RIO DE JANEIRO – As the second day of the BIO Latin America conference continued discussions on how to build a innovation-friendly infrastructure in the region, the talk turned to the role of clinical trials, particularly in Brazil, where, despite being one of the top global spenders in the pharmaceutical market, has trailed the other BRIC countries over the last few years in the clinical trial space.

According to a white paper published in May by Thomson Reuters, Brazil's regulatory approval process, though improving, still remains one of the lengthiest in Latin America, stretching anywhere from five months to a full year. The local regulatory agency, Agencia Nacional de Vigilancia Sanitaria (ANVISA), recently implemented steps to simplify the process.

But there's no question there have been "a lot of hurdles over the last decade," said Carlos Gil Ferreira, of the Brazilian National Cancer Institute, during a Wednesday morning session. He ticked off several, including a regulatory environment unfriendly for research and registration, politics that make it difficult to collaborate and to establish competitive clinical networks for research.

He also pointed to the "lack of expertise in early development." Brazilian clinical sites have "a lot of experience enrolling in global [late-stage] trials, but few centers have expertise in running phase I [and] phase II and transitioning from preclinical to clinical."

Yet, even as ANVISA works to streamline the clinical trials approval process, it also will have to consider the changing landscape in clinical testing that's been happening worldwide to accommodate the increasing demand for more efficient and less costly clinical programs and the increasing shift toward personalized medicine.

As an expert in oncology, Ferreira has seen the latter firsthand, with the introduction into oncology the "era of molecular diagnostics" and treatment. For instance, in a subset of non-small-cell lung cancer (NSCLC) patients, the mutation anaplastic lymphoma kinase, or ALK, has been identified, and those patients can be treated with ALK inhibitors such as Pfizer Inc.'s Xalkori (crizotinib). But in Brazil, the estimated number of NSCLC patients with ALK mutations is unknown due to a lack of molecular epidemiology studies in the region.

"We don't know if the Brazilian population [has] similar mutations as Caucasians" or other groups, Ferreira said. If regulatory agencies in Brazil are going to cover ALK inhibitors, "we need to know how many patients in Brazil have that alteration."

The country also has a collaboration with the Cuban government in the area of in vitro diagnostics.

Ferreira said the hope is that moving forward with the epidemiology studies and collaborations will be able to offer successful examples to convince regulators in the ongoing discussions for improving the clinical trial process.

"All those studies can help us learn how to work and build a new environment for clinical research in Brazil," he said.

Of course, Brazil is hardly alone when it comes to figuring out how to speed up clinical testing – the aim to "fail soon, fail cheap," said Andrew Robertson, director of U.S. regulatory policy for Merck & Co. Inc.

In the U.S., the FDA has been wrestling with a new way of thinking about the traditional double-blind, randomized trial protocol, opting instead for new ideas – predictive biomarkers, adaptive designs, Bayesian statistics, Robertson said. The aim is "to look at the data as emerging and use that to inform" the process, reducing costs significantly and requiring small patient enrollments.

He pointed to two efforts in particular: The Lung-MAP program, a multidrug, biomarker-driven squamous cell lung cancer trial designed to match patients to multiple substudies testing investigational treatments targeting genomic mutations of their cancers, and I-SPY 2, which was designed to use Bayesian predictive probability to determine whether a regimen containing pan-HER tyrosine kinase inhibitor neratinib would prove statistically superior to standard therapy in metastatic breast cancer. (See BioWorld Today, Dec. 6, 2013, and April 9, 2014.)

"You get to a point where you can actually weed out not-promising drugs much, much sooner," Robertson said, adding that the "message is [the] FDA is receptive to this."