Battles for access to in-depth information on the benefits and harms of drugs usually target regulators' transparency policies, or else seek to encourage pharmaceutical companies' to voluntarily open their data vaults.
However, there is a third way to unlock key drug documents and make them available to the wider scientific community - and Germany's Health Technology Assessment agency IQWIG has shown that it works.
Numerous studies have consistently and conclusively shown that Clinical Study Reports (CSRs) contain vital information on drug safety and efficacy not found elsewhere in the public realm. (See here and here for examples.) Without access to CSRs, independent scientists' ability to evaluate whether a drug is safe or effective is very limited.
Pharmaceutical companies have to provide regulators with CSRs when they apply for a marketing license for a new drug, but regulators have traditionally locked these documents away and refused to share them with third parties.
Recent momentum towards greater transparency has been strong, but the battle is far from over:
The European Medicines Agency has made large and significant steps towards providing access to CSRs, but there are still some barriers to access, and the agency has recently backtracked on some of its transparency pledges.
A very timid FDA pilot has so far resulted in only one CSR being made available.
A court case in Canada this summer has raised hopes that CSRs will routinely be disclosed there in future, but the court's verdict may still be appealed.
While the ongoing tug-of-war between industry and patient advocates over access to CSRs via regulators' archives has consumed most energy and attention, Germany's IQWIG has long been dragging data contained within CSRs into the light through a simple yet highly effective mechanism:
Any data used in IQWIG's assessments is later made publicly available.
Hence, IQWIG publishes all data contained within CSRs that it used during a drug assessment in its assessment report. This disclosure policy is enshrined in national law. IQWIG's assessments inform price negotiations between German insurance funds and industry, the development of therapeutic guidelines, and treatment decisions by patients and their doctors, giving it considerable clout.
The German model adds value above and beyond the transparency measures contained in the European Medicines Agency's transparency policy, a 2015 article written by IQWIG staff notes:
"[E]ven after successful implementation of the new EMA policy  stipulating the publication of full clinical study reports, AMNOG documents will still provide additional relevant information.
Firstly, a systematic review of a new drug versus the appropriate comparator treatment, focusing on the approved subpopulation—AMNOG documents may thus contain subgroup analyses not available in the clinical study reports.
Secondly, under the EMA policy, only reports submitted to EMA will be published, many of which refer to placebo controlled studies. Especially AMNOG documents on reassessed drugs (see box 1) may contain information from additional clinical study reports of active-controlled studies conducted after drug approval.
AMNOG thus potentially enables access to information from a larger pool of clinical study reports than are available through EMA, with a higher proportion of reports of active-controlled studies."
The article further notes that:
"Our analysis shows that HTA [health technology assessment] can provide substantial information on new drugs as early as at the time of market entry. Although the primary goal of AMNOG was to inform price negotiations, the assessment process has made detailed information on a new drug’s effects available to the public, as a comprehensive reporting format and mandatory publication of thisinformation isrequired by law.
Although HTA processes in other countries also aim to include all relevant information, to the best of our knowledge no other HTA agency is supported by legislation to publish the extent of information included in AMNOG documents.
Our results emphasise that HTA should not be based on conventional, publicly available sources or on any selected presentation of data by pharmaceutical companies but should include comprehensive study information, including clinical study reports, wherever possible."
In 2017, the AllTrials campaign commended IQWIG's positive example, and recommended that other health technology assessment bodies:
"After initiation of the assessment process, post the Clinical Study Reports and all other data for trials cited in licensing application documentation online, with minimal redactions.
Post all Clinical Study Reports reviewed in past assessments online, with only minimal redactions."
As the European Union discusses greater harmonization and coordination between its many individual national HTAs, Germany provides a tried-and-tested model of how the rules of the game can be re-written to benefit patients, taxpayers and the scientific community.
Correction: An earlier version of this blog drew incorrect parallels between the roles of Germany's IQWIG and Britain's NICE. Their roles differ, and the text has been amended accordingly (26 Sept). TranspariMED would like to thank Dr Beate Wieseler of IQWIG for the correction.
Note: This blog was written based on research conducted by TranspariMED as part of a grant awarded by HealthWatch UK, an independent British charity. HealthWatch UK did not review this blog prior to publication. The content of this blog is the sole responsibility of TranspariMED, and should not be taken to reflect the position of HealthWatch UK.