The Rare Diseases Series: Interview with Ana Bozas (Part 2)

In the intricate and evolving realm of healthcare, effective communication stands as a critical pillar, especially in the underrepresented and complex field of rare diseases. As medical advancements burgeon, the need for clear, concise, and impactful dissemination of scientific knowledge becomes paramount. Read on here for more information on this topic.

The spotlight of this series of interviews is on the vital role of medical communications in bridging the gap between groundbreaking research and its application, providing clinicians, patients, and other stakeholders with access to essential information. We delve into the challenges and triumphs of this endeavor, through the lens of a leader in the field who has dedicated their career to enhancing the understanding and treatment of rare diseases. This discussion aims to illuminate the nuances of medical communications and its profound impact on the rare disease community.

For the inaugural edition, we approached Ana Bozas, PhD, CMP (Director, Ipsen) for an interview and she graciously accepted. Here’s the link for her interview (part 1). In part 2 here, we discuss more about her methods, strategies, and experiences in this field, as well as the important geopolitical changes in the rare disease landscape.

Q1. What methodologies or tools do you use to assess the impact and effectiveness of your rare disease product communication efforts?

“At the first level, we focus on quantitative metrics – the numbers. This includes tracking the number of readers, downloads, instances where our content is reposted, and overall traffic to our platforms. These are the most straightforward indicators of audience reach or attention for this particular publication in the greater pool of scientific communications and represent the ‘lowest hanging fruits’ in our evaluation process.

The second level delves deeper, focusing on comprehension and retention of information. We frequently poll our audience during conference presentations or webinars to gauge their understanding of the material presented. Additionally, we conduct postmortem memory checks after conferences and review papers to ensure that the key messages are not just heard but understood and remembered. This level is crucial for gauging the clarity and effectiveness of our communication – and it’s necessary because sometimes you’d be surprised what people actually remember versus what you think they’d remember!

The third and most challenging level evaluates the real-life impact of our communication. We aim to understand how our efforts are translating into tangible benefits for the patient and medical community. This includes observing changes in physician behavior, improvements in the quality of care, and shifts in treatment guidelines. To assess these changes, we conduct surveys with various stakeholders, including physicians, patients, and patient organization groups. We also review reports from market access/HEOR colleagues about how payers (insurance companies, etc) perceive current or shifting standard of care, to understand how our communication might be influencing the care route. However, measuring success at this level can be challenging due to the complexity of the variables involved, and it also take some time post-publication before an effect becomes visible enough.

Overall, our approach is comprehensive, incorporating quantitative metrics, comprehension assessment, and real-life impact evaluation to ensure that our communication efforts are not only reaching our audience but also making a meaningful difference in the rare disease community.”

Q2. What strategies do you employ to stay updated on the latest advancements and research related to rare diseases?

“To stay updated on the latest advancements and research related to rare diseases, I employ several strategies that help me keep up with key developments in this rapidly evolving field.

One of my primary methods is regularly reading scientific literature. This includes peer-reviewed journals, research papers, and clinical trial results that are directly related to rare diseases. Diving into these resources is necessary to stay informed about the latest findings, treatment approaches, and theoretical advancements in the field.

I also subscribe to a variety of professional newsletters. These newsletters are curated by experts and organizations that specialize in rare diseases. They provide a concise and timely summary of the latest news, research breakthroughs, and industry developments.

Finally, given the increasing role of artificial intelligence in healthcare, I make it a point to stay informed about the latest AI updates, especially those relevant to rare diseases. AI and machine learning are revolutionizing how we analyze data, recognize patterns, and even predict disease trajectories. Therefore, keeping abreast of these technological advancements is crucial for applying the latest techniques to my work in rare disease scientific communications.

These strategies collectively enable me to maintain a comprehensive understanding of the field, ensuring that I am well-equipped to provide advice that is up to date with evolving trends or standards and to contribute effectively to the body of knowledge on rare diseases.”

Q3. Have you encountered situations where you had to adapt your communication strategy for different rare diseases or specific stakeholders? How did you approach this?

“In my career, I’ve often faced the challenge of the right communication to the right person when communicating the effectiveness of various treatments or interventions, particularly when speaking to very different audiences, like physicians, health insurers, patients, and even the general public as they each have different concerns and perspectives.

In case of physicians, the focus naturally shifts towards the clinical aspects of a treatment. They delve into the specifics like lab values, clinical signs, and other measurable outcomes. It’s a language of precision, grounded in medical knowledge and expertise. For example, when discussing the effectiveness of a new drug with a physician, I would emphasize its impact on clinical outcomes or specific biomarkers associated with long-term clinical outcomes, its safety profile, and how it compares with the current standard of care.

However, the conversation changes significantly for patients or a lay audience. Here, the emphasis is more on how a treatment impacts their daily life, activities of daily living (ADL), and overall quality of life (QOL). Patients are generally less concerned with the minutiae of lab values or clinical jargon. They want to know if a treatment is improving their health, but they’ll equally want to know how a treatment will affect their symptoms, their ability to perform daily tasks, and their overall wellbeing.

Balancing these two perspectives requires not only a deep understanding of the subject matter but also empathy and effective communication skills. Medical communication professionals need to be able to translate complex medical information into something meaningful and understandable for non-specialists, without losing the nuances that are important for a professional audience.

In essence, whether I’m communicating with physicians or patients, my goal remains the same: to convey information in a manner that is both accurate and relevant to the audience I’m addressing. This approach ensures that all can make informed decisions about their healthcare, based on a clear understanding of the potential benefits and implications of different treatment options.”

Q4. How do you handle time-sensitive communication needs, such as responding to emerging data or crisis situations for rare disease products?

“Handling time-sensitive communication needs, particularly in the context of rare diseases, requires a nuanced approach. While rare diseases themselves are seldom emergent in the traditional sense, there are situations where urgent communication is paramount.

One such scenario was observed during the COVID-19 pandemic, which presented a unique challenge. The pandemic created a crisis where access to resources became critically limited. Hospitals were inundated with COVID cases, focusing on triage for emergency care. In such situations, patients with rare diseases often found themselves at the end of the line for medical attention. This was not due to the nature of their diseases being less severe, but because of the overwhelming demand on healthcare systems prioritizing acute and infectious cases.

In these times of crisis, our field’s (biotech and pharma) approach to communication had to be both immediate and empathetic. It involved keeping patients and their families informed about the changing healthcare landscape and what it meant for their treatment and care. We had to ensure that they were aware of the potential delays in receiving care and help them navigate these challenges. This involved directing them to alternative resources or care settings when possible and advocating for their needs amidst a strained healthcare system.

Another aspect of time-sensitive communication in rare diseases pertains to specific patient groups like those with pediatric (newborn) conditions or those at the end of life. These situations are inherently more urgent and are managed differently. For newborns with rare diseases, early intervention can be crucial for their long-term prognosis. Therefore, communicating with healthcare providers and parents quickly and effectively is essential to initiate timely assessments and interventions. Similarly, for patients with rare diseases who are at the end of their life, it’s vital to have prompt and compassionate communication about their care options, ensuring their comfort and dignity during their remaining time.

In all these scenarios, the medical communications role involves not just conveying information swiftly but also being sensitive to the emotional and psychological impact of these communications. It’s about providing clarity and guidance during times of uncertainty and stress, ensuring that, despite the urgency, the needs and concerns of rare disease patients are not overshadowed.”

Q5. What trends and innovations do you see in the field of scientific medical communication, particularly in the context of rare diseases?

“In the field of scientific medical communication, especially in the context of rare diseases, there have been significant trends and innovations in recent years. These changes are reshaping how we approach rare diseases from a research, regulatory, and treatment perspective.

Traditionally, the concept-to-market journey for rare disease products can span 10-20 years. This prolonged development period is due to the complexities inherent in researching and developing treatments for conditions that are not well understood and have small patient populations.

In 1983, the U.S. government passed the Orphan Drug Act (ODA) to help stimulate innovation in an area that is difficult to study or innovate. This move signaled a shift, placing rare diseases as a focus area. It recognized the unique challenges posed by these conditions and aimed to incentivize research and development in this area. Over the last 4 decades, there’s been a notable increase in the advent of products targeting rare diseases: The number of designations granted more than quadrupled between the 1990s and 2010s (Miller, 2021, https://ojrd.biomedcentral.com/articles/10.1186/s13023-021-01901-6) and is still on a growing curve. This shift is partially driven by regulatory incentives and a growing recognition of the unmet needs in this domain.

Currently, about 90% of rare diseases have no FDA-approved treatment, highlighting the significant unmet need. Orphan diseases, defined by a certain threshold of frequency of occurrence (1:200,000 in the US, https://www.ncsl.org/health/rare-and-orphan-diseases ), therefore benefit from additional help from the government to make the process of medical research more sustainable. This includes incentives like tax credits for the costs of clinical research, government grant funding, assistance for clinical research and a seven-year exclusive marketing period, allowing companies to recoup the substantial costs associated with rare disease research. In addition, both the EMA and FDA have played pivotal roles in shaping the landscape for rare disease treatment development through the orphan drug designation. This regulatory framework has been instrumental in encouraging pharmaceutical companies to invest in rare disease research.

Over the last few years, there have also been leaps in the speed of research and development, largely fueled by advances in computational power, big data analytics, and artificial intelligence. These technologies enable more efficient data analysis, better understanding of disease mechanisms, and faster drug development processes.

In summary, the field of scientific medical communication in the context of rare diseases is evolving rapidly, driven by regulatory changes, technological advancements, and a growing understanding of the unique challenges and opportunities in this area. As we continue to see progress in these domains, the hope is to address the significant treatment gaps and improve the lives of individuals with rare diseases.”

Q6. Can you share your experience in collaborating with cross-functional teams, such as R&D, regulatory affairs, and marketing, to achieve communication goals for rare disease products?

“Collaborating with cross-functional teams in the context of communication for rare disease products has been an integral part of my experience. While, in many ways, similar to other areas of medical communication, the rare disease field presents unique challenges that require a cohesive and aligned approach across different functions.

The foremost requirement in this collaboration is ensuring that everyone is “rowing in the same direction”. This means aligning the goals and strategies of R&D, regulatory affairs, medical affairs, and marketing/other commercial functions. Given the complexity and sensitivity surrounding rare diseases, it’s crucial that all departments understand and support a unified vision and approach.

My interaction with R&D teams typically involves bridging the gap between academic research and its practical application in the industry. Researchers in R&D are often more focused on the scientific and technical aspects of rare disease products. For example, when preparing a scientific poster or presentation, they might be more focused on presenting a specific set of experiments and less on thinking about the greater journey of discovery and overall key scientific message about that specific product – which is typically more of a medical affairs perspective. This is where my role becomes critical in ensuring that the scientific communication is accurate yet accessible to a broader audience, including non-specialists.

In addition, without proper coordination, there’s a risk of conflicting messaging. If R&D pursues one narrative while medical affairs or marketing another, it can lead to confusion and dilute the effectiveness of our communication. Therefore, my role often involves ensuring consistency in messaging across all functions. In my experience, medical affairs act as the custodian of science communication and represents the company’s voice in the scientific community. We ensure that the information disseminated is not only scientifically accurate but also aligns with the broader goals and compliance requirements of the company. This involves a delicate balance between scientific rigor and transparency and the practicalities of business and regulatory compliance.

Working with marketing teams is another critical aspect. They bring a strategic viewpoint to product communications, focusing on how to position the product in the market effectively. My collaboration with marketing ensures that while they are responsible for commercial messaging overall, the scientific integrity and sensitivity surrounding rare diseases are not compromised, and the message remains scientifically accurate.

In conclusion, achieving communication goals for rare disease products requires a collaborative, multidisciplinary approach. Each function brings its expertise and perspective, and my role often lies in harmonizing these diverse viewpoints to create a coherent, effective communication strategy. This collaboration is vital not just for the success of the product, but more importantly, for accurately and ethically representing information that significantly impacts patients’ lives.”

Q7. How do you ensure that your rare disease communication efforts are aligned with the overall business goals and objectives of your organization?

“Ensuring that my medical communication efforts align with the overall business goals and objectives of my organization is a multi-layered process that requires a thoughtful and strategic approach. It’s essential to integrate these communication efforts with the broader vision and mission of the organization.

It all starts with a clear understanding of the organization’s overarching business objectives. What is the ultimate goal we are trying to achieve? This could range from developing innovative treatments for rare diseases to providing comprehensive support systems for patients and their families. Understanding these objectives sets the foundation for all communication efforts.

A key part of our business objectives involves considering what we aim to achieve for our patients. This perspective is crucial, especially in the field of rare diseases, where patient needs can be complex and multifaceted. My communication strategies are designed to reflect and support these patient-centric goals.

We need to ensure that the communication strategies are in sync not just with the overall organizational goals but also with the specific goals of different departments, whether it’s R&D, HEOR, or regulatory affairs. This involves regular discussions and collaboration with various department heads to understand their priorities and how communication can support their objectives.

As part of a larger team, I also consider how my individual goals and actions contribute to the broader objectives. This reflection helps in fine-tuning strategic direction and ensuring that my efforts are adding value to the chosen objective.

The opposite of this structured approach is a lack of coordination and direction, leading to confusion or conflicting messages – a situation akin to anarchy. To prevent this, it’s essential to have clear, consistent communication channels cross-functionally and a shared understanding of our goals and how we plan to achieve them.

In summary, aligning rare disease communication efforts with the business goals of the organization involves a comprehensive understanding of the broader objectives, integrating patient-centric approaches, aligning with departmental goals, and ensuring individual efforts contribute meaningfully to the collective goal. This alignment is key to ensuring that communication efforts are not only effective but also resonate with the organization’s mission and values.”

Q8. Finally, what advice would you give to aspiring professionals looking to excel in the field of rare disease scientific publications and medical communication?

“If you want to succeed in medical affairs (rare disease or otherwise), you need to have passion, curiosity, and dedication. You need to give your best to this work every day. You don’t need to aim to be the top in your field (though if you do, that’s great!), but you do need to give 100% as often as you can and not give up when you face challenges or setbacks. You also need to remain interested in the many facets of science and keep your mind open to new things — new discoveries, new technologies, new points of view. You can continually learn from the latest research, your colleagues, the medical experts, and the patients. By coming to work with an ambition to do your best work every day and an open mindset to scientific novelty and wonder, you can both be personally and professionally motivated and make a difference in the rare disease community.”

I had a lot of fun developing this interview! Sincere thanks to Ana for giving me an opportunity to interact with her and glean important aspects of medcomms in rare diseases.

Disclaimer: The final content has been processed using AI for note-taking efficiency; however, the initial interview and all insights are derived from a human source.

Author:

Dr. Anupama Kapadia
General Manager, Enago Life Sciences
Connect with Anupama on LinkedIn