A decade of early benefit assessment of ophthalmic drugs in Germany: success story or not?

ABSTRACT

Objective

To analyze how ophthalmic drugs fared in the early benefit assessment (EBA) after its introduction in Germany up to 2020 and to quantify its impact on their negotiated prices.

Methods

Relevant documents were screened and essential content on added benefit outcomes and the underlying evidence was extracted next to pricing information. In addition to descriptive statistics, cross-stakeholder analyses and agreement statistics were implemented.

Results

Thirteen completed EBA were identified involving eight drugs. Only four drugs (30.8%) received an added benefit. The OR for no added benefit of ophthalmic drugs versus all other drugs was 2.971 (0.902–9.781). The agreement between manufacturers’ claims and decision-maker appraisals is fair (kappa 0.435). In all cases, evidence was derived for RCTs, but for different reasons, not all of them allowed direct comparisons with the comparator as defined by the decision-maker. The negotiated rebates on manufacturer’s selling prices varied from 6.8% up to 47.4%. Nevertheless, the rebates for ophthalmic drugs (median 14.5%) were lower than those for all negotiated drugs (median 24%).

Conclusion

Over the past decade, the EBA of ophthalmic drugs was not necessarily a success story, but in most of the cases, the drugs were successful in the market.

KEYWORDS:

• Early benefit assessment
• added benefit
• ophthalmic drugs
• Federal Joint Committee
• IQWIG
• appraisal
• price negotiations
• budget impact
• evidence-based medicine

Article highlights

• Early benefit assessment of new drugs based on relative effectiveness analysis was introduced in Germany in 2011 for cost-containment reasons. Regarding ophthalmic drugs, only a few successfully underwent the assessment. The majority failed mainly due to formal reasons.

• Counterintuitively, ophthalmic drugs lacking added benefit showed smaller rebates compared to drugs in other therapeutic areas and were also quite successful in market shares.

• Ophthalmologists who prescribe these drugs differ with the results of this assessment as to the drugs’ value. However, the newly introduced and currently implemented physicians’ digital information system might impact their prescribing behavior by offering concise data on benefit assessment through medical office software.

• With the market entrance of biosimilars for established ophthalmic biologicals in the near future, a stronger cost-containment effort is expected as these biologicals are within the 10 highest-revenue prescription drugs.

Declaration of interest

In addition to his academic affiliation, the author is employed by Bayer Vital GmbH in Leverkusen, Germany. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewers Disclosure

Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.

Author contributions

Charalabos-Markos Dintsios is the sole author and responsible for the manuscript regarding all

the elements of the contribution.

Data availability statement

Except for some price data, the data that support the findings of this study are publicly available in the stated information sources of the methods parts.

Additional information

Funding

This paper was not funded.