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Supplemental Process Can Aid in Assessing Value of Rare Disease Treatments


A study looking at the ways in which different countries handle appraisal and reimbursement processes for rare disease treatments (RDTs) revealed that implementing supplemental processes can aid in patient and physician decision making.

The implementation of supplemental processes is helpful to assess the true value of rare disease treatments (RDTs), according to a study published in the Orphanet Journal of Rare Disease.

RDTs can make it difficult to justify reimbursements as cost-effectiveness analyses often result in much higher willingness-to-pay (WTP) thresholds than common diseases and they are only applied to a small population of patients within each country. However, researchers noted that there has been an increase in the opinion that conventional appraisal approaches aren’t appropriate for assessing the value of RDTs.

“The processes enable flexibility with standard processes but seek to promote some consistency when handling RDTs within a country and are being adapted as a result of legislative direction or stakeholder feedback,” wrote the researchers.

Supplemental processes can help relieve some of the uncertainties and high price concerns that are associated with RDTs as they allow different standards to demonstrate the value of RDTs, support the interpretation of limited clinical evidence, and help overcome a lack of cost-effectiveness and aim to fast track the health technology assessment (HTA) process to guarantee more timely patient access to RDTs.

The study looked at HTA appraisal processes for RDTs in 30 European countries, Canada, and New Zealand and found that 41% (13/32) of the participating nations had supplemental processes for RDTs.

In addition, 31% (10/32) of the countries using standard processes utilized other special features that facilitate the appraisal of RDTs and 6% (2/32) of countries accounted for appraisal criteria that favors RDTs.

“The high number of countries with standard processes that are applying some of the features facilitating appraisal of RDTs or that are planning changes in their systems suggests that these countries are adopting similar approaches to supplemental processes, but in a less systematic and more ad hoc manner,” the authors wrote.

Knowing what supplemental processes for RDTs are being used in each country can allow countries to learn from each other about how to better deal with RDTs. Researchers said that this information could be helpful when implementing cross-border joint assessment collaborations.

For the countries with supplemental processes for RDT, 5 out of 13 (38%) have a higher level of integration with standard processes that are either expeditated processes (3/5), which allow for an earlier start for the assessment process, or processes where rarity is weighted (2/5), which use points systems to determine reimbursement status and WTP thresholds.

Many supplemental approaches are considered new and countries continue to update them as they gain more experience using them.

Supplemental approaches such as allowing for disease specific input to inform appraisals or accepting different WTP thresholds or granting exemption from submitting a cost-effectiveness models can relieve uncertainties in the interpretation of clinical evidence and can aid in overcoming the issue of lack of cost-effectiveness.

Researchers noted that although having supplemental processes does not mean that all issues are being dealt with, these processes do “allow for more consistent, adapted and flexible decision-making processes and improved accountability for reasonableness.”

“It is important that as more RDTs come to market, there is transparency in national appraisal processes not just to ensure fair and accountable decisions within countries, but to determine if a common appraisal framework could be developed for RDTs or ultra-RDTs across Europe,” the authors concluded.


Nicod E, Whittal A, Drummond M, Facey K. Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches. Orphanet J. Rare Dis. 2020;15(189):1-14. doi:10.1186/s13023-020-01462-0

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