Brazil's health technology assessment body CONITEC approved unanimously a prioritisation list for its Clinical Protocols and Therapeutic Guidelines for rare disease treatments to be incorporated before the end of 2015.
IHS Life Sciences perspective | |
Significance | CONITEC has approved a list of rare disease treatments to be prioritised for its 2015 Clinical Protocols and Therapeutic Guidelines (CPTGs). |
Implications | It approved the final list that includes a total of 43 rare diseases, which are considered the most frequent and important rare diseases in Brazil. |
Outlook | CONITEC has decided to incorporate 12 CPTGs before the end of 2015. Pharmaceutical companies should expect clear requirements for clinical data that CONITEC would expect to see as a minimum in order to issue reimbursement recommendations for new orphan drugs. |
During the 31st meeting of Brazil's health technology assessment body CONITEC, it unanimously approved the prioritisation list for its Clinical Protocols and Therapeutic Guidelines (CPTGs) for rare disease treatments. CONITEC's decision can be accessed here in Portuguese.
Methodology
With more than 8,000 rare diseases to consider, and the need to prioritise groups of more frequent and important diseases to be included in the prioritisation list, CONITEC used three criteria:
- criteria inherent to the disease;
- criteria relating to clinical treatment; and
- political and strategic criteria.
Final list
After the public consultation, in which the pharmaceutical industry, Ministry of Health, and other public health institutions had strong participation, CONITEC approved the final list that includes a total of 43 rare diseases, which after applying the aforementioned criteria are considered the most frequent and important rare diseases in Brazil.
CONITEC has decided to elaborate a total of 12 CPTGs before the end of 2015, as detailed below.
CONITEC's final priority list of rare diseases | ||
Axis/Group | CPTG classification | Disease/disease group |
Axis I: Congenital anomalies or late manifestation | 1 | Pure autonomic failure (PAF) |
2 | Anomalies of determination and sex differentiation | |
3 | Primary immunodeficiency | |
4 | Chromosomal abnormalities and complex malformations (including: craniostenosis/dysostoses craniofacial; osteochondrodysplasias; Marfan syndrome and similar; DNA repair disease/chromosomal instability; Noonan syndrome and similar, and phakomatoses) | |
Axis I – Mental disability | 1 | Mental disability associated with syndromes and complex malformations Mental disability associated with chromosomal alteration Rett syndrome and related Fragile X syndrome X-linked mental deficiency Teratogenic mental disability Non-syndromic idiopathic mental disability Autosomal non-syndromic mental disability |
Axis I – Inborn errors of metabolism | 1 | Aminoacidopathies (including those affecting neurotransmitters in conjunction with hyperphenylalaninemia) |
2 | Inborn errors of metabolism with acute manifestation (including: intolerance to sugars defects of beta oxidation of fatty acids; disorders of the urea cycle; glycogenoses; organic aciduria) | |
3 | Linked adrenoleukodystrophy X chromosome disorders and peroxisome | |
4 | Metabolic disorder of metals and Porphyrias | |
Axis II – Infectious | 1 | Infection with atypical mycobacteria and BCGite |
Axis II - Inflammatory | 1 | Adult Still's disease |
Axis II - Autoimmune | 1 | Primary systemic vasculitis |
Axis I: composed of rare diseases of genetic origin | ||
Outlook and implications
Decree 199, which was published in January 2014 as a national policy, establishes the provision of integral healthcare for patients with rare diseases. The goal of this regulation is to develop and use technologies for health promotion and disease prevention, including the availability of treatments and nutritional formulas in the Unified Health System (SUS).
After facing strong criticisms regarding its weak policies for rare conditions, Brazil's Ministry of Health (MoH) has proposed a number of initiatives to improve care for patients with rare and genetic-based diseases (see Brazil: 27 March 2014: Brazilian MoH improves SUS services for patients with rare diseases).
CONITEC is the internal body within the MoH in charge of developing clinical protocols to facilitate the incorporation of innovative treatments in the SUS reimbursement list. Therefore, its final approval of the rare disease list and the decision to elaborate 12 CPTGs before the end of this year is a large advance in the improvement of public health funding for orphan drugs in Brazil.
Following CONITEC's publishing of the 12 CPTGs for rare diseases, pharmaceutical companies should expect clear requirements for clinical data that CONITEC would expect to see as a minimum in order to issue reimbursement recommendations for new orphan drugs. For the pharmaceutical industry, the new regulation and potential incorporation of new treatments would open a new niche in the already attractive Brazilian market (see Brazil: 18 November 2014: CONITEC to introduce rare-diseases clinical protocols in Brazil).