Oncology drugs for orphan indications: how are HTA processes evolving for this specific drug category?

Abstract

Orphan drugs (ODs) are intended for the diagnosis, prevention, or treatment of rare diseases. Many cancer subtypes, including all childhood cancers, are defined as rare diseases, and over one-third of ODs are now intended to treat oncology indications. However, market access for oncology ODs is becoming increasingly challenging; ODs are prone to significant uncertainty around their cost-effectiveness, while payers must balance the need for these vital innovations with growing sensitivity to rising costs. The objective of this review was to evaluate different mechanisms that have been introduced to facilitate patient access to oncology ODs in five different countries (Australia, Canada, England, France, and Sweden), using eight oncology ODs and non-orphan oncology drugs as examples of their application. A targeted literature review of health technology assessment (HTA) agency websites was undertaken to identify country-specific guidance and HTA documentation for recently evaluated oncology ODs and non-orphan oncology drugs. None of these countries were found to have explicit HTA criteria for the assessment of ODs, and therefore, oncology ODs are assessed through the usual HTA process. However, distinct and additional processes are adopted to facilitate access to oncology ODs. Review of eight case-study drugs showed that these additional assessment processes were rarely used, and decisions were largely driven by proving cost-effectiveness using standard incremental cost-effectiveness ratio (ICER) thresholds. The predominant implication arising from this study is that application of standard HTA criteria to oncology ODs in many countries fails to take into account any uncertainties around their clinical- and cost-effectiveness, resulting in disparities in HTA reimbursement decisions based on differences in addressing or accepting uncertainty. In order to address this issue, HTA agencies should adopt a more flexible approach to cost-effectiveness, as typified by the Tandvårds-och Läkemedelsförmånsverket in Sweden, which takes into account the small patient numbers involved, limited budget impact, and high unmet medical needs.

Cost-effectiveness analysis of isavuconazole versus voriconazole for the treatment of patients with possible invasive aspergillosis in Sweden

“…Data for between 2005 and 2011 for TLV decisions on the inclusion of drugs in the pharmaceutical benefits scheme show that the lowest cost per QALY declined was 700,000 SEK while the highest accepted was 1,220,000 SEK [35]. This compares favourably also to oncology drugs that have been assessed positively by the Swedish council for novel therapeutics with ICERs per QALY up to 1,080,000 SEK (with pertuzumab being the exemption, having an ICER at 2,600,000 SEK) [36].…”

Can the Consideration of Societal Costs Change the Recommendation of Economic Evaluations in the Field of Rare Diseases? An Empirical Analysis

“…Although the rarity of the disease is one of the variables that are normally mentioned in the literature to consider higher than usual thresholds, Adkins et al did not identify country-specific guidance and health technology assessment (HTA) documentation for recently evaluated ODs. 80 None of these countries were found to have explicit HTA criteria for the assessment of ODs; therefore, ODs are assessed through the usual HTA process. As far as we know, only the National Institute for Health and Care Excellence seems to consider a higher threshold linked to the NICE's Highly Specialised Technologies Programme.…”

Reimbursement of Orphan Drugs in Europe in Relation to the Type of Authorization by the European Medicines Agency and the Decision Making Based on Health Technology Assessment

“…Adkins et al ( 2017 ) made a review to evaluate different mechanisms that have been introduced to facilitate patient access to oncologic orphan drugs in 5 different countries (Australia, Canada, England, France, and Sweden), using 8 oncologic orphan drugs and non-orphan oncologic drugs as examples of their application. It was revealed that additional assessment processes were rarely used and decisions were mostly driven by proving cost-effectiveness using standard incremental cost-effectiveness ratio thresholds.…”