Survey among pharmaceutical companies: Implementation of health services research in Germany

Health services research – or, in international parlance, with comparable meaning, „Real World Evidence (RWE) studies“, „Health Services Research“ or „Health Economics Outcomes Research (HEOR)“ – are scientific disciplines that address „real patient care“ – have been predicted to gain increasing importance for years, particularly in the area of pharmaceutical products.1 A similar trend can be observed in Germany,4 as evidenced by statements from politicians and self-governing bodies, discussions on the current German Statutory Health Insurance Strengthening Act (GKV-Versorgungsstärkungsgesetz), and published positions from stakeholders in the medical profession or the pharmaceutical and medical technology industries.2,3.

The pharmaceutical industry plays a significant role in the development of health services research, both nationally and internationally. To the authors' knowledge, precise data on research expenditures in the field of health services research do not exist. However, considering the proportion of companies' expenditures on health services research relative to their expenditures on clinical research, an estimated 5 million euros are currently allocated to health services research studies. This can vary from company to company, depending on the importance placed on the topic. It is known, however, that the pharmaceutical industry bears the majority of total research expenditures in the healthcare sector. Looking at the percentage shares of various areas in total research and development expenditures, a 2013 US survey found that 47 million euros are allocated to clinical trials (excluding Phase IV). The remaining shares are allocated to preclinical studies, regulatory affairs, pharmacovigilance, and other areas.7 National and international research are difficult to distinguish. The Federal Health Reporting System indicates approximately €3.9 billion in research expenditure for the health sector in 2013. In the same year, the pharmaceutical industry in Germany invested €6.1 billion in R&D. It can therefore be assumed that the pharmaceutical industry is also increasingly becoming a significant player in the field of health services research – despite the extensive health services research activities of health insurance funds and the anticipated substantial financial resources in connection with the expected establishment of the Innovation Fund at the Federal Joint Committee (G-BA).
However, it is unknown how intensively and methodologically uniformly the instruments of health services research are currently embedded or implemented in pharmaceutical companies. The aim of this article is therefore to illustrate, based on the results of a survey of member companies of the German Association of the Pharmaceutical Industry (BPI), how health services research studies are currently assessed as a research area in pharmaceutical companies in Germany, how frequently they have been implemented to date or are planned for the future, and what challenges the pharmaceutical industry faces in implementing such studies.
Survey of pharmaceutical companies
The BPI is a trade association representing 240 companies with approximately 70,000 employees in the fields of pharmaceutical research, development, approval, manufacturing, and marketing. These companies include traditional pharmaceutical companies, pharmaceutical service providers, and companies in the biotechnology, herbal medicine, and homeopathy/anthroposophic sectors.
The company survey was planned and implemented by the BPI's Health Services Research working group, addressing the following topics:
• Definition/understanding of healthcare research in companies
• Previous implementation of health services research studies: study types and frequency of implementation
• Challenges in the implementation of health services research studies
• Planned implementation of healthcare research studies in the future.

The online questionnaire was sent to the management and healthcare research experts of the companies. A total of 184 full members were contacted. 55 companies participated in the survey. Multiple responses were possible for many questions, and participation was not mandatory.
Survey results
Internal company definition of healthcare research
The surveyed companies – or rather, the respondents – have quite different understandings of health services research. While retrospective studies of health insurance/billing data (67.3%), observational, non-interventional studies (58.2%) and post-marketing surveillance studies (49.1%) are very frequently considered health services research, patient record studies (34.5%), patient surveys (25.5%), physician surveys (23.6%) and pragmatic, randomized trials (23.6%) are only interpreted as part of health services research in a quarter to third of cases. The distinction between health services research and market research within pharmaceutical companies is also not uniform: 791 of the respondents see study protocols as a criterion for differentiation in health services research studies, 631 see resource and time expenditure in general, 611 see the ability to publish and 451 see the representativeness of the results as a criterion for differentiation.

Previous, current implementation of health services research studies
On average, the surveyed companies conducted 2.7 (median: 1.0) health services research studies in the past three years. The most frequently conducted health services research types by the surveyed companies to date (at least one study in
(over the past 3 years) were observational non-interventional studies (60%)
as well as post-marketing surveillance studies (64%); retrospective cash register/billing data studies (38%), pragmatic trials (26%), patient surveys (20%) or patient record studies (12%) were conducted far less frequently (Figure 1). Currently, companies are conducting an average of 1.5 studies, of which 45.6% are in the context of products before market access, 38.5% in the context of drugs after their market access and 15.9% in other contexts.
To differentiate the surveyed companies, three company types were distinguished: companies with high healthcare research intensity (>3 different types)
with a high intensity of health services research studies in the past three years (13 companies), with medium intensity of health services research (2-3 different types of health services research studies in the past 3 years (20 companies)) and with low intensity of health services research (maximum one type of health services research study in the past 3 years (17 companies)).
Planning of health services research studies and challenges
Pharmaceutical companies plan health services research studies for three reasons: as an information base in the AMNOG benefit assessment process (69%), as an information base for marketing and product positioning (75%), and as a basis for subsequent discussions with health insurance funds (69%). 42% of the companies are currently planning health services research studies. The main reasons for not currently planning studies are high financial costs (64%), excessive time commitment (41%), and a lack of added value from the results (27%). The surveyed companies primarily plan to conduct retrospective health insurance data/billing studies (69%), observational non-interventional studies (50%), and patient record studies (38%) in the future. A comparison with Figure 1 shows that cash register/billing data studies (+31 percentage points), patient record studies (+26 percentage points) and pragmatic trials (+5 percentage points) will gain in importance, while prospective non-interventional studies (-10 percentage points) and observational studies (-8 percentage points) will slightly lose in importance.
Figure 2 summarizes the data on the current implementation and planning of health services research. It shows that approximately half of the surveyed companies with a high to medium level of previously conducted health services research studies are planning new ones. However, among companies with a low level of previously conducted health services research, only two companies (18.2% of the responding companies) are currently planning to conduct a health services research study. The most frequently cited reasons for not planning studies are excessive costs, followed by excessive time expenditure.

Internal processes in the planning and approval of health services research studies
The survey aimed to describe the internal planning and approval processes of pharmaceutical companies when establishing and conducting health services research studies. The survey data reveal that health services research studies are planned in a far less formalized manner than, for example, clinical trials (Fig. 3). Approximately 54 companies with a high level of health services research activity to date have standardized approval processes (national or international), and approximately 38 companies have similarly standardized planning processes/templates for health services research studies (national or international). For companies with a medium level of health services research activity to date, these rates are 25 (standardized approval processes) and 20 (standardized planning processes); as expected, the standardization of these processes is less established in companies with lower levels of health services research activity.
The vast majority of responding companies (35 out of 36) indicated that there is no automated start to the planning of health services research studies when a product under development has reached a specific stage of development.
According to the respondents, significant barriers to the implementation of health services research studies include a lack of know-how within their own organization, a lack of acceptance of the data within the benefit assessment process and the clinical community, as well as a lack of cooperation partners (Fig. 4).
Interpretation of the survey results and forecast of future developments
The survey results show that health services research is a significant topic for pharmaceutical companies. These studies involve considerable effort, both financial and in terms of time. In particular, there is uncertainty regarding the acceptance of the data, which is why ultimately only 42% of the responding companies plan to initiate health services research studies in the future. Based on the survey results, retrospective analyses of health insurance/billing data, patient record studies, and prospective pragmatic trials will become increasingly important for companies in the coming years.
The survey results reveal surprising findings, firstly, the differing definitions of health services research across the surveyed companies, and secondly, the limited formalization of planning and approval processes for health services research projects. The responses thus indicate significant potential for further development in establishing health services research within pharmaceutical companies. Uncertainty regarding the value of health services research data in alternative applications (exploratory and comparative health services research), as well as considerable uncertainty concerning the effort involved in study planning and execution, including data availability, evidently leads to reluctance in many companies to implement health services research studies. Furthermore, the low degree of formalization in the planning of health services research studies and the lack of involvement of higher-level internal departments demonstrate that health services research data is currently often perceived as having a more localized, rather than a systematic, benefit.
The authors hypothesize that health services research studies will gain in importance, accompanied by more formalized and thus more transparent planning and approval processes within pharmaceutical companies.6 This development will be partly initiated by the companies themselves, but will also be shaped externally by the authors' prediction of greater acceptance of health services research data due to the following macro trends: (1) implementation of the German Statutory Health Insurance Strengthening Act (GKV-Versorgungsstärkungsgesetz), (2) increasing reimbursement of pharmaceutical products based on their "real-life benefit" and possibly even on the basis of "real-life health economic models," and (3) implementation of innovative/new forms and structures of care that also include pharmaceutical products.
The survey results presented show that this development has already begun in pharmaceutical companies, but there is still a clear need for formal, procedural, and substantive development before health services research in the implementation of pharmaceutical companies has the status it (apparently) already has in official statements by representatives of politics/self-governing bodies.