We have every attribute needed to become one of the world’s leaders in the development of new drugs

By Joaquim Augusto Melo de Queiroz and Silvio Borges dos Santos Fachim

After almost ten years of discussion, the law establishing the legal framework for clinical research in Brazil was signed this Tuesday (5/28).

Clinical research consists of studies carried out with human participants to develop medicines, therapies and medical devices. For a molecule to be researched and eventually reach pharmacy shelves, a long, costly and complex path is required. According to the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA), up to 10,000 experimental compounds may be tested in the preclinical phase. Of these, only one innovative molecule will obtain approval from the FDA (the US health authority) — at an average cost of USD 2.6 billion and, most of the time, without any success.

In addition to these risks, in Brazil in particular, pharmaceutical innovation suffered from another problem: legal uncertainty, due to the lack of a legal framework on the subject. This scenario may change with the publication of the new law.

There were, it is true, scattered rules governing clinical research in the country. However, they favored a slow process, generating many doubts and controversies — which prevented more clinical research from being carried out in Brazil. There was uncertainty, for example, about limiting the liability of study researchers and sponsors. This drove away investment, talent retention and scientific innovation in the country. We were wasting valuable opportunities in this field.

In practical terms, the new legal framework speeds up the process for approving new research, with clear deadlines set by law. There is now a single body for the ethical review of research involving human beings. Its main function is to ensure the protection of the rights, safety and well-being of participants, before and during the research.

To this end, it will carry out the ethical analysis, review and approval of research protocols, as well as of the methods and materials to be used to obtain and document the consent of research participants. Finally, the National Research Ethics Body — to be regulated by an act of the Executive Branch — will serve as the appellate and oversight body for research activities in the country.

From the standpoint of predictability for investment in science in Brazil, one of the main issues that sparked extensive debate concerned the obligation to supply the experimental medicine after the study ends. There was uncertainty about the applicable rules, which drove away companies interested in conducting research, especially in cases involving experimental medicines for treating rare diseases, which are generally high-cost. The new law brought greater clarity to the cases in which this supply is required — and this may be one of the great strengths of the new legislation: providing greater legal certainty for both companies and patients.

However, the President vetoed the possibility of discontinuing the supply of the experimental medicine five years after it became commercially available. It is worth reflecting: if the medicine is already commercially available, the obligation to supply it could be waived in certain cases. This will inevitably cause Brazil to lose opportunities, contrary to countries that have already moved past this issue.

An interesting question to be assessed concerns the application of the new rules to ongoing clinical studies — specifically, the cases in which the obligation to supply medicines for rare diseases after the research ends may be waived. Indeed, doubts remain given the lack of a transition rule for studies already under way. It is an issue that deserves monitoring.

In general terms, for clinical research to be robust, it is essential that it can include great diversity among volunteers. This makes it possible to better identify the efficacy of the experimental medicine, possible adverse effects, appropriate dosage and other factors. A recent study by the Research-Based Pharmaceutical Industry Association (Interfarma) points to the advantages that signal Brazil’s potential to be among the world’s leading countries in clinical research. We have a highly heterogeneous population, with wide variation in climate, culture and socioeconomic conditions. Our health system is solid, tested and proven during the pandemic. We also have good health regulation. And we have a head start in terms of competitive costs for conducting clinical studies compared with other countries. We have the world’s seventh-largest population, the largest urban population in Latin America and the greatest biodiversity in the world. And we rank fifth in the world by number of pharmaceutical companies established here. These are extremely advantageous conditions for conducting scientific research. We therefore have every attribute needed to stand out in research on new medicines and become one of the world’s leaders in the development of new drugs. And the new law comes at a good time to boost this potential.

Originally published by Estadão (in Portuguese).