Unlocking innovation and access for rare disease patients in Europe

Feb 22, 2021 - Feb 22, 2021, Online

To mark the opening of Rare Disease week, you’re invited to join this high-level policy debate, that will address the future of European collaboration on rare diseases, to reimagine and transform rare disease policy through access, awareness, and innovation.

Bringing innovation to rare disease patients in Europe

Johanna Friedl-Naderer, President, Europe, Canada & Partner Markets, Biogen on how collaboration between governments and industry can lead to better patient outcomes.

Why is Biogen focusing on rare diseases? 

About 6,000 different rare diseases have been identified to date and these are estimated to affect 30 million people across Europe. It is essential that we collaborate across the medical community, industry, and governments to develop new therapies, explore new technologies and ensure equitable access to treatment. We must innovate across every aspect of treatment to transform the lives of people in these underserved communities. 

At Biogen, we have shown that pioneering in neuroscience can change the course of treatment for certain rare diseases. We have transformed the treatment of Spinal Muscular Atrophy (SMA), a rare neurodegenerative disease which is one of the leading genetic causes of infant mortality in Europe. Since 2016, we have helped more than 10,000 SMA patients live longer, fuller and better lives. Today, infants born with SMA have a chance to walk, run, play and go to school. 

There is still so much more work to be done and our commitment drives us to continue researching, investing and innovating to find new ways to improve the lives of those living with SMA and other rare diseases. Few companies are pursuing disease modifying treatments in as many difficult areas as Biogen is. We are currently developing a range of potential therapies for rare diseases, including Choroideremia, a genetic retinal disorder and major cause of blindness, and Amyotrophic Lateral Sclerosis (ALS), a rare terminal neurodegenerative disease. Innovation is not limited to the lab either: we are taking a holistic approach to the challenges faced by the Rare Disease Community, exploring the potential for new digital tools and neurotechnology to significantly improve treatment and patient outcomes.  

We are also committed to address the barriers that prevent equitable access to treatment across Europe. This has never been more important than it is today. As the global pandemic necessarily absorbs the focus of healthcare systems, we must work even harder to ensure that people living with rare diseases are not forgotten.  


How has the COVID-19 pandemic impacted your work and the lives of rare disease patients? 

The pandemic has put the world’s healthcare infrastructure under extreme pressure and, for people living with rare diseases, this has intensified many of the challenges they already face daily. A survey by EURORDIS, Rare Disease Europe, indicates that access to diagnosis, care and treatment has been significantly disrupted by the pandemic. 

Across the medical community, we responded with speed and agility to safeguard medical supply chains and access to treatment. As a result, Biogen has maintained uninterrupted drug supply. Digital innovations have been especially important in supporting health care professionals on the front-line of treatment. 

The pressures created by the pandemic are also accelerating some positive transformations, which could deliver long-term benefits for patient care. It is opening doors to e-health, remote monitoring, and other digital solutions that have the potential to improve outcomes. We need a uniform regulatory framework in Europe to turn these solutions into standard medical services, accessible to every European patient.  It is essential that governments, health care professionals and industry work together on building the right infrastructure so that patients and healthcare systems benefit from digital innovations. 


What is needed to accelerate innovation for rare diseases in Europe, especially in underserved areas?

The current pace of innovation in advanced therapies is incredibly promising. Progress is, however, still too slow for many patients living with untreated rare diseases. Advancements often require decades of committed scientific research with high failure rates for new therapies. This means that responsible investment and supportive regulation continue to be essential. The European ‘orphan drug’ legislation, put in place 20 years ago to support the development of treatments for rare diseases, has been critical in accelerating new treatments and improving the lives of hundreds of thousands of patients. At a time when too many rare diseases still lack a treatment option, this legislation is more important than ever, and we need to protect it. An ambitious and stable incentive framework remains essential to drive innovation where it is needed the most and continue this successful path of bringing treatments to patients in dire need.  


 ‘Access’ and ‘innovation’ are often talked about as trade-offs: how do we continue to foster innovation while expanding access at the same time? 

At Biogen, we share a common goal with all partners in healthcare to ensure that patients have rapid, sustainable access to the latest effective and lifesaving medicines. We are working holistically with governments and healthcare systems to make medicines accessible and healthcare sustainable, whilst also investing in future medical innovations. 

Biosimilars are one important part of the solution to expand both access and innovation. In 2020, Biogen’s biosimilars opened access to treatment for 240,000 patients and generated €2.4billion in savings across healthcare systems in Europe. These savings create headroom to invest in innovative new treatments to address unmet needs, including in rare diseases. 

We also need to understand the multiple and complex factors that can lead to delays in access. The implementation of the EU Pharmaceutical Strategy, recently presented by the European Commission, represents a unique opportunity for stakeholders to work together to pursue this shared aspiration. As an industry, we have proposed the creation of a High-Level Forum on Better Access to Health Innovation to identify challenges and concrete actions. Two immediate actions to accelerate access across Europe include the implementation of novel pricing and payment models and the approval of the EU health technology assessment regulation to help minimize national duplication in clinical assessments.  

Finally, we must also develop and embrace new technologies to accelerate innovation and broaden access. Increasingly, technology advancements will work in synergy with scientific advancements to transform healthcare. Collaboration and dialogue between governments and industry will be critical in harnessing technology, from Artificial Intelligence to wearable devices, to further accelerate healthcare innovations safely and ethically. 


What opportunities do you see in digital health for patients with RD? What is Biogen doing in this space? 

We have an important opportunity to deepen our knowledge around disease progression and personalized treatment by combining insights from digital health, clinical research and healthcare systems. This is especially relevant for rare diseases, where information is generally a lot scarcer because of smaller patient populations. More data enables better decision-making and better patient outcomes. 

At Biogen, we are focused on maximizing the opportunities offered by digital technologies. For example, we are currently exploring a personalized digital healthcare solution that could enable patients to remotely self-assess and monitor their neurological functions using their smartphone. This is currently in a validation study with people living with Multiple Sclerosis and a further study is planned this year to support its validation for rare diseases such as SMA and ALS. 

We also recently announced a significant new partnership with Apple to develop digital biomarkers that could help monitor cognitive performance in high-risk populations over time and potentially identify early signs of mild cognitive impairment, using Apple Watch and iPhone as a monitoring tool. I believe that such tools will become critical in the future in predicting and preventing disease progression by accelerating early diagnosis and treatment of neurodegenerative diseases. 

The importance of the policy and regulatory environment in this area cannot be underestimated. The development of an enabling and agile European framework is essential to encourage collaboration and establish clarity and trust in the use of data for healthcare research and care. We must create a sustainable health data ecosystem to unlock the value of the data generated within healthcare systems and through patients’ daily experiences. This will help foster meaningful connections between disease registries and maximize the use of decentralized virtual trials and mobile technologies in clinical trials and in the real world.  

The convergence of digital technology with scientific and medical advances means that we are at a very important moment in time for healthcare. Collaboration across the entire ecosystem will be critical if we are to translate these opportunities into lifesaving and lifechanging outcomes for patients, especially in underserved areas such as rare diseases. 


Unlocking innovation and access for rare disease patients in Europe

Feb 22, 2021 - Feb 22, 2021, Online

To mark the opening of Rare Disease week, you’re invited to join this high-level policy debate, that will address the future of European collaboration on rare diseases, to reimagine and transform rare disease policy through access, awareness, and innovation.