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Treatment Preferences in Germany Differ Among Apheresis Patients with Severe Hypercholesterolemia

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Abstract

Background

Severe hypercholesterolemia is a major risk factor of death in patients with coronary heart disease. New adjunctive drug therapies (proprotein convertase subtilisin/kexin type 9 [PCSK9] inhibitors) have gained approval in Europe and the USA.

Objective

In this empirical study, we documented preferences regarding adjuvant drug therapy in apheresis-treated patients with severe familial hypercholesterolemia.

Methods

We conducted a systematic literature search to identify patient-relevant outcomes in patients with severe hypercholesterolemia currently undergoing apheresis. Data were used to generate a semi-structured qualitative interview that enabled seven patient-relevant characteristics with three levels each to be identified. For the discrete choice experiment, an experimental design (7 × 3) was generated using NGene Software that consisted of 96 choices divided into eight blocks. The survey was conducted between November 2015 and April 2016 using computer-assisted personal interviews.

Results

The survey was completed by 348 patients (64.9% male). The random parameter logit estimation showed predominance for the attribute ‘reduction of LDL-C (low-density lipoprotein cholesterol) level’. ‘Risk of myopathy’ and ‘frequency of apheresis’ dominated next. Within the random parameter logit estimation, all coefficients were significant (P ≤ 0.01). The latent class analysis identified three patient groups. The first group (126 patients) found ‘reduction of LDL-C level in blood’ to be most important. This group focused solely on this treatment outcome independently of apheresis frequency or additional injections. The second group (106 patients) focused on three attributes: ‘frequency of apheresis’, ‘risk of myopathy’, and ‘reduction of LDL-C level in blood’. Respondents clearly considered a high frequency of apheresis to have a negative impact. The third group (116 patients) demonstrated the highest preference for apheresis. These patients have adjusted to apheresis for > 10 years.

Conclusion

Regarding patient preference, clinical efficacy seems to dominate. Hence, ‘reduction of LDC-C in blood’ was ranked highest above patient-relevant modes of administration and adverse effects. In the patient groups identified, reduction of apheresis was important for only a subsegment (30%) of patients. Another 30% wanted effective LDL-C reduction by whatever means necessary. Most strikingly, another 30% preferred higher frequencies of apheresis.

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Acknowledgements

The authors thank Spreeconsult GmbH for conducting the survey interviews. Susanne Bethge made technical contributions to the organization of the study and the early study design and assisted in the pretesting of the survey. Finally, the authors would like to thank all survey participants.

Funding

This study was financed by Sanofi Deutschland GmbH, Berlin, Germany.

Author information

Authors and Affiliations

  1. Professur Gesundheitsökonomie und Medizinmanagement, Hochschule Neubrandenburg, Brodaer Straße 2, 17033, Neubrandenburg, Germany

    Axel C. Mühlbacher, Andrew Sadler & Christin Juhnke

  2. Gesellschaft für empirische Beratung GmbH (GEB), Denzlingen, Germany

    Axel C. Mühlbacher, Andrew Sadler & Christin Juhnke

  3. Sanofi Deutschland GmbH, Evidenzbasierte Medizin/HEOR, Berlin, Germany

    Franz-Werner Dippel

Authors
  1. Axel C. Mühlbacher
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  2. Andrew Sadler
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  4. Christin Juhnke
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Contributions

Axel Mühlbacher designed and directed this study as the principal investigator. Axel Mühlbacher and Andrew Sadler conceived and planned the experimental design of the study. Andrew Sadler performed analysis on all samples. Axel Mühlbacher, Andrew Sadler, and Christin Juhnke contributed to the interpretation of the results. Christin Juhnke helped with the qualitative interviews and took the lead in preparing the manuscript. Franz-Werner Dippel provided critical feedback and commented on the manuscript. All authors finally approved the latest version to be published.

Corresponding author

Correspondence to Axel C. Mühlbacher.

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Conflict of interest

Axel Mühlbacher, Andrew Sadler, and Christin Juhnke declare that they have no further conflicts of interest. Franz-Werner Dippel was an employee of Sanofi Deutschland GmbH during the time of the conduction of this study.

Data availability

The datasets on the respondents’ choices generated and/or analyzed during the current study are available from the corresponding author on reasonable request.

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Mühlbacher, A.C., Sadler, A., Dippel, FW. et al. Treatment Preferences in Germany Differ Among Apheresis Patients with Severe Hypercholesterolemia. PharmacoEconomics 36, 477–493 (2018). https://doi.org/10.1007/s40273-018-0614-9

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  • DOI: https://doi.org/10.1007/s40273-018-0614-9

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