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Blog-Article 30th of May, 2025

ATMPs: From Long-Held Dream to New Reality in Medicine

For decades, it was a long-held dream in genetic medicine to repair the broken genes underlying incurable diseases. While mutations could be identified, the tools to correct them were missing. Direct gene modification remained an aspiration — and now, it is accelerating a new era in medicine. Advanced Therapy Medicinal Products (ATMPs) — encompassing cell and gene therapies — allow us to correct broken or misspelled genes and to reprogram the immune system itself, engineering our own cells to recognize and destroy malignant targets. An era that is bringing curative treatments for conditions once considered untreatable.

However, this new era comes with its own set of challenges. The industrial infrastructure — the machinery and systems needed to manufacture these highly complex therapies — was not designed for the precision and individualization required by ATMPs. Unlike conventional drugs or even first-generation biologics, cell and gene therapies demand bespoke production processes, strict control over biological variability, and highly specialized logistics. As a result, the traditional pharmaceutical manufacturing model — optimized for large batches of standardized medicines — has been stretched to its limits.

Beyond manufacturing, regulatory frameworks have also struggled to keep pace with the innovation. Many ATMPs do not fit neatly into existing approval pathways, and long-term outcome data — critical for assessing true therapeutic value — often remain incomplete at the time of initial authorization. Regulatory agencies are adapting, but uncertainties around clinical trial design, endpoints, and post-approval requirements continue to create challenges for developers.

Pricing and market access present yet another layer of complexity. The high upfront costs of ATMPs reflect their curative potential and the complexity of development and manufacturing. However, existing reimbursement systems — typically built for chronic treatments rather than one-time interventions — are not always equipped to handle such models. Aligning the value of these therapies with sustainable healthcare financing remains a critical, ongoing conversation among developers, payers, and policymakers.

In parallel, another revolution has been quietly reshaping the field. Advances in biocomputing, particularly the rapid rise of AI and machine learning, have introduced new capabilities into medicine. AI is now being applied across the ATMP development pathway — from optimizing gene-editing techniques and vector designs to predicting patient-specific responses and enhancing manufacturing efficiency. These technologies not only support innovation but also offer solutions to some of the most critical bottlenecks in bringing advanced therapies to patients.

The convergence of genetic medicine with biocomputing represents a profound shift. It is not only redefining what is scientifically possible but also demanding that the entire system — from research to manufacturing to clinical delivery — evolves alongside it.

While the journey is still ongoing and challenges remain, the foundation for a new therapeutic paradigm has been laid. ATMPs are no longer a future promise — they are a present reality, steadily reshaping medicine and opening the door to treatments once thought unattainable.

It is a remarkable time to be part of the field of medicine. The convergence of genetic innovation and computational power is not only advancing science but also redefining how we can help patients. For those working in medicine today, few moments in history have offered such a combination of discovery, impact, and opportunity to shape the future of healthcare.

Mira Kyttälä  30th of May, 2025

Blog 5th of April 2025

How Machine Learning Rekindled My Enthusiasm for Small Molecule Drug Development

Embarking on a machine learning (ML) course, I expected to deepen my technical understanding of ML applications. Yet, I didn't foresee it profoundly altering my perspective toward small molecule drug development—a field I had somewhat overlooked due to my deep involvement and enthusiasm for gene therapies (GTx) and cell therapies, areas aligned closely with my own research background.

The course shed light on the potential of ML to model proteins and their cellular functions with remarkable precision. This was a pivotal insight for me, illuminating how small molecules, often overshadowed by the innovative allure of biologics and ATMPs, could be revitalized through sophisticated ML techniques.

Having been focused on ATMPs—drawn to their promise of curing diseases at a genetic level, an area that fascinated me since my days in gene identification research—I had been somewhat blind to the broader utility of small molecules. This course opened my eyes to their potential in offering scalable, cost-effective solutions that could address global health challenges where ATMPs, despite their transformative potential, fall short due to logistical and financial constraints.

Small molecules have always been fundamental in drug therapy for their manufactural simplicity and ease of use. Yet, it's through the lens of ML that their full potential is re-emerging—enhancing drug specificity, reducing side effects, and accelerating discovery.

This ML course didn't just enhance my skills—it transformed my outlook on pharmaceuticals, reigniting my enthusiasm for the potential of small molecules in a new era of drug development. As we continue to harness the power of ML, I am optimistic about our ability to make significant strides in crafting tailored, effective small molecule therapies that can truly be accessible worldwide.

Mira Kyttälä  5th of April 2025

Blog 23rd of June 2024

Reflections on Medical Affairs in the ATMP & AI Era: A Delicate Balancing Act

The pharmaceutical industry is rapidly evolving with the emergence of Advanced Therapy Medicinal Products (ATMPs) and the exponential growth of artificial intelligence (AI). This evolution is prompting a reevaluation of traditional strategies. While some hesitate, others are boldly venturing into this new frontier, even without fully understanding the implications.

Amid this uncertainty, one thing is clear: Medical Affairs is undergoing a profound transformation. The traditional role of communicating scientific information is expanding to encompass a wider range of responsibilities. Today, these professionals are called upon to generate real-world evidence (RWE), manage complex regulatory landscapes, and become passionate advocates for patient access to these transformative therapies. As personalized, potentially curative treatments like gene and cell therapies become more prevalent, the Medical Affairs function is more important than ever, bridging the gap between cutting-edge science and real-world patient care.

Real-World Evidence (RWE) ATMPs are often approved based on smaller, shorter trials due to the rarity of the diseases they treat. To understand their long-term efficacy and safety, robust RWE is crucial. Medical Affairs professionals are at the forefront of designing and conducting these post-market studies. AI-powered tools aid in efficient data collection and analysis, but it's the expert interpretation by Medical Affairs that transforms these findings into actionable insights for healthcare providers and payers.

Partners in Regulatory Success The unique mechanisms of action and potential risks associated with ATMPs demand specialized regulatory expertise. Medical Affairs professionals are crucial in supporting Regulatory Affairs colleagues by generating and disseminating local data and existing evidence, enhancing the access process for novel therapies and indication extensions. This includes collaborating on the development of comprehensive data packages, addressing regulatory queries, and ensuring the clear communication of the therapeutic value of ATMPs to regulatory authorities.

Advocates for Patient Access The high cost of ATMPs is a major barrier to access for patients. Medical Affairs teams work alongside Market Access colleagues to build a compelling value proposition for these therapies. This involves demonstrating their clinical and economic value to payers and health technology assessment (HTA) bodies, advocating for fair pricing and reimbursement policies, and ultimately ensuring that patients who can benefit from these life-changing treatments can actually get them. The recent U.S. approval of Sarepta's Elevidys for Duchenne Muscular Dystrophy (DMD) at a price of €2.9 million has amplified these concerns in Europe.

Educators and Trusted Partners As ATMPs become more common, Medical Affairs professionals are stepping into the roles of educators and trusted partners. They're tasked with explaining the complex science behind these therapies to HCPs in a way that's clear and understandable. This involves developing educational materials, hosting training sessions, and fostering open communication to ensure that HCPs are equipped to deliver the best possible care to their patients.

The AI Advantage: A Powerful Ally Artificial intelligence is a powerful tool that can supercharge the work of Medical Affairs professionals. It can automate time-consuming tasks like literature reviews and medical information requests, freeing up valuable time for strategic initiatives and patient engagement. AI can also help analyze vast amounts of data, uncovering patterns and insights that might be missed by the human eye.

However, AI is not a replacement for human expertise. It requires human oversight to ensure accuracy and to make sense of complex findings. Medical Affairs professionals remain the decision-makers, using their knowledge and experience to interpret AI-generated insights and apply them in a clinically meaningful way.

Adapting to Diverse Healthcare Landscapes The regulatory and healthcare landscape for ATMPs varies across Europe, and Switzerland has its own unique considerations. Medical Affairs teams must be adaptable and responsive, tailoring their strategies to local requirements and cultural nuances. This involves engaging with national regulatory agencies like Swissmedic, understanding local reimbursement policies, and collaborating with patient advocacy groups to ensure that the needs of diverse patient populations are met.

The Future: A Human-AI Partnership The future of Medical Affairs in the ATMP and AI era is one of collaboration and co-evolution. By embracing technological advancements and developing new skills, Medical Affairs professionals will continue to play a pivotal role in ensuring that these revolutionary therapies reach their full potential and transform the lives of patients worldwide. This is not a story of AI replacing humans, but rather of a powerful partnership where human expertise and AI capabilities work together to advance this complex and promising new frontier in medicine.

Mira Kyttälä  23rd of June 2024

Blog 28th of February 2024

Charting Success: Pharma Product Launches

Launching a pharmaceutical product in a new market is akin to setting sail on uncharted seas. Each of us in the pharma community knows this journey well, marked by the challenges of navigating through regulatory icebergs, cultural currents, and the diverse healthcare practices that define each new territory. It's a path that demands not just a keen understanding of the scientific and medical landscape but also a deep appreciation for the local nuances that can make or break a product's journey to market.

At Medconmirakoo, our collective voyage in this realm is powered by experience and a shared commitment to innovation in healthcare. We understand that launching a product is a monumental task that no one should navigate alone. Our team, seasoned by journeys across the regulatory and commercial waters of countries, stands ready to guide you through these complex terrains. We are the navigators who know where the hidden obstacles lie, from regulatory requirements to local healthcare practices that could derail a launch if not navigated with care.

Our approach is collaborative, recognizing that the strength of a launch lies in the shared wisdom and efforts of the pharma team and the key stakeholders within the country. We engage with Key Opinion Leaders, delve into local studies, and foster partnerships with healthcare professionals to ensure that every strategy is informed, every decision is data-driven, and every action is tailored to the unique demands of the market.

In the critical pre-launch phase, our focus intensifies on the role of medical affairs. This pivotal team, with their finger on the pulse of the medical community, ensures that our strategies are not just compliant but are also resonant with the healthcare professionals who will ultimately be the stewards of our products. They are the cartographers, mapping out the detailed terrain of local medical landscapes to ensure our product's value proposition is both compelling and relevant.

As we near the shore of a new market, the collective efforts of our team and the broader pharma community begin to coalesce. The once-daunting task of launching in a new territory becomes a coordinated symphony of expertise, insight, and strategic acumen. With us the journey to market becomes less about avoiding pitfalls and more about seizing opportunities for meaningful impact.

Reflecting on our past voyages, we see not just the challenges we've overcome but also the successes we've achieved together. Each launch is a testament to the power of collaboration, the importance of local insight, and the transformative impact of medical affairs in bridging the gap between innovation and care. As we conclude, we invite you to bring your country-specific launch plans to life with our expert guidance. We're more than just a consultancy; we're your partners in success, just a phone call or email away. 

Mira Kyttälä 28th of February 2024

Blog 1st of January 2024

Reflecting on 2023 and Welcoming 2024: A Year of Constant Change and Innovation. As we stand at the turn of a new year, it's a natural moment to pause and reflect on the journey we've traversed. The year 2023 has been a testament to the ever-evolving landscape of the pharmaceutical industry, marked by relentless change. As we say goodbye to this year and welcome 2024, let's take a moment to look back at the milestones and envision the future that lies ahead.

Embracing Change with Optimism. Change is the only constant, they say, and this year has reinforced that expression. Each twist and turn brought its challenges, but more importantly, it unveiled new opportunities. As an eternal optimist, I've witnessed how change is not just about navigating the unknown but about embracing the possibilities it brings. It's about being at the forefront, ready to adapt and innovate.

The Rise of AI in Pharma. 2023 marked a significant leap in how artificial intelligence is introduced into our workflows. AI has transitioned from a novel concept to a ubiquitous tool, enhancing our ability to create, present, and illustrate complex ideas and concepts. It has streamlined our processes, from research and development to marketing strategies, making precision and efficiency the new standards.

Breakthroughs: The CRISPR-Cas Revolution and Beyond. The growing pace of scientific breakthroughs continues. This year, we celebrated a monumental milestone with the world’s first CRISPR–Cas9 gene editing therapy receiving regulatory approvals among many other therapies that change patients´ lives. The promising new era of treatment options are accelerating and we need to ensure the new advancements will be accessible for all patients in need.

A Look Ahead: The Pace of Innovation. As we peer into 2024, one thing is clear: the pace of innovation is not slowing down. With each passing day, we're moving closer to novel groundbreaking therapies and medical solutions. My anticipation for the coming year is brimming with curiosity and excitement for what the future of healthcare holds. 

Gratitude and Collaboration: The Heart of Our Progress. Reflecting on the past year, my heart is full of gratitude for the many projects and collaborations that have come to fruition. It's been a privilege to work alongside dedicated professionals, each committed to delivering optimal care and making a tangible difference in patients' lives. These collaborations are not just about shared goals; they're about shared passion and commitment.

Together Towards a Brighter 2024. As we step into 2024, let's carry forward the spirit of collaboration and innovation. Let's continue to push boundaries, embrace new technologies, and work together to bring novel therapies available to those in need. Here's to another year of making an impact, driven by our collective efforts and unwavering commitment to bettering lives.

Mira Kyttälä 1st of January 2024

Blog 16th of November 2023

Optimizing Pharmaceutical Launches at the Country Level: Our Strategic Medical-Marketing Collaboration Approach

Many of us have witnessed the critical importance of meticulous planning in drug launches, especially for advanced medicinal products. In small countries like Finland, a market I'm deeply familiar with, the precision in timing and resource allocation can make or break a product's success. This is where our mini-launch strategy comes into play, offering a tailored solution for pharmaceutical companies.

The first step in our mini-launch process involves a comprehensive market analysis, forming the foundation of our in-country go-to-market model. We delve into the specifics of the local pharmaceutical landscape, assessing the potential patient population, current treatment outcomes, and key opinion leader (KOL) engagement. This initial phase is crucial in identifying the unique challenges and opportunities in the market.

Our approach is iterative, allowing us to refine our launch strategy continuously. By analyzing the market and potential patient impact, we ensure that resources are allocated effectively, mitigating the risk of missteps in the launch process. This iterative approach is particularly beneficial for advanced medicinal products, where market dynamics can be highly complex and rapidly evolving.

A significant aspect of our strategy is the emphasis both on medical affairs and marketing expertise. We believe that a strong medical foundation in seamless synergy with marketing expertise is essential for a successful launch. Our experts, with experience in both local and global markets, provide insights that are crucial in navigating the intricate process of bringing new treatments to the market.

While we focus on analyzing and iterating future launches through our mini-launch projects, you can confidently allocate your local resources to projects that are evaluated based on solid, data-driven insights. This approach not only optimizes resource allocation but also significantly reduces the uncertainty and risks associated with country-level launches.

Thank you for taking the time to read about our mini-launch strategy. We are excited about the potential to collaborate and bring our collective expertise to your upcoming projects. Our approach is all about crafting solutions that fit your unique needs, ensuring we successfully introduce new treatments together. If you're interested in discussing how we can optimize your future launches, we are just an email or call away. Looking forward to catching up and exploring possibilities.

Mira Kyttälä, 16th of November 2023

Blog 6th of August 2023

Welcome to the inaugural post on the MedConMiraKoo blog! I'm Mira Kyttälä, the founder of MedConMiraKoo. Since our inception in 2019, our journey has been nothing short of remarkable. We've been at the forefront of supporting launches of innovative medications in the Nordic countries and Finland, delving deep into new therapeutic areas, and forging partnerships with a diverse range of stakeholders.

Despite the whirlwind of exciting developments, our blog has been somewhat neglected. But that changes now! This blog will serve as a platform to share our experiences, insights, and observations in the field, with the intention of providing more frequent, real-time updates. So, let's break the silence and dive into our journey so far.

The Journey So Far

It's hard to believe that it's already been four years with MedConMiraKoo. We've explored new therapeutic areas, navigated the complex landscapes of the business, brought medical expertise and evidence to support access to groundbreaking medications, and initiated novel collaborations with a variety of stakeholders providing critical insights to our projects, aiming to develop therapy areas in close partnership. Despite of some challenges we've faced, we've emerged stronger, more resilient, and even better equipped to navigate our field.

Adapting to Change: The Impact of COVID-19

Less than a year into our journey, MedConMiraKoo found itself in the midst of the COVID-19 pandemic. This crisis could have spelled the end, as it did for many small businesses, but we adapted. We embraced digital platforms for collaboration and communication, ensuring our work could continue. This period underscored the importance of our work and taught us the value of resilience, adaptability, and digital transformation. We believe the whole industry is stronger than ever after the pandemic.

The Promise and Challenge of Novel Therapies

The pandemic has equipped us with more tools to tackle the challenge of bringing transformative novel therapy innovations to market. These therapies typically represent a one-time treatment with a high upfront cost, but they can be more cost-effective in the long run compared to traditional treatments, especially when considering the overall costs associated with managing a patient's worsening condition over years. The promise of these therapies is high, but there is also a lot of unknown - we cannot know nor promise how long the therapy effects will last, and effects vary from individual to individual. However, these therapies are the only hope for certain patients and can be transformative for them. We need to work together and find appropriate models for countries to enable access to these therapies for patients in lethal conditions without any other therapy options.

Looking Ahead

As we look to the future, we're filled with anticipation. We're eager to continue working on the current therapeutic areas, exploring new ones, collaborating with relevant stakeholders, and supporting access to transformative medications for patients with high unmet medical needs. We're committed to growing as a trusted partner in the pharmaceutical industry and look forward to sharing this journey with you through this blog.

Wishing you all the best for the beginning of the second half of 2023 - both happiness and success. Let's stay in touch.

Mira Kyttälä, 6th of August 2023