Protected: Beyond the Blueprint: Rethinking Due Diligence in Rare Disease Investments
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The Unseen Force
The Unseen Force By Aaron BlockerSenior Advisor: Advocacy / Market Access Empowering Market Access for Rare Disease Therapies Through Patient Advocacy Developing a life-saving therapy for a rare disease is a triumph of scientific innovation and perseverance. However, translating this groundbreaking research into real-world patient access is a complex undertaking, particularly in the multifaceted global […]
The Rare Disease Market in APAC: Challenges and Opportunities
The Rare Disease Market in APAC By Chad JiRare Disease Expert – South Korea The rare disease sector can broadly be divided into oncology and ultra-rare disease areas. Today, we will focus on the ultra-rare disease segment (referred to as “rare diseases” hereafter). Most of the insights and content discussed are based on my personal […]
US Biotech in Europe
US Biotech in Europe How HTA Bodies Can Boost Your US Payer Negotiations The rare disease landscape presents unique challenges and opportunities for pharmaceutical and biotech companies. While the US market offers a large patient population and potential for high drug prices, navigating the complex regulatory and payer landscape can be daunting. For US-based companies […]
Hurdles to Harmonization
Don’t Sleep On The EU! Hurdles to Harmonization A Strategic Guide for Biotech and Pharma Facing FDA Setbacks in Rare Disease Therapy Development Experiencing a setback with the FDA in your rare disease therapy development can feel like a significant hurdle. However, it’s important to remember that the journey doesn’t end there. The European Medicines […]
The Global Ambition
Don’t Sleep On The EU! The Global Ambition The rare disease landscape is evolving rapidly, with groundbreaking scientific advancements and novel therapeutic approaches emerging at an unprecedented pace. For biotech and pharmaceutical companies venturing into this dynamic space, navigating the complex regulatory pathways for approval in both the US and Europe is paramount. A well-orchestrated […]
EU As A Launch Pad
EU As A Launch Pad Think Global, Start European: A Strategic Launchpad for Rare Disease Therapies The global rare disease landscape is evolving rapidly, with increasing investment and a growing pipeline of innovative therapies. Yet, navigating the complex regulatory and market access pathways remains a significant challenge for biotech and pharmaceutical companies. In this context, […]
Bringing Cell Therapies to Rare Disease Patients in Europe
Bringing Cell Therapies to Rare Disease Patients in Europe Rare diseases are defined by sheer numbers of new patients per population. This definition is not helpful for the affected patient and his/her family. These conditions often lack effective treatment options due to their low prevalence. In addition, there are challenges in developing traditional pharmaceuticals for […]
Hope for Rare Diseases: Drug Repurposing for Better Patient Care in Europe
Hope for Rare Diseases Drug Repurposing for Better Patient Care in Europe For patients with rare genetic diseases, the struggle extends far beyond the challenges of the disease itself. Multiple co-morbidities often accompany these conditions leading to complex polypharmacy treatment. The reliance on multiple medications exposes patients to a higher risk of adverse effects from […]
The Global Landscape of Rare Disease Treatments
The Global Landscape of Rare Disease Treatments Legal and Regulatory Considerations for Pharma and Biotech Companies Rare diseases, affecting a small percentage of the population, often face a lack of research funding and treatment options. However, the global pharmaceutical and biotechnology industry is increasingly focusing on developing therapies for these conditions. To incentivize this crucial […]
