Addressing the lack of evidence
Regulatory agencies and health technology assessment (HTA) bodies require robust evidence to show that a drug treatment is both effective and safe. In the case of rare diseases, it may be difficult for researchers to generate sufficient evidence, especially on how effective the treatment is compared to other available treatments. The lack of evidence may hinder market access and the subsequent reimbursement of treatments for these conditions. This may slow down or even block patient access to novel treatments. The HARMONY Alliance aims to help researchers to develop appropriate mitigation strategies for new treatments for Hematological Malignancies (HMs).
Additional considerations
Katy Harrison and colleagues from NICE studied the additional considerations (other than clinical considerations and cost effectiveness) that HTA agencies may include in their decisions. Examples of such additional considerations are unmet need, rarity of disease, issues with current treatment, patient impact, national priority, tolerance, indirect benefit, and end of life. The researchers explored the influence of these additional considerations on the assessment decision across eight HTA agencies. They concluded that inclusion of these considerations in HTA assessment was associated with positive reimbursement recommendations. This indicates that, given the challenges in producing robust evidence for these rare HMs, scientific and social value judgments are an important part of the decision processes for these drugs.
Evidence gap
In the second study, HARMONY Partner NICE analyzed evidence base differences across regulator and HTA agencies. For 12 HM innovative products, the researchers extracted clinical evidence data sources from assessment reports from the EMA and eight national HTA agencies. They studied the overlap in the HTA evidence used for the assessment by these different agencies. The results indicate a closer evidence alignment than was expected. This suggests caution with regard to narrowing the evidence gap further: if fewer additional evidence sources and earlier phase studies are used to meet HTA requirements, this may lead to negative decisions.
Katy Harrison, Scientific Advisor National Institute for Health and Care Excellence (NICE), UK and HARMONY Task Lead: ‘’It is great to be presenting our work at ISPOR - this is the leading European conference for health economics and outcomes research and attracts attendance from professionals in market access from HTA bodies and pharmaceutical companies to academics working on new methods for health economics. The work presented will hopefully highlight the need to include a wide range of factors beyond clinical and cost effectiveness for HMs and for robust clinical data within their submissions for reimbursement as most HTA agencies use this information in their decision making."
The virtual oral presentation at ISPOR2021 is planned at 30 November 2021 (11am-3pm CET) - All session recordings of ISPOR2021 will be available for conference participants until 2 January, 2022.
Both abstracts will be published in the ISPOR journal 'Value in Health', Volume 24, Issue 12, S2, to be released in December 2021.
Virtual ISPOR Europe 2021, the leading European conference for HEOR
In recent years, rare diseases, digital therapeutics, personalized medicine, cell and gene therapies, and other new therapeutic approaches have stretched our data and methodological capabilities. Furthermore, issues associated with these special populations and technologies have important implications for a range of healthcare decisions, from regulatory pathways to coverage, reimbursement, and shared clinical decision making. ISPOR Europe 2021 will feature scientists and stakeholders who work on these key challenges and highlight innovative solutions, as well as examples of how partnerships and dialogue with other disciplines are contributing to address these issues.
The HARMONY Alliance is a European Public–Private Partnership for Big Data in Hematology that is capturing and mining Big Data on various Hematologic Malignancies. The HARMONY Alliance, funded by Innovative Medicines Initiative, unites more than 100 organizations such as European medical associations, hospitals, research institutes, patient organizations, pharmaceutical and IT companies. HARMONY uses Big Data analytics to accelerate the development of more effective treatments for blood cancer patients. The aim is to accelerate the development of more effective treatments. Data are stored in HARMONY’s Big Data Platform, which has already identified over 80,000 anonymized patient records, making it one of the largest databases of its kind. Leading research teams are currently using this wealth of information to answer critical questions about hematologic malignancies that cannot be addressed with other methods. In addition, HARMONY is running projects to develop core outcome sets, as well as Health Technology Assessment projects and multi-stakeholder activities. The HARMONY Alliance is currently running two projects: HARMONY (November 2016-June 2023) and HARMONY PLUS (October 2020-October 2023). Both have work packages dedicated to HTA and multistakeholder engagement.
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Public-private partnership for Big Data in Hematology
HARMONY and HARMONY PLUS are funded through the Innovative Medicines Initiative (IMI), Europe's largest public-private initiative aiming to speed up the development of better and safer medicines for patients. Funding is received from the IMI 2 Joint Undertaking and is listed under grant agreement for HARMONY No. 116026 and grant agreement for HARMONY PLUS No. 945406. This Joint Undertaking receives support from the European Union’s Horizon 2020 Research and Innovation Programme and the European Federation of Pharmaceutical Industries and Associations (EFPIA).
HARMONY Coordination Office
Institute of Biomedical Research of
Salamanca (IBSAL)
Salamanca, Spain
HARMONY Communications
European Hematology Association (EHA)
The Hague, The Netherlands
