Introduction 20 19 8 15 7 8 27 10 However, the fact that policy problems and associated solutions tend to shift over time, different actors with different background theories being involved successively, renders this rather complicated. In other words, a policy measure is not invented at a specific moment in time, but develops over time. Nevertheless, the challenge is to identify policy measures that cohere with the views of all actors involved. If the argumentative policy theory is correct, then it is advisable to identify the policy’s target populations and involve them in the process of policy development. An interactive process of policy making could thus ensure that policy coheres with and prevent that it diverges from the views of target groups. The objective of this paper is to analyse a specific instance where target populations were involved in the process of policy development. The policy institution in this example was the Health Care Insurance Board in the Netherlands (HCIB). It is an advisory board to the Ministry of Health, particularly with respect to coverage and reimbursement issues. The Department of Policy Analysis of Medicines (PAM) is responsible for identifying developments that may jeopardise optimal medical care, analysing the nature and size of such threats and conducting further research that may provide a basis for policy decisions. The annual work programme in which topics are prioritised is submitted to the Ministry of Health for approval. The policy objective discussed in this paper was to promote the appropriate use of a recently introduced drug for patients with multiple sclerosis. Multiple Sclerosis (MS) is a neurodegenerative disease characterised by neurological dysfunction. The drug interferon-beta (IFNβ) appeared to be a promising treatment. However, its costs were high (€ 12,000 per patient annually) and the evidence of its long-term effectiveness was limited. Therefore, policy measures to guide the prescription of this medicine were considered necessary. Representatives of prescribing physicians (neurologists), health insurance companies, and patients were invited to participate in two workshops. During these workshops, participants discussed policy measures that could promote the appropriate use of the new drug. Although two concrete policy measures had been proposed, these have never been implemented. In this paper we evaluated whether the theory of the argumentative policy analysis could explain the proceedings in this case study. It offers a description of the process of the policy development that included the two workshops, as well as an analysis of the views of policy makers and target populations in order to assess whether the proposed policy measures fitted the perceived problems and underlying background theories. Methods Relevant documents were analysed and semi-structured interviews were held with the various stakeholders. These documents included correspondence, reports from the Board, internal memos on this subject, a report from Health Council on interferon-beta, reports from meetings, research proposals, and research reports. Interviews were held with three HCIB employees, two employees from the Ministry of Health, the organisers of the workshops, two neurologists, a patient, and a medical advisor from a health insurance company. Participants of the workshops were contacted and interviewed to reconstruct the proceedings of the workshops. MS patients were contacted via a Dutch MS patient organisation. Representatives of target populations were interviewed to assess their problem definitions and underlying argumentation. 10 11 18 Results Proceedings of Project The content of the workshops was, to a large extent, determined by two reports concerning IFNβ. These had been issued by the HCIB and by the Health Council, respectively. In July, 1995, the Ministry of Health asked both institutions to provide advice regarding the introduction of IFNβ on the Dutch market. The Ministry asked for recommendations with respect to whether or not restrictions could be imposed on the reimbursement of the drug. 9 30 In 1999, the department of Policy Analysis of Medicines (PAM) started a project on IFNβ. Initially it they considered evaluating other, less expensive interventions that could be effective, but are of no interest to the industry. Indications had been received from the field that a much cheaper drug, namely methotrexate (used in oncology and rheumatoid arthritis), could be as effective as interferon. Furthermore, PAM considered developing a national database of MS patients. PAM later decided that conducting a clinical trial was not its responsibility. PAM staff, in collaboration with neurologists, decided to initiate the development of a treatment protocol and a national registry of MS patients. They proposed the organisation of two workshops. Workshops 4 2 2 Proposed Policy Measures 5 Have neurologists develop an evidence-based guideline for the treatment of patients with multiple sclerosis. Have reimbursement requests for IFNβ appraised on medical grounds. Possibly, implement a central pre-utilisation approval by experts (neurologists). Define clear criteria for (dis)continuing IFNβ reimbursement. Establish a national registry of MS patients. The database should enable scientific research, the improvement of treatments, monitoring, and the evaluation of efficiency and therapeutic value of new interventions. In 2003, a medical advisor from a health insurance company mentioned that some small changes had been made to the procedures established for judging reimbursement requests (requests for continued use). A neurologist mentioned working on a guideline for diagnosis and treatment of MS patients. According to a PAM staff member, neither a central pre-utilisation approval nor a national database to prospectively register patient data had been established. Reconstructed Interpretative Frames 1 2 Table 1 Views towards guideline concerning IFNβ use Actor a Problem definition Background theories Normative values Ministry of health (1996)   Only if evidence of effectiveness is available, a medicine can be at the expense of the community   PAM staff       PAM staff (2003)       Medical advisor health insurance company  Unclear when IFNβ is (still) meaningful   Cost control  Neurologist  Increase possibilities for diagnosis and therapy; variance in MS care As in other fields increasing attention to evidence based medicine  Provide clarity for neurologists on how to care patients Patient  Differences between hospitals care (IFNβ not offered in every hospital; contradictory advises on lifestyle)  Improve or maintain quality of life a Table 2 Views towards a national registry of MS patients or IFNβ users Actor a Problem definition Background theories Normative values Ministry of health (1996)  Uncertainty on (meaning of) effects of IFNβ  Efficiency PAM Staff      Aim of national database to optimise IFNβ prescription: to monitor use of IFNβ in practice (which patients (indications), volume, costs) and to evaluate effectiveness IFNβ  Efficiency  PAM Staff (2003)   Central pre-utilisation judgement provides a minimal registration of patient data  Efficiency  Medical advisor of health insurance company  Judgement of relative value as compared to alternative interventions not systematically but only in the neurologists head   Neurologist      a Ministry of Health Initially, the Ministry contended that the long-term effectiveness of the drug was not established sufficiently, while the acquisition costs were high. Trials had shown that IFNβ decreased the number of exacerbations, but it was unknown whether IFNβ would prevent disability. The potential target population, namely patients with MS, is large and the expectations of both patients and physicians may also have been high. As a result, the risk that the drug could be used inaccurately was considered to be high. Consequently, additional policy measures to control IFNβ use in clinical practice were requested. 5 PAM Staff According to the PAM staff, the main problem was that the use of IFNβ was expected to extend to other subgroups of patients. A central pre-utilisation approval in combination with a national registry of MS patients could provide a solution. This contention was made because the situation concerning IFNβ resembled the situation concerning other drugs for which a central pre-utilisation approval had already been established. In that case, requests for reimbursement of drug use were judged by a central committee of medical experts. For this purpose, a clear protocol including criteria for IFNβ use is needed. Simultaneously, patient data were recorded in a national registry. At the time that PAM staff started their project, it was undesirable to evaluate why the proposed policy measures had not been implemented until then. Reason was a change in the relationship between the HCIB and the Ministry of Health. In 1999, the HCIB, an independent advisory board, was established as the successor to the Sickness Funds, which was a politically involved advisory board comprised of actors from the field. After completion of the project, the PAM staff considered the option of having reimbursement requests judged by a central committee no longer meaningful. They considered the implementation of policy measures difficult because the drug had already been introduced several years earlier. Limiting or discontinuing the reimbursement of IFNβ would have been practically impossible. Furthermore, the scale of problems relating to the prescription and/or reimbursement of IFNβ and the costs related to IFNβ use turned out to be much smaller than expected. PAM considered the initiation of a national MS patient registry without the central pre-utilisation judgment to be unfeasible. Medical Advisor Health Insurance Company According to a medical advisor from a health insurance company, the problem was attributable to a lack of clarity on exactly what kind of patients benefit from IFNβ. IFNβ is an expensive drug and the costs are either carried by the community (public health insurance) or reimbursed on an individual basis (private health insurance). Preferably, physicians should develop a guideline on the relative position of IFNβ in relation to alternative interventions. At the time of IFNβ’s introduction, a central pre-utilisation approval could have been relevant. However, time had passed and the prescription of IFNβ had become common practice. Unfortunately, new drugs are often introduced and included in the health care package long before all medical specialists agree on criteria for treatment. Professionals cannot develop guidelines quickly. In contrast, the time that passes between a drug being introduced into the market and its inclusion in the health care package is regulated by law and, in most cases, quite short. As a temporary solution, the drug could be included into the health care package under certain conditions, such as the registration of patient data. Neurologist A neurologist mentioned working on a guideline for diagnosing and treating MS patients as part of an initiative from the Dutch Society for Neurology. A guideline for diagnosis and treatment was considered relevant as it could support evidence-based practice. As in other medical fields, knowledge and treatment options have increased. The development of a guideline is a time-consuming endeavour as, often, it has to be done alongside the professional’s usual activities. A central pre-utilisation approval was no longer relevant. Prescribing IFNβ was considered common practice, also in small general hospitals. Most neurologists were already familiar with the indications for treatment. IFNβ was proven to be effective in one type of MS that is characterised by invalidating exacerbations (relapsing remitting MS) through clinical trials that demonstrated IFNβ’s ability to decrease the severity and frequency of these exacerbations. A national database could still be useful. However, its purpose must be clear. An appropriate goal could be to evaluate the long-term effectiveness of IFNβ. The neurologist questioned whether participants would have come to agreement on specific recommendations, such as which data should be collected and who should become the owner of the database. Most neurologists do have “their own kingdoms” and do not want to share these with others. Patient The patient claimed that agreement amongst neurologists about MS treatment could be useful. For him, the main problem was that MS care differs between hospitals. He conveyed his experiences with receiving contradictory answers to questions from numerous health professionals and the option of using IFNβ had not been discussed in the hospital where he was initially treated. For this patient, the exacerbations are highly invalidating. Obviously, his preference is to lead a normal life, in so far as that is possible. MS has a high impact on his life. Discussion Although results from the workshops showed that all respondents agreed that developing a guideline and a national database could be meaningful, these policy measures have never been implemented. A number of factors can provide an explanation for the proceedings in this project. 3 16 23 26 13 21 22 28 1 17 6 14 Thirdly, during the workshops, the proposed policy measures remained rather vague and were not elaborated in detail. As a result, participants could easily consent without violating their background theories and preferences. They had not felt the urge to (re)consider these theories and preferences. This, however, was necessary, as is clearly illustrated by the respondents who questioned whether participants would have come to agreement on specific recommendations, such as which data should be collected and who should become the owner of the registry. Such an agreement requires that policy measures are made sufficiently substantive for every actor to understand what are the consequences. Moreover, it requires that background theories and normative preferences are elicited and scrutinised. The interaction should cover the whole process from problem structuring to policy implementation, in order to be able to deal with problem shifts and changes in the socio-political context. It is not enough to reduce interactivity to workshops at one or two moments in time. Actors involved should resist the temptation to think that policy problems can be understood at a first order level. That is, background theories and normative preferences should be explicated in the problem definition phase. Only if problem structuring is taken seriously in this sense can one think of developing solutions that could meet with the approval of all target groups. 12 24 25 29 10 Proposed policy measures should be sufficiently elaborated, as to enable target groups to assess their consequences and constructively engage in the interactive process. As the case of IFNβ shows, actors involved are not willing to reconsider their background theories and preferences, should proposed policy measures not be sufficiently elaborated and actors involved not understand what is at stake. In sum, this study has shown that the organisation of workshops in which target populations participate does not qualify as an appropriate process of interactive policy development. From the beginning, emphasis had been put on a limited number of interventions aimed to control the expected increase in treatment costs. Although target populations participated in policy development, perceived problems and which interventions could provide a solution had been discussed insufficiently. For policy development to be successful, interactive methods are needed, in which problem definitions and assumptions are explicated and discussed, providing an opportunity for mutual learning between actors involved.