risks that are disproportionate to the expected benefits’.

However, the constitutional right to health protection must be balanced with other constitutional limitations, such as the need to control health expenditure (Apollis and Truchet, Reference Apollis and Truchet2024). As, under Art. 34 of the Constitution, it is the law that shall determine the fundamental principles of social security, striking this balance remains subject to the legislature’s discretion. Since the constitutional revision of 22 February 1996, the financial equilibrium in social security has been explicitly recognised as a constitutional requirement. The Constitutional Council Decision of 12 August 2004, no. 2004-504 confirms the legislature’s ample discretion in reconciling the protection of health with the financial equilibrium of social security. The Council underscored that the aims of improving the quality of care and reducing the financial imbalance of the health insurance are legitimate aims that the legislature can pursue through proportional means.

In sum, for reimbursement decisions, the legislature must provide for an assessment of health technologies’ clinical benefit. Not only does this ensure adequate health protection in view of the established medical knowledge, as set out in Art. L1110-5 CSP, but it also guarantees the financial equilibrium of social security by focussing expenditure on effective healthcare.

2.3.1. Concepts that shape the assessment

Clinical effectiveness shapes which healthcare products and services are covered by England’s National Health Service (NHS). Under the statutory framework, decision makers must determine which services should be commissioned (reimbursed) and for these determinations clinical effectiveness is one relevant consideration.

This is evident in relation to the National Institute for Health and Care Excellence (NICE). NICE (1) makes binding recommendations on health services that must be reimbursed and (2) issues persuasive, guidelines on reimbursement (Section 237 Health and Social Care Act (HSCA) 2012). NICE must have regard to ‘the broad balance between the benefits and costs of the provision of health services’, ‘the degree of need of persons for health services’ and the ‘the desirability of promoting innovation in the provision of health services’ (Section 233(1)(a-c) HSCA 2012). Clinical effectiveness, ‘[t]he extent to which an intervention produces an overall health benefit, taking into account beneficial and adverse effects, in routine clinical practise, ‘is relevant to the implementation of these principles (R (Bayer plc) v NHS Darlington CCG [2020] EWCA Civ 449; NICE, 2018). Similarly, clinical effectiveness is considered by the integrated care boards (ICBs) and NHS England, as a part of their function in arranging for the provision of health services.

Effectiveness’ assessment requires a specification of the ‘benefit’ and of the ‘need of persons for health services’ (Section 233(1)(a-c) HSCA 2012). Here, NICE has regard to the health condition and populations that a technology is used for, as well as the clinical setting that it is deployed in. The evaluation will turn on the measurement of ‘health benefits and adverse effects that are important to patients and their carers’ (NICE, 2022a). Patients’ length of survival and their health-related quality of life are particularly relevant factors. But clinical outcomes are understood broadly, encompassing: ‘informational outcomes of value to the patient for the relief, or imposition, of anxiety or for personal planning’, ‘health-related behaviours such as anxiety and depression’ and a technology’s ability to improve patients’ progression through the clinical pathway (NICE, 2022a).

Clinical benefit is, further, defined in a manner that is relative to established practise. First, the evaluation will be made in comparison to a baseline of established interventions (or non-interventions) that are routinely used in the NHS. Second, it will be considered how closely adduced studies reflect routine clinical practise in England (NICE, 2023).

Separately, the evaluation accounts for the possibility that the technology may eliminate unlawful discrimination, advance equality of opportunity and foster good relations between people with protected characteristics and society as a whole. For eg., by addressing conditions that are more common in disadvantaged groups (NICE, 2022a).

The described evaluation is closely linked to NICE’s cost-effectiveness analysis. For this, separate standards are provided on gathering and analysing evidence and health benefits will be equated with so-called quality-adjusted life years (QALYs). In the end, this wider evaluation standardises the benefits obtained from health services and seeks to maximise the health gains that can be obtained from a fixed NHS budget.

2.3.2. Overarching normative framework

Within England, the outlined legal framework is not so much influenced by substantive constitutional provisions, as by overarching procedural requirements (see Domenici, Reference Domenici2023). Partially, these are laid down in the NHS Constitution. Despite its name, this is merely a declaratory document that does not establish new legal rights. Rather, it sets out a number of principles and values that all NHS bodies must have regard to in their decision making (Section 2 Health Act 2009). In particular, it establishes that patients have a right to receive clinically appropriate drugs and treatments, which have been recommended by NICE, and to expect local funding decisions to be made rationally following a proper consideration of the evidence. As has been stated, the definition of effectiveness is thereby left to a number of parties, fulfilling different functions.

This has not been without difficulties, as is illustrated by R (British Homeopathic Association) v NHS Commissioning Board ([2018] EWHC 1359). Here, NHS England had carried out a consultation and issued guidance that turned on the ‘efficacy’ of homoeopathic treatments. Although the court recognised that ‘efficacy’ and effectiveness’ are distinct, the consultation conflated the two terms: purportedly asking whether the treatment ‘is clinically effective’ to determine its efficacy. Simultaneously, consultees were asked to consider a report that rejected homoeopathy’s efficacy, even though it did not completely reject its effectiveness. As a result, the claimant argued that the consultation had been unfair – it did not ‘permit of intelligent consideration and response’. In spite of the recognised problems, the court rejected this challenge. Consultees were likely to have understood the true meaning of the two terms and, in any case, all consultees understood that they had to respond to the alleged ‘lack of robust evidence that [homoeopathy] actually works’. They could provide whatever evidence they deemed appropriate to this, more general question.

Overarching procedural obligations can further be derived from the broader principles of administrative law. These require decision makers to engage with the evidence on efficacy and/or effectiveness when they determine which health services the NHS ought to offer. In relation to health technology assessments, NICE has been obliged to determine whether sufficiently strong evidence as to efficacy had been submitted or not and to state its conclusion (R (Servier Laboratories Ltd) v NICE [2010] EWCA Civ 346; Syrett, Reference Syrett2024). Similarly, where health services are commissioned ‘it is vital for an authority: (1) accurately to assess the nature and seriousness of each type of illness; (2) to determine the effectiveness of various forms of treatment for it; and (3) to give proper effect to that assessment and that determination in the formulation and individual application of its policy’ (R v North West Lancashire Health Authority [2000] 1 WLR 977). Furthermore, a higher justificatory burden is placed on commissioning authorities who depart from non-binding NICE guidelines (R (Rose) v Thanet CCG [2014] EWHC 1182). Since a competent authority has already considered effectiveness, ‘clear, lawful reasons’ must be given for this departure.

While effectiveness is therefore a real constraint on reimbursement decisions, decision makers retain a great deal of discretion. As was stated in R (Bayer) v NHS Darlington CCG, a commissioner of health services ‘is entitled to take its own view on the clinical effectiveness of a particular medicine or procedure in setting its policies and making commissioning decisions’. In short, that effectiveness must be accounted for is certain under English law; how it must be accounted for will often be influenced by the executive’s discretion.

3.2.1. Germany

Institutionally, the most important question in Germany is whether and to what extent the specification of the general statutory requirements may be left to the Federal Joint Committee (GBA). This committee is part of the ‘joint self-administration’, a specific form of corporatism bringing together the conflicting interests of insurance providers and service providers to arrive at a legally binding resolution that reflects a balance of their interests. To this end, the SGB V establishes a complex system of collective bargaining and statutory instruments that convert the statutory rights to medical treatment into more concrete forms. The GBA is at its centre, deciding on the admission of new methods of medical treatment and possessing the power to enact legally binding provisions comparable to statutory instruments (Richtlinien). It is mostly made up of representatives of sickness funds, as well as representatives of physicians and hospitals (see Section 91 SGB V).

As the members’ mandate is not based on governmental decisions, the Committee’s democratic credentials are perennially contested (Kingreen, Reference Kingreen2017). In consequence, relatively strict legal requirements govern the committees’ decisions. Nevertheless, administrative committees have discretion when fulfilling their statutory tasks and the GBA may, therefore, decide on the effectiveness of health services based on its own assessment, decoupling it from direct political influence.

The German legislature does not rely solely on the joint self-administration’s expertise. Rather, the Institute for Quality and Efficiency in Health Care (IQWIG) was founded in 2004 as a private law foundation to ensure the inclusion of specialised expertise, the greatest possible neutrality and to address ‘issues of fundamental importance for the quality and cost-effectiveness of services provided under the SHI’ (Section 139a(3) SBG V). IQWIG ‘must ensure that the assessment of medical benefits is carried out in accordance with internationally recognised standards of evidence-based medicine and that the economic assessment is carried out in accordance with the relevant internationally recognised standards, in particular those of health economics’ (Section 139a(4) SBG V). It must also report publicly at regular intervals on its work processes and results, including the basis for its decisions. IQWIG does not take any external action itself, but provides the technical basis for the GBA’s decisions and it is the GBA that bears sole legal responsibility for these (Section 139b(4) SGB V).

3.2.2. France

The National Health Authority (Haute Autorité de santé, HAS) conducts HTA as an independent public institution with a scientific focus and financial autonomy (Art. L161-37 CSS). Established in 2004, the HAS is mandated to carry out specific missions and reports annually to the government and Parliament. Its board members are selected based on their expertise and competence by the President of the Republic, the Ministries of Health and Social Security, the President of the National Assembly, the President of the Senate and the President of the Economic, Social and Environmental Council (Art. L161-42 CSS). HAS strives for scientific rigour, independence and transparency.

HAS is divided into nine specialised commissions responsible for various activities within its areas of competence. The Transparency Commission (Commission de la Transparence) assesses drugs for statutory health insurance reimbursement, while the Medical Device and Health Technology Evaluation Committee (Commission nationale d’évaluation des dispositifs médicaux et des technologies de santé, CNEDiMTS) evaluates medical devices and procedures. In addition to the CNEDiMTS members, the chair may call upon external expertise, particularly in cases involving unrepresented professional specialities.

HAS’s primary role is to provide recommendations to the French Ministries of Health and Social Security regarding the reimbursement of health technologies. For drugs and medical devices used directly by patients, lists are established by ministerial decree and published in the Official Journal. For products used in medical procedures, reimbursement depends on the list of healthcare acts and services performed by professionals. This is adopted by the National Union of Health Insurance Funds (UNCAM), following consultation with the HAS regarding the clinical benefit evaluation. These decisions are deemed to have been approved unless the Ministry of Health objects with a reasoned statement.

3.2.3. England

NICE is the organisation that plays the leading role in conducting HTAs in England. It is an independent public-law organisation: an executive non-departmental public body. Although it is sponsored by the Department of Health and Social Care (DHSC), it enjoys an outward-facing independence from it and, relatedly, from the NHS. Hence, Section 237(4) of the HSCA 2012 specifies that directions of the Secretary of State should not concern ‘the substance of advice, guidance or recommendations of NICE’, as well as in the framework agreement that has been concluded between the two (DHSC and NICE, 2018).

Furthermore, internal mechanisms ensure an objective selection and evaluation of the scientific evidence. NICE relies on independent advisory committees to develop its recommendations – with members being selected for their knowledge and experience from a range of backgrounds: the NHS, academia, related industries and organisations that represent health service users.

Decisions are made by using a mixture of ‘giving advice or guidance, providing information or making recommendations’Footnote ⁴ . With regard to their binding force, NICE’s Technology Appraisal Guidance (TAGs) shall be distinguished from NICE Guidelines. TAGs must, unless otherwise stated, be commissioned by the aforementioned NHS bodies within a period of 3 months after publication.Footnote ⁵ In comparison, the implementation of Guidelines is discretionary and dependent upon the prioritisation decisions of the relevant commissioners. Although, as mentioned above, the courts have required a meaningful engagement with NICE’s assessment even here.

3.3.1. Germany

Section 2(1) sentence 3 SGB V contains the legal determination of evidentiary requirements for efficacy. According to this provision, the ‘generally recognised state of medical knowledge’ is decisive: medical progress must be ‘taken into account’. In its established case law, the Federal Social Court (BSG) elaborates on this provision as follows: ‘In principle, the quality requirement entails that the vast majority of the relevant experts (doctors, scientists) are in favour of the treatment method and that, apart from individual, insubstantial dissenting voices, there is a consensus on the appropriateness of the therapy. As a rule, this presupposes that reliable, scientifically verifiable statements can be made about the quality and efficacy of the method - which must be assessed in its entirety and not just in relation to partial aspects. Success must be evident from scientifically sound studies on the number of cases treated and the efficacy of the method. The therapy must have been successful in a sufficient number of treatment cases for a reliable assessment’ (BSG, 16.08.2021, B 1 KR 18/20 R, par. 10).

Under Section 92(1) sentence 1 SGB V, the GBA has the authority to issue guidelines: it ‘may restrict or exclude the provision and prescription of services if, according to the generally recognised state of medical knowledge, the diagnostic or therapeutic benefit, medical necessity or cost-effectiveness have not been proven’. Under its Rules of Procedure, its task is to ascertain ‘the generally recognised state of medical knowledge on the basis of evidence-based medicine’ (Section 5(2) 1st Chapter). The BSG has highlighted the importance of this link to evidence-based medicine by connecting the consensus of the relevant experts with scientifically sound studies. In some cases, it refers to the ‘evidence-based consensus of the vast majority of relevant experts’ (BSG, 13.12.2022, B 1 KR 33/21 R, par. 16). Similarly, under Section 139a(4) SGB V IQWIG is responsible for ensuring the medical benefit is assessed in accordance with internationally recognised standards of evidence-based medicine.

3.3.2. France

In delegating HTA to HAS, the Code of Social Security does not specify concrete evidentiary standards other than the general need for clinical benefit. However, to fulfil the principle of transparency, HAS’ commissions must specify the procedures and principles according to which they implement the criteria for evaluating health services under Art. L161-37 CSS. These are contained in the methodological guides issued by HAS and its sub-committees.

For medical devices used by the patient directly, the methods and conditions for a high-quality clinical evaluation are listed in CNEDiMTS’ guides ‘Methodology for the clinical development of medical devices’ (HAS, 2021) and ‘Assessment principles for medical devices for individual use to determine their eligibility for reimbursement’ (HAS, 2025). The latter states that all claims made by manufacturers regarding data must be scientifically demonstrated, with the data analysed according to the criteria of evidence-based medicine. HAS recognises double-blind, randomised controlled trials as the gold standard. However, the commission takes into account that these are sometimes difficult to implement for medical devices and allows for different types of evidence. These should be supported by an explicit scientific rationale. The document ‘Transparency Committee doctrine: Principles of medicinal product assessments and appraisal for reimbursement purposes’ (HAS, 2023) provides guidance on the criteria used to evaluate medicines for reimbursement purposes. It states that the assessment is based on an analysis of all the available clinical data for the indication being evaluated at a given time. Therefore, this assessment is provisional and may change in light of new effectiveness and safety data.

3.3.3. England

As in Germany and France, the evidentiary standards that have been devised to carry out HTA do not follow directly from the statutory framework, but from an exercise in delegated decision making. Hereby, the technical standards that NICE applies are found in the various forms of guidance that it issues – most especially TAGs and Guidelines.

NICE has stated that it will consider all types of evidence, including even unpublished evidence, non-UK sources, databases of ongoing clinical trials, and expert elicitation (NICE, 2022a). In the course of the appraisal, a systematic review of the relevant evidence on any given technology should be carried out, minimising the risk of selection bias, but in an iterative way; beginning with sources of evidence that are preferred by NICE, most especially RCTs, and being expanded, first, to more robust forms of non-randomised evidence and then less reliable forms. In how far one must go beyond the scientifically ‘preferred’ types of evidence, will depend on the limitations of relevant RCTs, as well as the residual uncertainties and on the practicalities of the evidence search.

NICE therefore employs a hierarchical approach, with some forms of evidence being, on balance, regarded as possessing a high validity and other forms (e.g. elicitation of expert opinion) generally being treated more cautiously, due to their susceptibility to uncertainty and bias. Hence, no type of evidence is accepted as such, but is subjected to a context-specific critical appraisal.

4.1.1. Individually justified

It is widely accepted that German health insurance law provides for certain ‘exceptions’ to the prerequisite of effectiveness. First, there is a certain need for individualisation: Evidentiary standards refer to typical situations, and they may have to be adopted to individual characteristics. The BSG considers an exception to the obligation to comply with general requirements to be possible and necessary not only in the event of ‘system failure’, but also in so-called rare cases (BSG, 03.07.2012, B 1 KR 25/11 R, par. 18 ff).Footnote ⁶ In addition, one must consider the cases of life-threatening illness, introduced by the decision of the Federal Constitutional Court on the right to healthcare services, and now addressed by Section 2(1a) SGB V.

In both cases, exceptions to the general evidentiary standards are well-founded and, indeed, constitutionally required. However, an exception is not synonymous with a complete renunciation of efficacy: at least the possibility of the desired effects must be plausibly demonstrated. Section 2(1a) sentence 1 SGB V requires ‘a not entirely remote prospect of a cure or of a noticeable positive effect on the course of the disease’. This wording, taken from the Federal Constitutional Court Decision of 6 December 2005, is relatively far-reaching and yet, it leaves the exact standards to be applied open (since then, they have only been partially clarified by the case law, on the developments see Roters, Reference Roters2015). Existing scientific knowledge must be taken into account, but the evidence requirements are also set in relation to the severity of the illness (Scholz, Reference Scholz2024). The outlined principles apply also in the case of their off-label use (BSG, 19.03.2002, B 1 KR 37/00 R, par. 16 ff; Becker and Wilman, Reference Becker and Wilman2012) although the bar is set relatively high, as the BSG requires ‘research results that suggest that the medicinal product concerned can be authorised for the relevant indication’ (BSG, 24.01.2023, B 1 KR 7/22 R, par. 16).

Further, one can infer from the function of individual exceptions that they are subsidiary. The justification for these exceptions lies precisely in the fact that the generally recognised methods and means cannot be applied. Therefore, not only must the individual circumstances that lead to this misalignment be proven, but priority must be accorded to the generally recognised methods and means. This subsidiarity limits these exceptions to a level that is sustainable for the system as a whole: their utilisation must always be justified for the individual case and the related requirements are considerable.

Section 73(3) of the German Medicinal Products Act (Arzneimittelgesetz, AMG) provides for an individual exception to the general ban on the importation of medicines that are not approved or registered in Germany.Footnote ⁷ However, unlike the individual exceptions mentioned before, this scheme does not even require that the plausibility of effectiveness is demonstrated and it operates outside of the statutory health insurance system, thus excluding any coverage of costs.

4.1.2. Collectively justified

The second exception concerns innovations. That there must be an openness to innovation can be derived from Section 2(1) sentence 3 SGB V, according to which the quality and effectiveness of services must take medical progress into account. This provision is also a specification of constitutional requirements, as the effectiveness of healthcare cannot be maintained in the long term without the inclusion of innovative forms of treatment.

That is why, unlike in the outpatient sector, new methods are not strictly prohibited in the inpatient sector. Still, they must be reviewed by the GBA, which assesses whether they are necessary for the adequate, appropriate and economical care of insured persons, taking into account the generally accepted state of medical knowledge (Section 137c(1) SGB V). If the review finds that the benefits of a method have not yet been sufficiently proven, but that it offers the potential of a necessary alternative treatment, the GBA decides on a guideline for testing in accordance with Section 137e (see Section 137c(1) sentence 3 SGB V). Furthermore, new methods for which the GBA has not yet made a decision, may be used as part of hospital treatment if they offer the potential of a necessary alternative treatment and their use is in accordance with the rules of medical practise, i.e. if they are medically indicated and necessary (Section 137c(3) SGB V).

This testing procedure is of twofold importance. In terms of its substance, it introduces a new standard, that of ‘potential’, which, in the opinion of the BSG, takes precedence over the general quality standard as lex specialis (BSG, 13.12.2022, B 1 KR 33/21 R, par. 16). In terms of its development, it has been marked by an unusually intense struggle between judicial and legislative decision makers (Becker, Reference Becker2024), and remains highly controversial in its interpretation (Deister and Felix, Reference Deister and Felix2022; Deister, Reference Deister2023; Felix, Reference Felix2023).

4.1.3. Special therapeutic approaches

A further deviation from evidentiary standards, specifically addressed in the SGB V, is difficult to classify under the outlined categories. This concerns the inclusion of therapeutic approaches that are outside ‘conventional’ medicine, such as alternative treatment methods, homoeopathic remedies and the prescription of cannabis products under Section 31(6) SGB V. According to Section 2(1) sentence 2 SGB V, special therapeutic approaches are not excluded from the benefits provided by health insurance funds. This does not address the question of the conditions under which they are included. From a systematic point of view, it is notable that quality and effectiveness are mentioned in the following sentence as general conditions, which would suggest that they also apply to measures of special therapeutic approaches (Scholz, Reference Scholz2024).

However, in the case of special therapeutic approaches one might argue that the general evidentiary standard is not appropriate. The BSG case-law emphasises how special therapeutic approaches are associated with an ideological approach (Weltanschauung) that distinguishes itself from conventional medicine (BSG, 19.02.2002, B 1 KR 16/00 R, par. 26). The BSG has not decided whether the ‘individually justified’ reasons for deviating from the decisions of the GBA (see above, a) are transferable. However, as the deployed legal terminology of special ‘therapeutic approaches’ already suggests, this exception is not concerned with individual cases but rather a question of taking into account fundamentally different ideological approaches to treatment – which would speak for the development of a suitable standard, comparable to the one for a collectively justified reduction of proof (above, c)). However, the aforementioned subsidiarity principle seems to apply to special therapeutic approaches. The BSG’s cannabis decision insists that ‘when using a treatment method for which there is not yet any reliable evidence regarding its application, efficacy and safety, … the protection of the patient requires one to choose the path of proven benefit’ which calls for consideration of all therapeutic alternatives (BSG, 22.03.2005, B 1 A 1/03 R, par. 35). Consequently, as supported by the overall system of the SGB V, priority must be given to methods for which there is a body of scientific evidence for their effectiveness.

4.2.1. Individually justified

In France, instruments of compassionate access are provided for cases involving serious, rare or rapidly evolving medical conditions, where delaying treatment until sufficient evidence is available could be clinically unjustifiable. These allow individual patients to benefit from innovative medicines before full market authorisation is granted. This ‘exceptional’ access is governed by Art. L5121-12-1 CSP, and requires that the product’s effectiveness and safety be strongly presumed on the basis of available clinical data. While the effectiveness requirement remains intact in principle, it is applied through a graduated logic that balances the demand for evidence with the need to ensure early access to potentially life-saving treatments.

Compassionate access covers two distinct frameworks, both within the responsibility of the French National Agency for the Safety of Medicines and Health Products (ANSM). The Compassionate Access Authorisation (Autorisation d’Accès Compassionnel, AAC) is intended for specific patients with rare conditions that cannot be treated with authorised medicines. It is granted on an individual basis, upon a request made by a prescribing doctor, typically for medicines without a national marketing authorisation and with no intention of obtaining it in the future. The Compassionate Prescription Framework (Cadre de prescription compassionnel, CPC) applies to situations where off-label use is considered appropriate. According to Art. R5121-76-1 CSP, the Director General of the ANSM may initiate the establishment of a compassionate access scheme, or start this procedure at the request of the Ministers of Health and Social Security. For both compassionate access schemes, the presumption of benefit must be grounded in available scientific data. As set out in Art. L162-16-5-2 and L162-16-5-4 CSS, the costs for medicines approved for compassionate access are fully covered by the national health insurance.

4.2.2. Collectively justified

Openness to innovation plays a major role in France (Dubromel et al., Reference Dubromel, Geffroy, Aulagner and Dussart2018; von Hardenberg et al., Reference von Hardenberg, Domenici and Grinblat2021). This is proven by the multiple special procedures existing for early reimbursement of various innovative services in different clinical settings. The forfait innovation, for instance, is a mechanism that can partially or fully finance, for a limited period, new medical devices and treatment methods expected to provide additional benefits (Art. L165-1-1 CSS). A condition for reimbursement is participation in clinical or other studies with data collection in order to obtain the missing evidence of benefit. Another prerequisite is the assessment of the potential, risks and possible general healthcare benefits by HAS, which has 15 days to review the formal requirements and 75 days for the substantive requirements (Art. R165-68 CSS). Clinical or medico-economic studies demonstrating either reduced expenditure or a significant clinical benefit in the form of a therapeutic, diagnostic or prognostic effect, thereby covering an unmet or inadequately met medical need, must already be available (Art. R165-63 no. 4 CSS). In 2019, the procedural deadlines were adjusted again and another mechanism was introduced to prevent innovations from failing due to the length of the procedure: the prise en charge transitoire. This provides advance payment for medical devices with a CE mark that are not yet on the list of reimbursable products (Art. L165-1-5 CSS). The requirement is that, based on the results of clinical studies, the products in question must demonstrate clinically relevant efficacy and a significant effect that is acceptable in terms of their side effects (Art. R165-90(I) no. 5 CSS). The Ministry of Health ultimately decides on whether either of these instruments are granted, basing its determination on the opinions of the relevant HAS commission. The reasons on which the Ministry may reject an application for the Forfait Innovation are set out in Art. R165-70 CSS.

Another instrument of early reimbursement is the recently reformedFootnote ⁸ Référentiel des actes Innovants Hors Nomenclature (RIHN) covering, among others, in vitro diagnostics procedures. This is a mechanism allowing for the reimbursement of innovative products that, based on available data, are likely to offer significant clinical or medico-economic benefits and meet a medical need that is not covered or only insufficiently covered (Art. R162-122 CSS). Reimbursement is subject to the collection of clinical or medico-economic data.

Finally, for medicines, a pathway for early access authorisation (authorisation d’accès précoce) has been designed for products that the manufacturer intends to market for a specific indication. This allows reimbursement to be provided on a temporary basis prior to obtaining marketing authorisation or formal inclusion in the reimbursement lists. Access is dependent on the absence of any appropriate therapeutic alternatives, the clinical urgency of the treatment and a strong presumption of effectiveness and safety based on clinical trials (Art. L5121-12 CSP). The medicine must also be considered innovative, particularly when compared to existing therapeutic options. In such cases, the ANSM first evaluates the presumption of effectiveness, while the HAS makes the final decision regarding eligibility.

4.2.3. Special therapeutic approaches

Up until 2019, homoeopathic medicines under generic names were reimbursed by the national health insurance at a rate of 30%. However, in 2019, the Ministry of Health asked the HAS Transparency Commission to issue an opinion on maintaining health insurance coverage for homoeopathic medicines (HAS, 2019). Effectiveness was one of the main criteria used in the assessment: HAS noted that for most of the health conditions listed, the effectiveness data available did not show statistically significant differences between homoeopathic medicines and placebos or comparators. Therefore, HAS concluded that effectiveness could not be demonstrated. Following this opinion, reimbursement for homoeopathy was phased out, with all measures removed from the relevant lists by 2021.

4.3.1. Individually justified

At the individual level, there are two sets of special provisions. The first special provision is made for the purposes of the HTA and establishes a pre-defined pathway that is applicable to highly specialised technologies (HSTs).Footnote ⁹ These are technologies that fulfil four criteria: (1) they are for ultra-rare and debilitating diseases – having a point of prevalence of 1:50,000 or less in England, having a life-long effect with current treatment(s) and having an exceptional negative impact on people, their families and carers, (2) the technology represents an innovation (3) no more than 300 people in England are eligible for the technology in its licenced indication, and (4) the existing clinical management of the ultra-rare disease is considered inadequate, while the new technology is likely to offer substantial additional benefit (NICE, 2025). The HST assessment pathway is explicitly deemed ‘exceptional’ and is justified on the basis that the covered technologies relate to a small number of patients, that there are limited or no treatment options for their conditions and that this generates considerable difficulties for research and for collecting evidence (NICE, 2022b).

The main purpose of HSTs is not to alter the evaluation of clinical effectiveness, but to specify more favourable rules for the ultimate cost-effectiveness assessment (NICE, 2017). That being said, HSTs are part of a restricted class of technologies where NICE may include a managed access arrangement (MAA) in its recommendation. These essentially hold that, if evidence on clinical effectiveness is either absent, weak or uncertain during the initial evaluation, then a technology may still be approved for use for a limited period, within which further data is gathered and then subsequently evaluated (NICE, 2017).

At the level of commissioning health services, an individual claim may be made for exceptional treatment through an individual funding request (IFR) against NHS England or an ICB.Footnote ¹⁰ Such a request is possible where there is no binding NICE recommendation that entitles that patient to receive a given treatment and the relevant commissioning body has not exercised its discretion to fund it (NHS England, 2023a). Here, a physician can apply for their patient’s treatment to be exceptionally funded if they consider that their individual clinical circumstances are so different from the typical patient population that general policies on funding ought not to apply. A panel must then consider whether the individual case is truly exceptional and whether the proposed treatment is likely to achieve the anticipated outcomes. Here, clinical effectiveness is assessed somewhat more leniently, as it is recognised that ‘Inevitably, the evidence base put forward in support of an IFR is unlikely to be as robust as in more common presentations of the condition or the more usual use of the treatment’ (NHS England, 2023a). The decision of the IFR panel can be reviewed under general administrative law principles, such as rationality (R (SB) v NHS England [2017] EWHC 2000).

4.3.2. Collectively justified

In carrying out its functions, NICE must generally have regard to ‘the desirability of promoting innovation in the provision of health services’ (Section 233 HSCA 2012). In addition to this general obligation, two specific funds have been created under which health services can be reimbursed without first applying the usual evidentiary requirements. Namely, funds for cancer drugs and for innovative medicines (NHS England, 2022). In these instances, NICE can recommend the conditional funding of the respective treatment.

Similar to the case of HSTs, this conditional use will be implemented within the framework of an MAA. The MAA ensures that the use of the relevant technology is monitored and that further data is collected for a pre-defined period of time (NHS England, 2022; NHS England, 2016). In principle, the requirements of clinical effectiveness (and cost-effectiveness) apply in these cases, but a degree of leeway is granted in terms of the intensity of the initial assessment (NICE, n.d.). This is because, almost by definition, this framework strives to overcome existing uncertainties – especially in relation to long-term clinical effectiveness (NHS England, 2022). Simultaneously, the evidence on which the evaluation is based should be able to identify those technologies that show ‘significant clinical promise’ or are deemed the ‘most promising’ in terms of the novel clinical benefit that they can provide (NHS England, 2022; NHS England, 2016).

4.3.3. Special therapeutic approach

There was a common practise for NHS commissioners to fund homoeopathic treatments, which was never subject to a NICE determination (House of Commons Science and Technology Committee, 2010). Only with NHS England’s issuance of guidance in November 2017, was this discretion limited, with homoeopathic treatments being decommissioned to this day and with similar restrictions applying to herbal treatments and other natural products (NHS England, 2023b).

Until this point, partial justifications had been advanced not subjecting homoeopathy to a conventional effectiveness assessment. For eg., the Government was clearly aware of the limited evidence base for the effectiveness of homoeopathy, but had stated a preference for leaving decisions to local commissioners and to individual physicians (House of Commons Science and Technology Committee, 2010). It was argued that this would best respect ‘the geographical, socioeconomic and cultural diversity in England, that involves a whole range of considerations including, but not limited to, efficacy’ and would enhance patient choice (Secretary of State for Health, 2010). However, following the executive’s change of strategy, these special therapeutic approaches can no longer be considered to be an exceptional class of health services.