Read the Conversation

EF: What attracted you to the sector? 

FA: My passion for science, fueled by my family's experiences, has driven my choice to pursue a career in the pharmaceutical industry. Over the years, I have devoted myself to oncology and hematology, knowing the immense impact these fields can have on patients' lives. This shared purpose has been the cornerstone of my unwavering commitment to my work. It inspires me to go above and beyond the day-to-day tasks of my profession and collaborate with my team to make a tangible and positive difference. 

EF: What innovative plans does Vertex have for 2023 in terms of the portfolio? 

FA: We find ourselves at an incredibly exciting moment for Vertex in Brazil, particularly in our journey to bring transformative therapies against cystic fibrosis (CF) to the country. Until now, cystic fibrosis patients in Brazil have primarily had access to medicines that address symptom management, only scratching the surface of what is currently possible. With eight years of presence in the Brazilian market, including establishing a quality assurance lab near São Paulo, we are now experiencing a pivotal evaluation with the Health Regulatory Agency regarding our latest triple combination therapy. This evaluation marks a significant step forward, unveiling the profound impact of our therapies, which target the root cause of the disease. 

As a leading biotech in the field of rare and serious diseases, our approach to research is rooted in addressing the underlying causes of diseases. This focus enables us to develop innovative medicines that try to significantly impact patients' survival and quality of life. We are optimistic about future access in Brazil. Although it presents challenges, ensuring the availability of our innovations to patients is a clear mission for us. We are committed to collaborating with healthcare authorities to contribute to sustainable reimbursement and positively impact the CF community in the country. 

EF: Could you elaborate on how you are building strategic partnerships in the region to increase access? 

FA: As a company, we recognized the increasing complexity associated with personalized medicine and we have actively embraced collaborations with other companies, understanding that collective endeavors yield greater impact. This approach is evident at every stage of our research and development. We strive to foster a culture of collaboration and partnership at the local level, reinforcing our commitment to innovation. 

Firstly, we forged a significant partnership with a renowned private laboratory in Brazil, known for its exceptional quality standards. This collaboration focuses on genotyping the country's cystic fibrosis (CF) patients. Our support has contributed to more than 83% of CF patients being included in the national CF registry managed by the medical society. By comprehensively understanding the mutation profiles among Brazilian CF patients, we determine treatment eligibility and gather valuable insights into the overall population's mutation profile, potentially aiding in prognostic assessments. 

Another noteworthy example revolves around the nutritional needs of CF patients. Considering that the average survival age of CF patients in Brazil is still relatively low compared to the United States and Europe, maintaining a high-calorie diet is crucial. To address this requirement, we have established partnerships with international nutrition companies enabling us to support CF patients by facilitating high-calorie intake. These collaborations exemplify our dedication to providing comprehensive care for CF patients beyond medical interventions. 

Furthermore, we actively contribute to medical education initiatives through various societies encompassing the multidisciplinary management of CF patients, enhancing knowledge and expertise within the CF community. 

EF: Can you elaborate on your research on rare diseases and their relation to personalized medicine? 

FA: As a company, our strategic focus encompasses various indications, including emerging hematological conditions like sickle cell disease, which exhibits a notably high prevalence in Brazil, type 1 diabetes and kidney disease research. The common thread connecting these areas lies in our sandbox strategy, which targets conditions with high unmet needs and understood the biological cause of the disease. By comprehending the underlying mechanisms and specific mutations responsible for these diseases, we can design more tailored and personalized clinical trials that address the root causes of the conditions. This paradigm shift contrasts with the traditional approach of testing a compound's preclinical activity without a deep understanding of the disease. We can approach treatment more specifically by gaining insights into the genetic mutations driving the diseases and understanding their mechanisms of action. 

EF: How do you rate the potential of Brazil as an innovation hub and how can more investment be attracted to the region? 

FA: Brazil consists of 27 distinct regions. Hence, when discussing Brazil, it is essential to exercise caution and avoid seeking a single definitive response. Undeniably, Brazil holds immense potential, a notion that is frequently reiterated. However, it is time to move beyond these assertions and substantiate them through concrete actions. 

Over the past few years, there have been ongoing discussions regarding the need for streamlined and expedited approval processes for clinical trials within the country. The approval system involves multiple decision-making layers, including a national ethical body, a coordinating reference center, and participating centers. Brazil has previously exhibited a propensity to introduce reforms that foster progress, particularly in big pharma.  

This country possesses one of the swiftest recruitment rates in the world. Its exceptional centers, scattered throughout the nation, uphold the highest quality standards. Furthermore, the prevalence of certain pathologies in these areas is substantial, presenting a remarkable advantage.  

Meanwhile, the protection of intellectual property rights is imperative for enticing investments and integrating new technologies into the existing framework. Brazil has made commendable efforts in this regard. Nevertheless, given the complexity of the region, it is paramount that we uphold and maintain these protective measures as they constitute a foundational element. 

As for political stability: historical evidence demonstrates that when this country experiences a period of stability, it exhibits remarkable growth, resulting in higher GDP levels and increased foreign investment.  

It's important to recognize that the equation for sustainability differs between developed and mid-level GDP countries. In a country as vast as Brazil, it becomes crucial to align our strategies with specific states and countries. We openly discuss the costs associated with our technologies for the public healthcare system and strive to engage in dialogue with authorities, offering assistance in their pursuit of sustainability. This often involves exploring risk-sharing agreements that mitigate patient response rates’ uncertainties.  

Highly specialized companies like Vertex exemplify a remarkable sense of purpose. Rare disease-focused organizations are driven by an unwavering commitment to a small patient population. Each new patient entering treatment is cause for celebration. These celebrations are not fueled by mere sales figures but rather by the profound impact these treatments have on patients' lives. Working for such a company is a privilege, as it entails representing medicines that directly influence patients’ quality of life. In this context, every patient holds immense value and is cherished with utmost importance. 

EF: What are Vertex's additional initiatives to raise awareness about cystic fibrosis? How does the company leverage digitalization to enhance overall awareness efforts? 

FA: First and foremost, dispelling a prevailing myth regarding Brazil. Having spent considerable time here, I can confidently assert that the notion of the limited availability of reputable organizations and high-quality data is false. Therefore, the foundation lies in leveraging data to enhance our understanding of the market and inform decision-making.  

One of our initial and commendable decisions was to ascertain the essential requirements for supporting and optimizing the national registry, making it accessible to the 52 reference centers across Brazil. This endeavor has proven to be a pivotal step, offering significant support. The longevity of the national registry, spanning over 12 years, attests to its high quality and reliability. Moreover, it strongly correlates with medical education, facilitating discussions beyond international prevalence numbers. By incorporating the perspectives of physicians and experts, the registry sheds light on the reality of cystic fibrosis in Brazil.  

Today, we possess a comprehensive understanding of disease patterns in Brazil, and we dare to acknowledge the influence of migration patterns within the country and across Latin America. This nation, characterized by its cultural diversity, serves as a melting pot, as evident in the invaluable insights derived from the Gemma project and the interconnectedness of diseases with historical migration patterns. 

The tireless efforts of patients have driven our initiatives, such as virtual roadshows, which initially commenced as physical events but adapted to the post-pandemic context. These roadshows bring together multidisciplinary teams, including psychologists, nutritionists, and physiotherapists, engaging in sessions with groups of patients. During these sessions, experts and medical professionals deliberate on vital therapy aspects significant for cystic fibrosis patients. This comprehensive approach to patient care exemplifies the entirety of the patient journey and showcases the adaptability of our services to the evolving post-pandemic landscape. Here, Telemedicine, which experienced a significant surge during the pandemic, has become a transformative component of our portfolio, signifying how companies are shifting towards more service-oriented approaches. 

EF: What are the main pillars to keep a sustainable business in innovative pharma like Vertex? 

FA: The key factor that can truly drive progress and foster breakthroughs lies not in individual efforts alone but rather in the collaborative endeavors of a team or company, even when faced with formidable obstacles.  

Amidst the challenges of the pandemic, financial crises, and political turmoil, it is crucial for us to reflect upon the profound lessons we have learned. The pandemic served as a poignant reminder that we rely on one another, dispelling any notions of self-sufficiency. The enduring lesson that will resonate throughout my life is the recognition of interconnectivity. This realization reinforces the notion that if we seek support from others, we must also contribute in kind. The concept of partnerships emerges as the cornerstone of this understanding. In this era, collaboration becomes paramount, whether pursuing specific goals or driving transformational objectives. At Vertex, we claim to possess only some of the answers. We emphasize the value of connections and partnerships, which undoubtedly shape the future landscape. 

Furthermore, in an industry renowned for its innovative prowess, it is essential to embrace humility, acknowledging that despite our deep understanding of certain areas, such as cystic fibrosis, a small proportion of patients remain ineligible for our therapies. Therefore, maintaining humility and persisting until every patient has a viable treatment option becomes paramount—a blend of resilience and forward-focused determination. The imperative to innovate extends beyond business practices and permeates our daily lives. Merely repeating past successes is inadequate in the face of rapid change. Thus, integrating an innovative mindset into corporate work culture becomes challenging yet indispensable for progress. 

June 2023