Health technology assessment agencies play pivotal role in patient access to medicines

Timely recommendation for drug reimbursement by health technology assessment (HTA) agencies is critical to ensuring patient access to the medicines they need, a study by the Centre for Innovation in Regulatory Science (CIRS) has confirmed.

As part of an ongoing effort to monitor regulatory and HTA performance, CIRS collected data on new active substances (NASs) appraised in 2014 and 2015 by eight HTA agencies in Canada, Australia and Europe, analyzing synchronization between the regulatory decision and the first HTA recommendation in terms of timing and outcome.

Overall, more than 60% of NASs approved by regulatory agencies received a positive or positive with restrictions recommendation by HTA agencies in most of the studied jurisdictions.

A second recent report from CIRS looks at the significant differences within healthcare systems in the appraisal and reimbursement of new medicines which produce multiple challenges for patient access to new medicines. For industry, the lack of harmonized HTA methodologies leads to inefficiencies in drug development and uncertainty in outcome, even for those drugs approved by regulatory authorities, CIRS reported.  The varying requirements of different systems may result in delays in submission and when assessment requirements or methods are unclear, the quality of submission may also be compromised, the report said.

CIRS has developed a collection of HTA “process maps” which track the varying requirements of different systems around the world.

Both of these new “R&D Briefings” from CIRS, titled “Review of HTA outcomes and guidelines in Australia, Canada and Europe, 2014-2015”and “HTA Process Maps: Identifying similarities and differences for alignment,” are available for download below.

In the first study, recommendations were collected from the HTA agencies for products approved between 2012-2015 by the respective jurisdictional regulatory agencies, the Australian Therapeutic Goods Administration (TGA), Health Canada and the European Medicines Association (EMA). The HTA entities included the Australian Pharmaceutical Benefits Advisory Committee (PBAC), the Canadian Agency for Drugs and Technologies in Health (CADTH), the British National Institute for Health and Care Excellence (NICE), the French Haute Autorité de Santé (HAS), the German Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWIG), the Polish Agencja Oceny Technologii Medycznych i Taryfikacji (AOTMiT), the Scottish Medicines Consortium (SMC) and the Swedish Tandvårds-& läkemedelsförmånsverket (TLV).

The HTA decisions in the report were classified as positive, positive with restrictions or negative.  In cases in which more than one HTA dossier was submitted by companies for the same drug based on different sub-indications within an approved regulatory label and the final HTA outcome for these individual sub-indications differed, the outcome was classified as multiple.

Among the findings:

  • Of all studied HTA agencies, Australia had the highest percentage of products recommended within a year from regulatory approval (96% in 2014 and 90% in 2015), followed by Scotland in 2014 (95%) and Germany in 2015 (89%).
  • Products were rolled out (regulatory submission to receiving a HTA recommendation) in Australia within the shortest median time (1.1 years in 2014 and 1 year in 2015), while Poland had the slowest roll-out time, with a median time of 2.5 years in 2014 and 2.2 years in 2015.
  • The 60% positive or positive with restrictions rate applied to recommendations by all of the HTA agencies except for those in Australia and Poland. At 89%, products appraised in Poland in 2015 received the highest percentage of negative HTA decisions.
  • In 2014 and 2015, France and Sweden had the highest proportion (91% and 88%) of positive/positive with restrictions recommendations for NASs appraised by HTA agencies.


Looking at data from the eight jurisdictions, CIRS identified four products that received a decision by all of the HTA agencies. Interestingly, these products were the new, but costly hepatitis C virus (HCV) therapies.

Background to HTA process mapping project

As part of its overall HTA program, CIRS initiated an HTA process mapping project. HTA process maps were created using systematic methodology to facilitate the comparison for a range of different HTA systems. The maps followed the movement of new pharmaceuticals from regulatory approval through HTA appraisal, reimbursement and pricing to adoption at the national level.

By 2017, process maps had been created for more than 75 jurisdictions. The maps were built into an online platform, the CIRS Regulatory and Reimbursement Atlas. The atlas serves as a visual communication tool to facilitate the explanation and comparison of different jurisdictions to multiple stakeholders.

These different systems present a challenge to attempts at harmonization or alignment of methods of assessment or appraisal, including the sharing of resources across jurisdictions, CIRS noted in its briefing on the maps. The differences also act as a barrier to the understanding and meaningful comparison of the systems and the identification of the best and most efficient practices. Ultimately, the differences can lead to a variety in outcomes in which a drug may be reimbursed in one country but not another. Such outcomes can undermine public confidence in healthcare systems, CIRS noted.

Review of HTA outcomes and timelines in Australia, Canada and Europe 2014-2015 (CIRS R&D Briefing 64)

HTA Process Maps: Identifying differences and similarities for alignment (CIRS R&D Briefing 63)

About CIRS: The Centre for Innovation in Regulatory Science is a neutral, independent UK-based subsidiary company, forming part of Clarivate Analytics. The mission of CIRS is to maintain a leadership role in identifying and applying scientific principles for the purpose of advancing regulatory and HTA policies and processes. CIRS provides an international forum for industry, regulators, HTA and other healthcare stakeholders to meet, debate and develop regulatory and reimbursement policy through the innovative application of regulatory science. It is governed and operated for the sole support of its members’ activities. The organization has its own dedicated management and advisory boards, and its funding is derived from membership dues, related activities and grants.