Veto of part of the enacted law distorts the constitutional mandate that assigns to the State the duty to promote health

With the new legal framework for clinical research in Brazil about to be signed into law, researchers and investors dedicated to finding cures for millions of patients suffering from devastating diseases had high hopes. The new law was expected to bring legal certainty, speed and predictability to attract new investment in clinical studies in Brazil.

The law was signed by the President earlier this month (Law 14,874/24). It brought significant progress, it is true, and its publication should be celebrated. However, one of the President’s vetoes frustrated both researchers and patients, because it will cause the country to waste precious opportunities for new clinical research, particularly for medicines to treat rare and ultra-rare diseases. Disclaimer: controversial views will be presented in this article.

After almost a decade of intense and fruitful debate in Brazilian society, the Legislative Branch had consolidated, in early May, the text of Bill 6007/2023. Among its most important provisions was the establishment of clear rules on the dynamics for unlocking the approval of new research for the development of therapies and medicines.

It clarified, for example, the cases in which the study sponsor was obliged to guarantee free access to the new therapy for research participants, and the exceptional circumstances that would allow it to be discontinued. These included a 5-year period counted from the commercial availability of the innovative medicine in Brazil. The rule seemed reasonable. It had been refined after years of intense debate, with the participation of civil society and a long legislative process in the Senate and the Chamber of Deputies.

The reasonableness of this rule stems from a holistic understanding of the research process for a new therapy. To this end, it is essential to understand the entire arc, which begins with the first laboratory research, a necessary step to identify potential compounds and molecules.

Once promising compounds are identified, in vitro assays and animal tests are conducted, following research ethics guidelines, to assess the safety and efficacy parameters of the compound being researched. Only then does the clinical stage begin, consisting of three initial phases.

Phase I, aimed at a small group of healthy volunteers, is mainly intended to examine safety issues. Phase II aims to establish the efficacy of the therapy, with the participation of people with the disease. Phase III seeks to analyze the treatment’s risk-benefit, through large randomized, multicenter, placebo-controlled studies with hundreds or thousands of patients.

Once all these stages have been completed, on rare occasions, the medicine will prove safe and effective for use by patients. Phase IV, post-marketing, begins after the therapy is approved by the regulatory agency, in order to monitor unexpected effects and adverse reactions.

As can be seen, discovering a new therapy involves a long and tortuous path. This process is fraught with multiple uncertainties and requires significant investment before the invention can eventually translate into a safe, effective, high-quality treatment.

Billions of reais are often spent, frequently with no return, over decades. And there is no guarantee that the time and resources spent will be converted into an innovative treatment that benefits patients. It is a risky bet. Most of the time, fruitless and loss-making.

This context highlights the need for balance. On the one hand, guaranteeing access to the therapy for patients who agreed to take part in the research. Without them, there would be no invention. On the other, ensuring the economic incentive for research to be carried out in Brazil — especially in a scenario in which Brazil occupies an insignificant position in the ranking of countries that conduct the most clinical research.

There was logic, therefore, in the provision that free supply to research participants could cease 5 years after the medicine became commercially available. Indeed, if the therapy is available on the market, the patient should not be left without assistance — above all because of the constitutional right to health (art. 196 of the Federal Constitution), which requires the State to guarantee citizens access to proper treatment.

Despite this, the President decided to veto the rule setting this period. The choice was to impose on researchers and sponsors a perpetual civil obligation: to supply the researched medicine to clinical study participants free of charge and for life, even if it is already available on the market.

In plain terms, the veto of item VI of art. 33 of the law transfers to researchers a duty that, strictly speaking, falls to the Unified Health System (SUS) under art. 196 of the Federal Constitution. The veto also subverts the constitutional requirement that the Unified Health System be funded with public, not private, resources (art. 198, paragraph 1 of the Federal Constitution).

This is a political choice that distorts the constitutional mandate assigning to the State the duty to promote health. Contrary to the constitutional text, an unwarranted extension of an obligation was created by law, without even consistent grounds in the veto message prepared by the Presidency.

Note, in this regard, that the veto message itself is clearly contradictory, since it justifies the need to give effect to the constitutional precept of comprehensive public health actions and services by transferring to the private entity a duty that belongs to the State[1].

Distorting the democratic will expressed by the National Congress not only taints the new legal framework for clinical research with unconstitutionality, but also raises to the level of law a gap that already existed in Anvisa Resolution RDC 38/13.

That regulation, of an infra-legal nature, had introduced for private parties the institution of perpetual civil obligation in health law. In any case, it allowed for discussion about the actual extent of the obligation to supply the medicine free of charge after the study, especially in the case of rare diseases, which, it was hoped, would be clarified by the text sent for presidential approval. That is not what happened, however. And the signal sent to researchers was that the country could be more stable for promoting science.

What the veto message does not say, but can be inferred, is the real motivation behind it. And it is economic. After Anvisa grants health registration for the researched medicine and it becomes commercially available once its price is set by the Drug Market Regulation Chamber (CMED), there is a period during which it is not immediately available in the SUS.

This is because the medicine must go through a process to be included (incorporated) in the SUS, a decision that rests with the Ministry of Health, advised by the National Commission for the Incorporation of Technologies into the Unified Health System (Conitec). The process is usually slow. And for medicines to treat rare and ultra-rare diseases, it takes even longer. There is a hidden reason for this: these medicines are high-cost.

The Ministry of Health has no interest in incorporating them into the SUS because of their budgetary impact, preferring to leave the supply of these medicines to specific cases in which patients file lawsuits to compel the State to provide them.

In this context, the presidential veto seems to have a clear objective: to shield the State from lawsuits that would require it to supply the medicine in the interval between approval of its health registration and its incorporation into the SUS. The mechanism for doing so is to force the transfer of this constitutional duty to the researcher, on the pretext of not leaving the patient without assistance after the clinical study ends. The maneuver is ingenious and seeks to mislead public opinion.

This imposition must be called by its proper name. It is a lifelong civil obligation. And it is difficult to identify any other perpetual obligation in Brazil. In the legal system, there are examples of rights and obligations limited by the passage of time, such as copyright and intellectual property rights and Family Law obligations.

In other even more sensitive spheres, such as Criminal Law, there is a clear orientation against perpetuity. If even in matters involving public order — a fundamental pillar of the Republic — responsibilities are not perpetual, it is unreasonable to transfer to the sponsor a burden disproportionate to the benefits it obtains, and to those it provides to society, by conducting clinical research in Brazil.

In reality, sponsors should receive encouragement and incentives and enjoy greater legal certainty, since they invest massively in improving the population’s health, which brings long-term savings to the public treasury and fosters research that advances the life sciences.

The veto is even more problematic for studies researching treatments for ultra-rare diseases. These studies enjoyed a 5-year limit on post-registration supply under art. 3, paragraph 1 of CNS Resolution 563/17[2]. With the veto, there is a serious risk of new interpretations, bringing even more regulatory instability and driving away new studies in this field.

There is also an important unanswered question about the obligation to supply medicines for studies already under way or completed. Should these cases follow the rule in art. 33, item VII[3] if these therapies are incorporated into the SUS, or, as some argue, would the study sponsor retain the perpetual civil obligation? The debate is not simple. And the discussion presented here is not intended to strike down the rights of clinical research participants.

These volunteers agreed to take part in pioneering studies for the sake of science, and most of the time they live without hope of improving their quality of life or, worse, with terrible prognoses for the progression of their conditions. They do not, therefore, seek perpetual guarantees: they want scientific research to take place in Brazil and thus give them a dignified quality of life.

What is proposed is that the resources that would be spent on supplying the therapy free of charge for life, even after it becomes commercially available (when this obligation should, strictly speaking, belong to the State), be used to fund new research. These new studies would benefit science and national industry and, above all, improve the health and quality of life of Brazilian patients.

Much is said about the cost of innovating, but little is discussed about the burden of a lack of scientific innovation in the country. How much would the world have lost if other countries imposed the same perpetual obligation? Therapies now considered established might never have been discovered if researchers and sponsors in developing countries had had to bear similar obligations.

Perhaps this is one of the reasons why Brazil, with so much natural wealth, biodiversity and prominent scientists working abroad, has a modest track record in creating revolutionary health therapies compared with countries such as the United States and several members of the European Union.

There is, however, one last chance to revisit this situation. The President’s veto is under review by the National Congress. It must be assessed by June 28 so as not to block the legislative agenda. It is therefore essential that this debate continue until that deliberation, with broad participation and technical discussion of these far-from-trivial issues.

[1] “Therefore, to give effect to the constitutional precept of comprehensive public health actions and services, the experimental medicine must continue to be supplied to research participants, regardless of its commercial availability through the private sector”.

[2] Art. 3 In research on ultra-rare diseases, the sponsor must take responsibility for and ensure that all research participants, at the end of the study, have free access to the best prophylactic, diagnostic and therapeutic methods that have proven effective, for a period of five years after registration is obtained from the National Health Surveillance Agency (Anvisa).

• 1 In the case of medicines, the 5 (five) year period will be counted from the setting of the price in reais by the Drug Market Regulation Chamber (CMED).

[3] “Art. 33. Free supply of the experimental medicine under the post-study supply program may be discontinued, upon submission of a justification to the Research Ethics Committee (CEP) for review, only in one of the following situations:

(…)

VII – availability of the experimental medicine in the public health network”.

Originally published by Jota (in Portuguese).