CMED opens Targeted Consultation on pricing of advanced therapy products

On February 07, 2025, the Drug Market Regulation Chamber (“CMED”) published Notice No. 1/25 to receive contributions on the pricing criteria for advanced therapy products (“ATMPs”) (“Targeted Consultation”).

Interested parties have until March 28, 2025 to submit their contributions through the portal (Portuguese only).

The initiative seeks to address the regulatory gap of Res. CMED No. 2, of March 5, 2004, which does not cover new ATMP technologies within the regulated categories established, resulting in the categorization of these products as omitted cases, thus requiring the establishment of criteria for the pricing of the ATMPs.

What are ATMPs?

ATMPs are an innovative class of biological medicines for the treatment of complex conditions, rare diseases, or those without available medical alternatives, such as multiple myeloma, acute lymphoblastic leukemia, hereditary retinal dystrophy, among others.

CAR-T cell therapies (chimeric antigen receptor) are treatments in which the patient’s T cells (a type of immune system cell) are modified in a laboratory to attack specific cells. T cells are taken from the patient’s blood, and the gene for a special receptor that binds to a specific protein on the target cells is added to the T cells in the laboratory. This special receptor is called a chimeric antigen receptor (CAR). A large number of CAR-T cells are then grown in the laboratory and administered to the patient by infusion.

Unlike cells and tissues used in transplants, the manufacturing process of ATMPs can include cell expansion and differentiation in culture, as well as genetic material modification, with the aim of modifying gene expression ­- to repair, add, delete, or edit genetic sequences, performing functions different from what they would have in the body originally.

The Brazilian Health Regulatory Agency (“Anvisa”) establishes three categories of ATMPs, namely the tissue engineering product, the cellular therapy product, and the gene therapy product, defined below according to RDC Anvisa No. 505/21, which provides for the marketing authorization of ATMPs:

XXI – tissue engineering product: biological product consisting of human cells organized in tissues or organs that presents properties that allow regenerating, reconstituting, or replacing a human tissue or organ, with or without structural support consisting of biological or biocompatible material, provided that (a) it has been extensively manipulated; and/or (b) it performs in the recipient a function different from that performed in the donor;

XXII – advanced cellular therapy product: biological product consisting of human cells or their non-chemically defined derivatives, which has the purpose of obtaining therapeutic, preventive, or diagnostic properties, through its main mode of action of metabolic, pharmacological, and/or immunological nature, for autologous or allogeneic use in humans, provided that (a) it has been extensively manipulated; and/or (b) it performs in the recipient a function different from that performed in the donor;

XXIII – gene therapy product: biological product whose active component contains or consists of recombinant nucleic acid, which may aim to regulate, repair, replace, add, or delete a genetic sequence and/or modify the expression of a gene, with a view to achieving therapeutic, preventive, or diagnostic results.

From a regulatory perspective, Anvisa established the regulatory framework for ATMPs through the following normative acts, which regulate topics such as product characterization, manufacturing, clinical trials, safety, quality, and efficacy requirements for marketing authorization, distribution, and use of these products:

• RDC No. 505/21: Provides for the marketing authorization of advanced therapy products and other provisions.
• RDC No. 506/21: Provides for the rules for conducting clinical trials with investigational advanced therapy products in Brazil, and other provisions.  
• IN No. 270/23: Provides for Good Manufacturing Practices Complementary to Advanced Therapy Products.  

ATMPs represent a great therapeutic promise, offering the population the possibility of access to unique, innovative, safe, and high-quality treatments, and considering their potential benefits, in some cases, Anvisa may grant the marketing authorization of the ATMP subject to the subsequent presentation of additional data and evidence proving clinical efficacy. This is called conditional marketing authorization, aligned with approval models of other international regulatory authorities, such as the European Medicines Agency (EMA – Europe), the Food and Drug Administration (FDA – United States), and which requires the signing of a Term of Commitment (TC) between Anvisa and the company responsible for the medicine in Brazil.

What is the impact of regulation on the industry?

CMED is an interministerial body responsible for the economic regulation of the medicines market in Brazil, with the aim of promoting competition and innovation in the pharmaceutical sector, created by Law No. 10.742/03, which defines regulatory standards for the pharmaceutical sector.

CMED’s role is to prevent abusive and monopolistic practices, in order to promote pharmaceutical assistance to the population. CMED is composed of representatives from Anvisa, as well as the Ministry of Health, the Office of the Chief of Staff of the Presidency, the Ministry of Justice, the Ministry of Finance, and the Ministry of Industry, Commerce, and Services.

The Targeted Consultation seeks to obtain information from representatives of the pharmaceutical industry, healthcare professionals, patients, patient associations, researchers, government entities, among other interested parties, and is an essential step for the development of future regulation on the pricing of ATMPs. The information collected will support the Regulatory Impact Analysis to be prepared prior to the drafting of the regulation by CMED.

From the industry’s perspective, the regulation of ATMPs by CMED is a complex and strategic issue. Each step, from the choice of the active ingredient to the multicenter clinical trials, demands substantial resources and time, which is reflected in the final cost of the product.

These products represent the cutting edge of modern medicine, whose research, manufacturing, and commercialization demands a highly specialized biotechnological infrastructure, high-cost investments, and development pipelines that can take years, even decades.

Finally, regulation by CMED will need to balance the economic and social interests among the involved agents, providing a sustainable environment with legal certainty so that the pharmaceutical industry continues to invest, produce, and make innovative and safe products available to the population.

 Demarest’s Life Sciences and Healthcare team is available to provide any additional clarifications that may be necessary.