Brazil Evaluates Multiple Technologies for PNH Treatment: An Example of an Integrated Approach to HTA
Published 01 September 2026
Last update 03 September 2026
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In Brazil, an interesting approach to health technology assessment (HTA) is being applied: the simultaneous assessment of multiple competing technologies within a single therapeutic area.
In August 2026, Brazil's national HTA agency CONITEC reviewed several drugs simultaneously for the treatment of paroxysmal nocturnal haemoglobinuria (PNH):
— ravulizumab
— crovalimab
— pegcetacoplan
— iptacopan
This is an important example of an assessment not of a single drug, but essentially of an entire emerging therapeutic area.
What Was Compared
The assessment considered technologies with different mechanisms of action on the complement system.
CONITEC compared:
— mechanisms of action of the drugs
— routes of administration
— clinical data
— specific patient populations
— pharmacoeconomic evaluation results
— budget impact on the healthcare system
Based on the review, CONITEC recommended the inclusion of iptacopan for adult patients with persistent anaemia after C5 inhibitor therapy.
Crovalimab and pegcetacoplan did not receive a positive recommendation. At the same time, it was recommended to exclude the previously included ravulizumab from the public system.
Horizon Scanning as Part of the Assessment
Particularly interesting is that the assessment was complemented by horizon scanning.
An additional 8 potential technologies for the treatment of PNH were identified, including drugs already registered in individual countries and technologies in Phase II–III clinical trials.
Thus, the decision is made not only based on the characteristics of a single new technology.
The regulator considers existing and new alternatives, determines their potential place in therapy, and simultaneously assesses which technologies may enter the market in the coming years.
Why This Matters for Rare Diseases
For rare diseases, this approach is particularly relevant.
Several high-cost drugs may emerge almost simultaneously for a single small patient population. Sequential assessment of each drug individually does not always allow for a full view of the therapeutic landscape, optimal positioning of the technology in therapy, or advance assessment of the cumulative budget impact of new technologies.
The Brazilian example shows how horizon scanning can be directly linked to HTA and used for forward-looking assessment of an entire therapeutic area, rather than just individual drugs.
Source:
CONITEC — Preliminary Report on Ravulizumab, Crovalimab, Pegcetacoplan, and Iptacopan for PNH
In August 2026, Brazil's national HTA agency CONITEC reviewed several drugs simultaneously for the treatment of paroxysmal nocturnal haemoglobinuria (PNH):
— ravulizumab
— crovalimab
— pegcetacoplan
— iptacopan
This is an important example of an assessment not of a single drug, but essentially of an entire emerging therapeutic area.
What Was ComparedThe assessment considered technologies with different mechanisms of action on the complement system.
CONITEC compared:
— mechanisms of action of the drugs
— routes of administration
— clinical data
— specific patient populations
— pharmacoeconomic evaluation results
— budget impact on the healthcare system
Based on the review, CONITEC recommended the inclusion of iptacopan for adult patients with persistent anaemia after C5 inhibitor therapy.
Crovalimab and pegcetacoplan did not receive a positive recommendation. At the same time, it was recommended to exclude the previously included ravulizumab from the public system.
Horizon Scanning as Part of the AssessmentParticularly interesting is that the assessment was complemented by horizon scanning.
An additional 8 potential technologies for the treatment of PNH were identified, including drugs already registered in individual countries and technologies in Phase II–III clinical trials.
Thus, the decision is made not only based on the characteristics of a single new technology.
The regulator considers existing and new alternatives, determines their potential place in therapy, and simultaneously assesses which technologies may enter the market in the coming years.
Why This Matters for Rare DiseasesFor rare diseases, this approach is particularly relevant.
Several high-cost drugs may emerge almost simultaneously for a single small patient population. Sequential assessment of each drug individually does not always allow for a full view of the therapeutic landscape, optimal positioning of the technology in therapy, or advance assessment of the cumulative budget impact of new technologies.
The Brazilian example shows how horizon scanning can be directly linked to HTA and used for forward-looking assessment of an entire therapeutic area, rather than just individual drugs.
Source:CONITEC — Preliminary Report on Ravulizumab, Crovalimab, Pegcetacoplan, and Iptacopan for PNH
