The challenges of regulatory pluralism.

Countries with small and/or less-resourced regulatory authorities that operate outside of a larger medical product regulatory system face a regulatory strategy dilemma. These countries may rely on foreign well-resourced regulators by recognising the regulatory decisions of large systems and following suit (regulatory reliance); alternatively, such countries may extend formal decision recognition to regulators in multiple other jurisdictions with similar oversight and public health goals, following a system which we call regulatory pluralism. In this policy comment, we discuss three potential limitations to regulatory pluralism: (i) regulatory escape, in which manufacturers exploit regulatory variation and choose the lowest regulatory threshold for their product; (ii) increased fragmentation and complexity for countries adopting this approach, which may, in turn, lead to inconsistent processes; and (iii) loss of international bargaining power in developing regulatory policies. We argue that regulatory pluralism has important long-term implications, which may not be readily apparent to policy makers opting for such an approach. We advocate for the long-term value of an alternative approach relying on greater collaboration between regulatory authorities, which may relieve administrative pressures on countries with small or less-resourced regulatory authorities, regardless of whether countries pursue a strategy of domestic regulation or regulatory pluralism.

Explaining why increases in generic use outpace decreases in brand name medicine use in multisource markets and the role of regulation.

BACKGROUND: Healthcare systems worldwide face escalating pharmaceutical expenditures despite interventions targeting pricing and generic substitution. Existing studies often overlook unwarranted volume increases in multisource markets due to differential physician perceptions of brand name and generics. OBJECTIVE: This study aims to explain the outpacing of generic medicine use over brand name use in multisource markets and assess the regulatory role, specifically examining the impact of reference pricing on volume and intensity increases. METHODS: Analyzing German multisource prescription medicine markets from 2011 to 2014, we evaluate regulatory mechanisms and explore whether brand name and generic medicines constitute separate market segments. Using an Oaxaca-Blinder decomposition approach, we divide the differential in brand name versus generic medicine use rates into market structure and unobserved segment effects. RESULTS: Generic use rates surpass same-market brand name substitution by 3.87 prescriptions per physician and medicine, on average. Reference pricing mitigated volume increase, treatment intensity and expenditure. Disparities in quantity and expenditure dynamics between brand name and generic segments are partially explained by market structure and segment effects. CONCLUSION: Generic medicine use effectively reduces expenditures but contributes to increased net prescription rates. Reference pricing may control medicine use, but divergent physician perceptions of brand name and generics, revealed by identified segment effects, call for nuanced policy interventions.

Insurance barriers and inequalities in health care access: evidence from dual practice.

BACKGROUND: We investigate access disparities in pharmaceutical care among German patients with type 2 diabetes, focusing on differences between public and private health insurance schemes. The primary objectives include investigating whether patients with private health insurance experience enhanced access to antidiabetic care and analyzing whether the treatment received by public and private patients is influenced by the practice composition, particularly the proportion of private patients. METHODS: We estimate fixed effect regression models, to isolate the effect of insurance schemes on treatment choices. We utilize data from a prescriber panel comprising 681 physicians collectively serving 68,362 patients undergoing antidiabetic treatments. RESULTS: The analysis reveals a significant effect of the patient's insurance status on antidiabetic care access. Patients covered by private insurance show a 10-percentage-point higher likelihood of receiving less complex treatments compared to those with public insurance. Furthermore, the composition of physicians' practices plays a crucial role in determining the likelihood of patients receiving less complex treatments. Notably, the most pronounced disparities in access are observed in practices mirroring the regional average composition. CONCLUSIONS: Our findings underscore strategic physician navigation across diverse health insurance schemes in ambulatory care settings, impacting patient access to innovative treatments.

Using the Online Elicitation of Personal Utility Functions Approach to Derive a Patient-Based 5-Level Version of EQ-5D Value Set: A Study in 122 Patients With Rheumatic Diseases From Germany.

OBJECTIVES: Traditional preference elicitation methods, such as discrete choice experiments or time trade-off, usually require large sample sizes. This can limit their applicability in patient populations, where recruiting enough participants can be challenging. The objective of this study was to test a new method, called the Online elicitation of Personal Utility Functions (OPUF) approach, to derive an EQ-5D-5L value set from a relatively small sample of patients with rheumatic diseases. METHODS: OPUF is a new type of online survey that implements compositional preference elicitation techniques. Central to the method are 3 valuation steps: (1) dimension weighting, (2) level rating, and (3) anchoring. An English demo version of the OPUF survey can be accessed at https://valorem.health/eq5d5l. From the responses, a personal EQ-5D-5L utility function can be constructed for each participant, and a group-level value set can be derived by aggregating model coefficients across participants. RESULTS: A total of 122 patients with rheumatic disease from Germany completed the OPUF survey. The survey was generally well received; most participants completed the survey in less than 20 minutes and were able to derive a full EQ-5D-5L value set. The precision of mean coefficients was high, despite the small sample size. CONCLUSIONS: Our findings demonstrate that OPUF can be used to derive an EQ-5D-5L value set from a relatively small sample of patients. Although the method is still under development, we think that it has the potential to be a valuable preference elicitation tool and to complement traditional methods in several areas.

Policy as a strategy to innovate in health care? A content analysis of innovation policies in Germany, 1994-2017.

Determining what innovation means for healthcare is becoming increasingly complex. Health policy addresses this challenge by designing initiatives to improve healthcare quality and efficiency, one example being the German Innovation Fund of 2015. We investigate the innovation concept underlying 25 years of German health policy to analyse which and why some innovations are sustainable in a healthcare system. Expanding a previous approach to identify changes in the semantic understanding of 'innovation', we identify the semantic understandings of innovation, variation in health innovation policy contingencies. We use Henry Mintzberg's approach to classify patterns in health innovation policy to uncover predominant planning, adaptive, and entrepreneurial strategy modes. Systematic analysis resulted in 44 decision-relevant policy documents. Content was classified based on a qualitative content structuring method according to seven main categories and 57 subcategories. Results reveal that the innovation concept is undergoing a transformation from a science-based concept, dominated by planning and adaptive modes, towards an exploration of process innovations, dominated by adaptive and entrepreneurial strategy modes. This change in strategy is an essential contingency of high-volume instruments, such as the Innovation Fund, and their capability to support the emergence of process innovations that lead to structural changes in the healthcare system.

Availability of New Medicines in the US and Germany From 2004 to 2018.

Importance: Germany's unique approach to coverage determination and pricing has ensured that effective medicines remain on the market, often at prices reduced through negotiation. However, less is known about trade-offs of this approach with regard to initial availability of medicines. Objective: To examine differences in the timing and scope of new medicines available in Germany and the US. Design, Setting, and Participants: This retrospective cohort study analyzed initial availability of new medicines approved by regulatory agencies in Germany and the US between January 1, 2004, and December 31, 2018, and followed up through December 31, 2019. Data analysis was conducted from January 1, 2020, to July 1, 2022. A total of 599 novel approvals were reviewed. Generic, biosimilar, vaccine, and combination medicines were excluded. Exposures: US Food and Drug Administration approvals were reviewed for therapies categorized as new molecular entities or new active ingredients. German approvals were reviewed from secondary administrative data of authorized medicines that determine availability in Germany, including data presented by the European Medicines Agency. Main Outcomes and Measures: Approvals were analyzed to determine the percentage of medicines approved and available in the US, Germany, or both countries and compare the times to reach the market. Results: Analysis of 599 new medicines demonstrated that fewer were available in Germany compared with the US (80% vs 92% of all potential therapies) and that the median difference in time to market was 4 months (95% CI, -44.40 to 44.76 months). Forty-nine medicines were approved in Germany but not in the US, 75% of which were rejected by the US Food and Drug Administration, were withdrawn, or had US equivalent agents. Conclusions and Relevance: In this cohort study, fewer new medicines were available in Germany compared with the US between 2004 and 2018. In addition, drugs entered the German market later than in the US.

The Effect of Biosimilar Prescription Targets for Erythropoiesis-Stimulating Agents on the Prescribing Behavior of Physicians in Germany.

OBJECTIVES: This study aimed to aid decision makers by analyzing the impact of introducing biosimilar prescription targets on physician prescribing behavior in the prescription of biologic erythropoiesis-stimulating agents in Germany. METHODS: We combined secondary data of regional level biosimilar prescription targets and secondary data of routinely collected claims data of dispensed prescriptions by physicians operating within the statutory health insurance system in ambulatory care across 7 German regions from 2009 to 2015. Two-way fixed-effects regression analysis was used to identify the average treatment effect of introducing biosimilar prescription targets at the physician level. The main outcome of interest was the share of biosimilar prescriptions on all prescriptions within the substance group. We compared 6 regions that introduced biosimilar prescription targets with 1 region without any prescription target policy. RESULTS: Introducing biosimilar prescription targets increased the average share of biosimilars between 6 percentage points (P < .05) in Hamburg and up to 20 percentage points (P < .001) in Saxony-Anhalt. Stratification of specialists by prescription volume and adoption status indicated heterogeneous effects. We identified similar but higher effects for high-volume prescribers. Disentangling of effects with regard to the composition of biosimilar share suggested that the increase in biosimilar share was driven by increased biosimilar use accompanied by a nonsignificant decrease in original biologics prescriptions. CONCLUSIONS: Prescription targets to alter physician prescribing behavior meet their intended goals by increasing biosimilar share. Physicians partly responded to the policy by decreasing overall prescriptions of the target substance. Prescription targets might be a useful tool, but decision makers need to consider all aspects of potential responses.

Do Medicine Shortages Reduce Access and Increase Pharmaceutical Expenditure? A Retrospective Analysis of Switzerland 2015-2020.

OBJECTIVES: We analyze how shortages led to changes in access to and expenditure for pharmaceutical care in the Swiss health system between 2015 and 2020. METHODS: We combined cross-sectional and longitudinal data to study medicine shortages by incidence, duration, intensity, and pharmaceutical expenditure. We assessed 4119 markets defined by active ingredient, dosage form, and strength. We classified markets by essential medicine status and other characteristics. We differentiated shortages by the degree to which alternative options are still available. We investigated the first lockdown period of the pandemic, considering also the shortage of COVID-19-specific medicines. RESULTS: A total of 1964 markets never reported shortages, and 1336 markets reported some shortages; 819 markets reported shortages lasting at least 14 days. Markets with a higher number of manufacturers, a lower co-payment share, and lower prices more frequently reported shortages. We did not find differences by essential medicine status. In 50% of instances, the average price of substitutes available was lower than the price of the product on shortage. The total pharmaceutical expenditure attributed to shortages increased by CHF 17.00 million (€15.63 million) in 2018. CONCLUSIONS: Medicine shortages have substantially reduced access to pharmaceuticals. Switzerland has experienced shortages on a scale similar to that in other countries. Prices of substitutes available at the time of shortages can be higher or lower, indicating an unelastic demand for medicines.

Are patients more adherent to newer drugs?

The annual preventable cost from non-adherence in the US health care system amounts to $100 billion. While the relationship between adherence and the health system, the condition, patient characteristics and socioeconomic factors are established, the role of the heterogeneous productivity of drug treatment remains ambiguous. In this study, we perform cross-sectional retrospective analyses to study whether patients who use newer drugs are more adherent to pharmacotherapy than patients using older drugs within the same therapeutic class, accounting for unobserved heterogeneity at the individual level (e.g. healthy adherer bias). We use US Marketscan commercial claims and encounters data for 2008-2013 on patients initiating therapy for five chronic conditions. Productivity is captured by a drug's earliest Food and Drug Administration (FDA) approval year ("drug vintage") and by FDA" therapeutic potential" designation. We control for situational factors as promotional activity, copayments and distribution channel. A 10-year increase in mean drug vintage is associated with a 2.5 percentage-point increase in adherence. FDA priority status, promotional activity and the share of mail-order prescription fills positively influenced adherence, while co-payments had a negative effect. Newer drugs not only may be more effective in terms of clinical benefits, on average. They provide means to ease drug therapy to increase adherence levels as one component of drug quality, a notion physicians and pharmacy benefit managers should be aware of.

The impact of drug quality ratings from health technology assessments on the adoption of new drugs by physicians in Germany.

Payers are increasingly calling for the value of new drugs to be measured explicitly. We analyze how the availability of drug quality ratings by health technology assessment (HTA) agencies affects the adoption of new drugs by physicians in Germany. We combine data from drug quality ratings, promotional spending, and a physician panel. In a latent utility model, time to adoption is specified as a function of quality rating, promotional spending by manufacturers, and physician-specific variables. As expected, drugs with a positive rating were adopted faster (p < 0.001) than those without. However, our results suggest that it was the publication of the quality rating itself that affected adoption. Indeed, before a quality rating was published, drugs that went on to receive a positive quality rating were not adopted significantly faster than drugs that went on to receive a negative quality rating. In contrast, after the publication of the HTA quality rating, drugs with a positive rating were adopted significantly faster than those without (p < 0.05). The per physician value of a positive quality rating was EUR 393.50. Our results suggest that there are returns from HTAs beyond their use in price negotiations.

Physician-Level Cost Control Measures and Regional Variation of Biosimilar Utilization in Germany.

Biologic drugs represent a large and growing portion of health expenditures. Increasing the use of biosimilars is a promising option for controlling spending growth in pharmaceutical care. Amid the considerable uncertainty concerning physicians' decision to prescribe biosimilars, explicit cost control measures may help increase biosimilar use. We analyze the role of regional cost control measures for biosimilars and their association with physician prescriptions in ambulatory care in Germany. We collect data on cost control measures implemented by German physician associations and national claims data on statutory health insurance covering 2009 to 2015. We perform panel regressions that include time and physician fixed effects to identify the average associations between cost control measures and biosimilar share/use while controlling for unobserved physician heterogeneity, patient structure, and socioeconomic factors. We identify 44 measures (priority prescribing, biosimilar quota) for erythropoiesis-stimulating substances, filgrastim, and somatropin. Estimates of cost control measures and their consequences for biosimilar share and use are heterogeneous by drug, measure type, and physician group. Across specialists, biosimilar quotas accounted for 5.13% to 9.75% of the total average biosimilar share of erythropoiesis-stimulating substances. Explicit quota regulations are more effective than priority prescribing. Regional variation in biosimilar use can be partly attributed to the presence of cost control measures.

Service quality and perceived customer value in community pharmacies.

A patient's perception of the service provided by a health care provider is essential for the successful delivery of health care. This study examines the value created by community pharmacies-defined as perceived customer value-in the prescription drug market through varying elements of service quality. We develop a path model that describes the relationship between service elements and perceived customer value. We then analyze the effect of perceived customer value on customer satisfaction and loyalty. We use data obtained from 289 standardized interviews on respondents' prescription fill in the last six months in Germany. The service elements personal interaction (path coefficient: 0.31), physical aspect (0.12), store policy (0.24), and availability (0.1) have a positive significant effect on perceived customer value. Consultation and reliability have no significant influence. We further find a strong positive interdependency between perceived customer value, customer satisfaction (0.75), and customer loyalty (0.71). Thus, pharmacies may enhance customer satisfaction and loyalty if they consider the customer perspective and focus on the relevant service elements. To enhance benefit, personal interaction appears to be most important to address appropriately.