The highly expected guide to the shortened development path has come out, but before celebrating this, it is important to understand exactly what it is about, and the requirements necessary to succeed on this path.
The abbreviated development pathway is one where part of the information needed to prove safety and efficacy and subsidize the registration of a new or innovative drug, comes from studies not conducted by or for the applicant.
The main purpose of this registration route is to foster innovation without the need to repeat studies, since it is based on the premise of using prior knowledge of information about the API or about the drug. To follow through with this objective, the following are accepted as evidence to prove safety and effectiveness:
· Studies conducted with the drug being proposed for registration; studies conducted with the API.
· Studies conducted with another drug with the same API as the drug being proposed for registration,
· studies conducted with other salts, isomers, esters, ethers, complexes or other derivatives of the API that make up the drug that is being proposed for registration, or scientific literature.
But it is noteworthy that even using pre-existing data, it is necessary to present all the non-clinical and clinical information necessary for the risk-benefit assessment of the medication, remembering that ANVISA strongly recommends that the registration dossier be submitted in the format of the Common Technical Document (CTD), according to Guide nº 24/2019.
This implies that all items necessary for the evaluation of the benefit-risk of the drug, both from the non-clinical and the clinical part, must be filled with robust data from literature or studies carried out with the innovative product.
This is where the concept of bridge study comes in, with the purpose of establishing the correlation between drugs or populations in order to allow the extrapolation of efficacy and safety data. The bridge study does not necessarily have to be a clinical study and it will be up to the developing company to justify the choice of the study and prove that it allows the extrapolation of data.
But how to elaborate a good development rationale and choose what type of bridge study should be carried out?
There is no ready-made recipe, but when constructing the rationale for the development of an innovative medicine through the abbreviated route, it is essential to keep in mind that the generation of new safety and efficacy data must fill the existing knowledge gaps in view of the impact of the proposed changes and available data.
In other words, one should evaluate the available information, check if they are extrapolable for the drug being developed, and define which studies.
Additional steps should be taken to answer all questions regarding the safety and efficacy of the new drug in order to demonstrate a positive benefit-risk balance.
Keep in mind that ANVISA's decision-making process will consider the available efficacy and safety data, the consistency between these data, the balance of the benefits and risks of the drug in the light of the therapeutic context of the target indication and the result(s) clinical trial(s).
There is a lot of uncertainty and insecurity on how to carry out the developments in the shortened way, considering we do not yet have this expertise fully installed in Brazil, but with determination and partnership between the pharmaceutical sector and ANVISA; which includes the transparent discussion of projects based on well-studied and well-founded proposals I am sure, that this regulatory framework for innovation will be a great opportunity to generate innovative medicines with great benefits for patients and companies.