Advocacy - ataxia.info

Advocacy

Advocacy is the set of strategic actions taken by patients, family members, associations, and healthcare professionals to defend rights, influence public policies, and expand access to treatments and medications. The word "advocate" comes from the English word "to advocate ," which means to defend a cause .

This page offers an introduction to advocacy for ataxia patients and their caregivers in Brazil.

ATS Training

If you want to learn about Advocacy, there's a great free course available!

Unidos Pela Vida (UPV) is a non-profit social organization with national operations whose mission is to defend people with cystic fibrosis and other rare diseases. The UPV coordinates a course called "Understanding Advocacy and Impacting Public Policies." The course is FREE (in Portuguese) and can be taken online on the "Conexão SUS" platform. It lasts 40 hours and provides a certificate to those who pass the test. You can register at https://conexaosus.org.br

Digital NMO Agents Training

Another excellent course for those who want to learn about Health Technology Assessment (HTA) and the role of ANVISA, CMED, CONITEC, patients, and associations in this crucial process for incorporating medications and other health technologies into the SUS is the "Digital NMO Agent Training," offered by ABNMO (Brazilian Association of Neuromyelitis Optica). The course is FREE, available online (in Portuguese), has 9 video lessons, and provides a certificate of participation.

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General information about "Advocacy" for obtaining ataxia medications in Brazil

In Brazil, advocacy is often associated with the actions of organized groups such as patient associations, federations of associations, and other entities that advocate on behalf of people with rare diseases.

Examples of advocacy actions include:

Demanding specific public policies for rare diseases.
Pressuring government agencies (such as SUS, ANVISA, CONITEC, and the Ministry of Health).
Participating in public hearings, technical meetings, and public consultations.
Producing data and reports.
Running campaigns and mobilizations (e.g., through social media) to raise awareness among stakeholders.

In the Legislative Branch, advocacy actions may involve proposing new laws, amending existing ones, or even rejecting draft bills considered inadequate. This can be done through mechanisms such as social media awareness campaigns, participation in public hearings, or direct engagement with lawmakers or parliamentary groups aligned with the cause. For instance, the inclusion of a new disease (such as a genetic ataxia or another rare condition) in the list of serious illnesses recognized by the Federal Revenue Service for income tax exemption requires a bill to amend Article 6, item XIV, of Law No. 7,713/1988, which defines the list of serious diseases. Such a bill must be approved by the National Congress and sanctioned by the President of the Republic. Introducing such a bill through a congressperson or senator, with the support of patient associations, specialists, and technical evidence, is an example of legislative advocacy.

In the Judiciary, advocacy can take the form of direct action (e.g., public civil lawsuits) or indirect support, such as providing documents and information that may aid decisions favorable to the cause. The courts can also be approached to report violations—for example, when existing laws are not being upheld.

In the Executive Branch, advocacy aims to influence public policies, administrative decisions, and budgetary priorities in favor of a particular cause or group. These actions can occur at the municipal, state, or national levels, using strategies tailored to the context and responsibilities of each jurisdiction. At the national level, the key actors include regulatory agencies (ANVISA, ANS), CONITEC, CMED, SCTIE, and others, as well as national councils (e.g., the National Health Council). A major example of executive-level advocacy at the national level is working with the Ministry of Health to ensure the incorporation of a new drug into the SUS, by submitting clinical evidence and mobilizing civil society. Another example would be public mobilization to defend an existing public policy that is under threat of being modified or discontinued.

In the specific case of ataxias, advocacy actions may aim to:

1. Expand access to orphan drugs

Monitor and support the drug registration process at ANVISA. In particular, verify whether the pharmaceutical company has requested priority review (RDC 204/2017), since it involves a rare disease.
Participate in CONITEC public consultations that determine whether a drug will be incorporated into SUS.

2. Raise awareness among authorities

Engage with lawmakers, health departments, and regulatory agencies.
Propose bills and bring the issue to media attention.
Publish content showing the urgency and benefits of treatment.
Collect patient testimonies illustrating the impact of lack of treatment.
Advocate for the inclusion of ataxias in the Federal Revenue Service's official list of serious diseases (for tax exemption purposes).

3. Support research and innovation

Encourage the development of research and clinical trials for ataxia treatments in Brazil.
Prepare dossiers with clinical evidence demonstrating the safety and efficacy of drugs or therapies for ataxia.
Provide testimonials and data for natural history studies and other research efforts.

4. Empower patients

Educate patients and families about their rights.
Fight stigma and ableism.

For example, Abahe (Brazilian Association of Hereditary and Acquired Ataxias) has been tirelessly advocating for the interests of people with ataxia:

When requested, it provided aggregated data on the number of people with Friedreich’s ataxia in Brazil, broken down by region, gender, age group, and other criteria (data from Abahe’s patient registry).
It followed the submission process of the drug Skyclarys for registration with ANVISA by the pharmaceutical company Biogen.
It collected patient testimonies to illustrate the urgency of approval.
It sent official letters to ANVISA requesting updates on the Skyclarys registration process, even using the Access to Information Law.
It organized several social media campaigns to engage public opinion.
It participated in public hearings in the National Congress on rare diseases.

The mobilization paid off!
Skyclarys was approved by ANVISA for commercialization in Brazil on April 11, 2025.

Abahe remains mobilized and is now monitoring the drug pricing process at CMED, followed by the CONITEC review for incorporation into the SUS.

How can a new drug for rare diseases reach the SUS?

The arrival of a new drug for ataxia or other rare diseases to the SUS depends on success in three important stages , which we will briefly explain below:

(1) ANVISA's authorization to market the drug in Brazil,

(2) the definition of prices in CMED and

(3) a favorable opinion from CONITEC for incorporation of the drug into the SUS, and the final decision by the Ministry of Health.

If step 1 is unsuccessful (if ANVISA does not grant registration), the drug cannot be marketed in Brazil (the pharmaceutical company can file an administrative appeal). If step 1 is successful but step 3 is not (i.e., if CONITEC denies incorporation into the SUS), the drug will be commercially available in Brazil, but its cost will likely be very high , making it inaccessible to the vast majority of patients. In this context, many lawsuits are filed to force health plans to cover the costs, for example.

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Step 1 - ANVISA (Approval of the sale of the drug in Brazil)

The process of registering, evaluating and regularizing products as new medicines at ANVISA (National Health Surveillance Agency) follows a series of rigorous technical and regulatory steps.

Let's look at a summary of the main steps.

1. Research and Development (pre-ANVISA phase)
Before any submission to ANVISA, the drug is developed and tested in laboratories and in clinical studies (Phase I, II, and III) to evaluate its safety, efficacy, and quality. Many drugs already undergo ANVISA review after having been previously approved by other international regulatory agencies, such as the FDA (United States) or the EMA (Europe).

2. Submission of the Marketing Authorization Application
The pharmaceutical company submits the complete drug dossier to ANVISA. This dossier must include technical information on:

Pharmaceutical quality (manufacturing, quality control, stability)
Preclinical studies (animal testing)
Clinical trials (human testing)
Pharmacovigilance information
Labeling and package insert

3. Technical Evaluation
ANVISA conducts a comprehensive evaluation across several areas:

a) Quality
Verifies whether the product is manufactured in compliance with Good Manufacturing Practices (GMP), with proper control of purity, dosage, stability, etc.

b) Safety and Efficacy
Assesses clinical trial data to ensure that the medication is safe for human use and effective for the proposed indication.

c) Pharmacovigilance
Requires a post-marketing monitoring plan, especially for new drugs or those under conditional approval.

Review Timelines

Innovative drugs: the legal timeframe is up to 365 days.
Priority drugs (e.g., for rare diseases, emergencies): may undergo fast-track review, reducing the timeframe. According to WHO criteria, ANVISA defines a rare disease as one affecting up to 65 individuals per 100,000 inhabitants (see Note 3 below).

4. Marketing Authorization Decision
If approved, the drug receives a marketing authorization, which allows it to be sold in Brazil. The registration number is published in the Brazilian Federal Gazette (Diário Oficial da União). If denied, the company may file an administrative appeal.

5. Post-Marketing and Monitoring
After authorization, the product remains under continuous monitoring by ANVISA. Obligations may include:

Reporting of adverse events
Periodic re-evaluations
Renewal of registration every 10 years

Notes

Generic and similar drugs follow more simplified processes than innovative drugs.
Biological and biosimilar drugs have their own specific regulations.
Prioritization: Drugs for rare diseases (also known as orphan drugs) follow a differentiated process at ANVISA aimed at speeding access to treatments that the industry would not typically develop due to low profitability and low disease prevalence. ANVISA has specific regulatory mechanisms to prioritize these drugs. The average review time may drop from 365 days to as few as 120 days—or even less in some cases.
- This prioritization is formalized through a prioritization request, which must be justified and approved by ANVISA.
- See RDC No. 204/2017: Establishes criteria for prioritizing petition reviews, including for drugs for rare diseases.
Conditional marketing authorization: ANVISA may also grant conditional approval even if clinical trials are still in intermediate phases (e.g., Phase II), provided there is evidence of relevant clinical benefit, unmet medical need, and a commitment from the company to complete pending studies.
- This type of approval allows for early marketing of the product, with post-marketing obligations, such as periodic pharmacovigilance reports and completion of additional efficacy and safety studies (Phase IV).
- See RDC No. 205/2017: Regulates conditional approval for drugs intended to treat serious or rare diseases.
Data flexibility – In certain cases, ANVISA may accept more limited clinical data or foreign data (e.g., from the FDA or EMA), especially when:
- The disease is ultra-rare
- There is no alternative treatment available
- The data are robust and consistent, even if from smaller studies

ANVISA is also a member of international groups such as ICH and can participate in collaborative reviews with other regulatory agencies (e.g., FDA, EMA, Health Canada), which can accelerate approval of orphan drugs already registered abroad.

Step 2 - CMED (Definition of maximum consumer prices by the pharmaceutical company)

There is an intermediary process between ANVISA and CONITEC, which involves CMED (Chamber for the Regulation of the Medicines Market).

CMED is an interministerial body linked to ANVISA responsible for regulating and authorizing drug prices in Brazil, both for the private market and for the SUS (Unified Health System).

Immediately after ANVISA grants registration for a new drug (for example, Skyclarys for Friedreich's ataxia), the pharmaceutical company (in this example, Biogen) cannot yet sell the product in Brazil. It must first have a price approved by CMED.

The pharmaceutical company must submit a pricing proposal for the medication it wishes to sell in Brazil to CMED, including:

The proposed price and justification (based on international price comparisons, cost-effectiveness studies, etc.)
Economic and technical data
Comparison with similar treatments (if any)
Justifications based on innovation, lack of competition, etc.

With this information, CMED applies a methodology based on the criteria defined in CMED Resolution No. 2/2004, which considers:

Therapeutic category
Degree of innovation
Existing comparators in the market
Price in reference countries (Australia, Canada, Spain, USA, France, Greece, Italy, New Zealand, and Portugal)
Price practiced in countries where the drug has already been approved

CMED then:

Sets the PMC (Maximum Consumer Price): the price ceiling for sales in the private market
Establishes the government purchase price: used as a baseline for public procurement (e.g., for SUS)

These prices are published in the Official Gazette of the Union and on the CMED website. After that, the drug can be marketed in Brazil.

This pricing process must be completed BEFORE submitting a request for incorporation into the SUS, because CONITEC needs to assess the budget impact. It is essential to determine whether the medication is cost-effective. In other words, the price will directly influence CONITEC's decision on whether or not to incorporate the drug into the public system.

Important for advocacy!

The price negotiation starts with the company, but pricing control is regulated by the State.
If the approved price is too high, it may compromise CONITEC’s decision to incorporate the drug into SUS due to lack of cost-effectiveness. (High prices are one of the main barriers to incorporation.)
That’s why monitoring this process and advocating for fair pricing is a strategic part of advocacy. Transparency and social mobilization can help pressure for more equitable prices.
Following this pricing phase is crucial to preparing advocacy actions for the CONITEC stage.

Once the pricing is resolved, the next step is at CONITEC.

Stage 3 - CONITEC (Evaluation of requests for incorporation of the drug into the SUS)

Medicines for rare diseases—often called orphan drugs—undergo a rigorous evaluation process before being made available by the Unified Health System (SUS). This evaluation is conducted by CONITEC ( National Commission for the Incorporation of Technologies into the Unified Health System - SUS). CONITEC evaluates health technologies based on scientific evidence , taking into account efficacy, effectiveness, safety , and economic aspects , to support its recommendations.

CONITEC was created by Law No. 12,401/2011 to advise the Ministry of Health on the incorporation, exclusion, or modification of medications, products, and procedures within the SUS (Brazilian Unified Health System). In other words, CONITEC is the agency responsible for evaluating new health technologies and recommending whether or not they should be adopted by the SUS . This process encompasses everything from medications and equipment to medical procedures.

Legal Framework for CONITEC's operations:

Law 12.401/2021
Decree 7.646/2011

Furthermore, CONITEC is responsible for developing or updating Clinical Protocols and Therapeutic Guidelines (PCDT) , which define diagnostic and treatment criteria, including incorporating newly approved medications. In short, no medication (including those for rare diseases) enters the SUS without undergoing CONITEC's careful analysis, which seeks to ensure both clinical benefit to patients and viability for the public health system.

CONITEC is a collegiate body comprised of representatives from various government agencies and healthcare professionals (such as ministries, ANVISA, councils of health secretaries, the Federal Council of Medicine, among others). Recently (2025), its membership also expanded to include a civil society representative linked to the pathology under evaluation, with voting rights, as per Law No. 15.120/2025. This change reflects the importance of bringing the voices of patients and society into the decision-making process.

Regarding the governance structure, CONITEC is made up of an Executive Secretariat and specialized Committees.

Figure 1 - Diagram of CONITEC's structure

Incorporation Process

For detailed information on the incorporation process, visit the CONITEC website.

Below is a summary of some key points:

Before any incorporation, the medication must be registered with ANVISA (National Health Surveillance Agency) and, in the case of drugs, have a price established by CMED (Drug Market Regulation Chamber). The incorporation of procedures still considered experimental is prohibited.
Anyone or any institution can request that CONITEC evaluate the incorporation of a new technology into the SUS—this includes manufacturers, medical societies, patient associations, health authorities (such as state and municipal health departments), or even ordinary citizens. In practice, many requests for the evaluation of orphan drugs are submitted by pharmaceutical companies with the support of rare disease patient organizations, which gather documentation and advocate for the analysis of new treatments.
The request must be submitted to CONITEC’s Executive Secretariat (Department of Health Technology Assessment and Management – DGITS) along with all documentation required by law (outlined in detail further ahead). The DGITS team conducts an initial screening to verify whether the dossier is complete and compliant with legal requirements. If any documentation is missing, the request may be returned to the applicant for completion.
Under current legislation, the incorporation of new technologies into SUS (medications, therapies, equipment, etc.) must be evidence-based (with assessments of efficacy and safety) and supported by economic evaluation studies (cost-effectiveness analysis).
Once the documentation is deemed appropriate, the technical evaluation begins. CONITEC’s technical team analyzes the submitted data, reviewing in detail the studies on efficacy, safety, as well as cost-effectiveness and budget impact assessments provided by the requester. If needed, CONITEC may request additional information or studies.
A technical report is then produced containing a critical analysis of the data and, if necessary, a reanalysis with assumptions adjusted to the SUS context. This report forms the basis for deliberations, leading to a preliminary recommendation either in favor of or against incorporating the medicine into SUS. This recommendation is based on the evidence reviewed and considers comparisons with treatments already offered by SUS for the disease in question (if available).
Before the recommendation is finalized, it is submitted to public consultation. The report and CONITEC’s opinion are made available for public input for 20 days. In advocacy, this stage of social participation is essential. During this period, anyone can submit comments via an electronic form. Contributions may include patient testimonies about the use of the medication (real-life experience), or input from medical societies and researchers presenting additional data. In the case of rare diseases, public consultations often involve significant mobilization—patients share stories about the disease and the hope surrounding the medication, NGOs submit position papers, and professionals clarify questions about disease management.
In exceptional cases, the public consultation period may be extended or shortened (e.g., extended to 30 days or shortened to 10 days), but the general rule is 20 days.
Once the public consultation concludes, CONITEC compiles and analyzes all contributions. Patient experiences and public opinion may enrich the evidence and even introduce new data on quality of life, access barriers, or other practical aspects. Based on this, the committee reassesses its initial stance. The preliminary recommendation may be maintained or revised depending on the arguments raised during public consultation. Then, CONITEC issues its final recommendation on the technology (whether for incorporation or not), consolidated into a final report.
CONITEC's final recommendation is not the definitive decision, as the final word belongs to the Ministry of Health, according to legislation. The report is forwarded to the Secretary of SECTICS (Secretariat of Science, Technology, Innovation, and the Health Economic-Industrial Complex) of the Ministry of Health.
In practice, the SECTICS Secretary is responsible for making the final decision on whether to incorporate new technologies (such as medicines, procedures, or equipment) into SUS, after receiving CONITEC’s technical recommendation. Generally, the Secretary follows CONITEC’s recommendation but may also consider other management-related factors. In high-profile cases, additional public hearings or extended meetings may be called before the final decision, allowing for broader debate and public participation (although this is not mandatory). If public hearings are convened by the Ministry of Health, patient representatives, specialists, and health managers may engage in open dialogue.
Once the decision is made, it is published in the Federal Official Gazette (DOU) through an official ministerial ordinance. If the decision is to incorporate the medication, the ordinance formalizes the inclusion of the new therapy into SUS. After that, the Ministry of Health and SUS management bodies begin the necessary steps to offer the treatment in practice (including defining treatment protocols, financing, procurement, and distribution).
The legal timeframe for the evaluation process is 180 days, extendable by another 90 days. In other words, the maximum legal time for CONITEC to conclude a process is 270 days (about 9 months). This is established in Decree No. 7,646/2011, to prevent requests from remaining unanswered indefinitely. In practice, most evaluations are completed within the initial 180 days, and extensions are exceptions. The law also states that, after CONITEC’s recommendation, the decision on incorporation must be formalized in a timely manner. Although the law does not establish a long deadline for the Secretary's decision, in practice, the final decision is usually made quickly after CONITEC’s final meeting, and within a few weeks, the ministerial ordinance is published in the DOU.
When a drug is incorporated, CONITEC may also prepare or update the PCDT (Clinical Protocol and Therapeutic Guidelines) for the rare disease in question, including the new drug as a recommended treatment option within SUS. These protocols ensure that clear usage criteria are in place (e.g., recommended doses), specifying which patients and under what conditions the new therapy will be offered.
When the final decision is in favor of incorporation, the Ministry of Health has up to 180 days from the date of the ordinance to ensure that the new technology is available through SUS. This post-incorporation deadline is provided for in Decree 7,646/2011 and Law 12,401/2011. That is, within a maximum of 6 months, the Ministry—together with states and municipalities—must resolve issues such as drug procurement, distribution, staff training, classification under the appropriate Pharmaceutical Assistance Component (basic, strategic, or specialized), and other necessary actions so that patients can begin receiving treatment.
If the final decision is not to incorporate the orphan drug, it means SUS will not offer it for that indication. The rejection ordinance is also published in the DOU, along with the reasons (usually based on CONITEC’s technical report). In this case, the process ends without incorporation. The applicant and society may, in the future, submit a new evaluation request if additional evidence emerges or there are significant changes (e.g., new studies showing greater efficacy or a price reduction that improves cost-effectiveness). It’s worth noting that a negative decision does not prevent patients from accessing the drug through other means (such as private health plans or legal action), but SUS will not officially offer it until a future revision is made.

This process is standardized for any health technology, including drugs for rare diseases. In the case of orphan drugs, patient associations are often highly engaged during public consultations due to the lack of alternative treatments and urgent need for care. However, the evaluation criteria and procedural steps remain the same, ensuring that decisions are based on evidence and the public interest.

Figure 2 – CONITEC Technology Incorporation Flowchart

Formal Requirements for Submitting an Application (Decree No. 7,646/2011)
For CONITEC to review an incorporation proposal, the request must meet the documentary requirements set forth in Decree No. 7,646/2011 (which regulates the process of incorporating technologies into the SUS). Article 15 of this decree establishes that, when filing a request with CONITEC's Executive Secretariat, the interested party must present:

Completed submission form: A specific form, available on the CONITEC website, must be fully completed with information about the proposed technology. This standard form provides guidance on the required data (drug indication, target audience, etc.).
ANVISA Registration: The ANVISA health registration number and its expiration date are mandatory. Without valid ANVISA registration, the technology cannot be evaluated for use in the SUS (Unified Health System).
Evidence of efficacy and safety: Scientific evidence (clinical study results, for example) must be attached demonstrating that the new technology is at least as effective and safe as the options already available in the SUS for that disease. In other words, it must be proven that the orphan drug offers a relevant clinical benefit and is not inferior to existing treatments (if any).
Economic evaluation (cost-effectiveness) study: The presentation of an economic evaluation comparing the new technology with technologies already incorporated into the SUS for the same indication is required. This is usually a cost-effectiveness or cost-utility study, showing the cost per additional health outcome compared to current alternatives. This study must be conducted from the perspective of the SUS (considering costs and benefits within the Brazilian public system).
Product samples (if applicable): When applicable, samples of the product or inputs must be provided for evaluation. This requirement typically applies to technologies such as healthcare equipment or products, in cases where CONITEC deems it necessary to test or materially evaluate the proposed item. For medications, this sample requirement is not typically applied, but it is provided for in the standard for applicable situations.
Defined price: In the case of medications, the product's price, as approved by the CMED (maximum selling price), must be provided. Price is important for cost-effectiveness and budgetary impact analysis, so the proponent must present the official price at which they intend to offer the medication to the SUS.

These formal requirements are intended to ensure that the application already contains the minimum information necessary for a robust evaluation. Only applications that meet these legal requirements are accepted for review; otherwise, the proposal is returned to the applicant without consideration of its merits.

Additional comments

To submit an incorporation request to Conitec, it is necessary to use the System for the Electronic Management of Technology Incorporation Processes in the SUS (e-GITS).
Different types of studies can be used in the incorporation analysis process (Observational Study, Cohort Study, Clinical Trial, Cost-Effectiveness Analysis, etc.).
All documents must be submitted through the Ministry of Health's Digital Protocol service, which allows general documents to be submitted electronically without the need for in-person visits to the Protocol department or postal mail. The system only accepts files in .pdf, .html, .xls, and .xlsx formats up to 50MB in size.
Economic and budget impact studies must be available in .xlsm (Excel Macro-Enabled Workbook file) or .trex (TreeAge) formats.

Assessment Criteria Used by CONITEC

When evaluating the incorporation of a medicine (including orphan drugs) into the SUS, CONITEC uses a set of technical-scientific and socioeconomic criteria to support its recommendation.

The main criteria are:

Clinical efficacy and effectiveness: Efficacy refers to results under ideal research conditions (i.e., controlled clinical trials), while effectiveness refers to how well a treatment performs in real-world clinical settings. CONITEC reviews the available clinical studies to assess how much the medicine improves health outcomes (e.g., symptom reduction, increased survival, improved quality of life) in patients with the rare disease, compared to those who receive no treatment or an alternative. It is essential to demonstrate that the new medicine provides significant clinical benefit over existing SUS options or, if no alternatives exist, that it meets an unmet therapeutic need.
Safety: CONITEC evaluates the adverse effects and risks associated with the medicine. Orphan drugs often have limited safety data due to smaller populations studied, so the commission carefully reviews toxicity data, serious adverse events, long-term risks, etc. A medicine will only be recommended if its benefits clearly outweigh its risks. If there are signs of serious adverse events with no corresponding efficacy, safety concerns may prevent incorporation.
Quality of scientific evidence: While not a separate criterion per se, it is a cross-cutting consideration. CONITEC takes into account the methodological robustness of the studies. High-quality evidence (such as randomized clinical trials and systematic reviews) carries more weight than weak observational studies. For rare diseases, the evidence base may be more limited, but the commission still assesses confidence levels (e.g., confidence intervals, sample sizes, biases) to judge the consistency of the findings.
Cost-effectiveness: CONITEC analyzes submitted health economic evaluations, especially the incremental cost-effectiveness ratio of the new drug versus alternatives. In simple terms, this assesses how much it costs the SUS to achieve an additional unit of health benefit with the orphan drug (e.g., cost per life-year gained or per crisis avoided) compared to the current standard of care. Since 2023, CONITEC has adopted explicit cost-effectiveness thresholds to assist in this analysis, to determine whether the cost per outcome is within an acceptable range. A drug that is too expensive for the benefit it offers may receive a negative recommendation unless it is the only option for a severe condition (a context that raises ethical debates).
Budget impact: In addition to cost-effectiveness (a relative efficiency measure), CONITEC evaluates the absolute budget impact of incorporating the drug. That is, what will be the total cost to the SUS to adopt the treatment for the eligible population in Brazil? Even an effective medicine may have a significant financial impact if the eligible population is large and the unit cost is high. For rare diseases, the number of patients is often small, but the per-patient cost is extremely high. CONITEC estimates how many millions of reais per year the government would need to spend and whether this fits within the national health budget.
Availability of alternatives and medical need: CONITEC considers the clinical context. If there are no existing therapeutic alternatives in the SUS for a given rare disease, a new drug—even with limited evidence—may be extremely necessary. On the other hand, if there is already an available treatment with similar efficacy, the new medicine must show clear advantages (e.g., greater effectiveness or fewer adverse events) to justify incorporation. Therapeutic advantage is also evaluated: does the drug represent a significant innovation? Does it serve a subgroup of patients without alternatives? Does it offer greater convenience, such as oral instead of injectable administration? These factors influence the recommendation.
Logistical and organizational impact: CONITEC also examines how incorporating the medicine would affect the health system’s logistics and operations. This includes: Does SUS have the infrastructure to use the technology? (e.g., for a gene therapy, are there centers able to administer it?). Is training of professionals or adaptation of services needed? Does the drug require cold chain logistics, special monitoring, or expensive follow-up tests? These aspects are considered, as a technology that is unfeasible to implement may not be recommended—even if effective—until the system is prepared to support it.
Ethical, social, and legal considerations: Finally, CONITEC also takes into account social and ethical implications. For example, incorporating a very expensive drug for a very small number of patients can raise ethical questions about resource allocation (although rarity alone is not a reason to deny care, equity is always weighed). Legal issues, such as ongoing lawsuits or established patient rights, may also be considered. Public pressure and patient advocacy are addressed through public consultation, but CONITEC aims to balance these voices with technical evidence. As stated by the Ministry of Health, the commission’s analysis includes possible social, legal, and ethical impacts to align the decision with public health needs and SUS values (equity, universality).

In summary, CONITEC's decision is multifactorial. For a rare disease medicine to receive a positive recommendation, it must generally demonstrate relevant clinical benefit, acceptable safety, economic feasibility (reasonable cost-benefit ratio and budget compatibility), and practical feasibility for implementation.

References: The information above is based on current legislation (Law 12.401/2011, Decree 7.646/2011) and official sources from the Ministry of Health and CONITEC.
Text generated with the support of Artificial Intelligence and reviewed by the author.