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 […]
ENSURING PATIENT ACCESS: The Crucial Role of Market Access for Drugs
Ensuring Patient Access The Crucial Role of Market Access for Drugs Navigating the journey from clinical trials to market access requires a deep understanding of the European landscape. Partner Rare’s team of experts have extensive experience in designing and conducting clinical trials tailored to meet EU regulatory requirements, while simultaneously conducting market access research to […]
Navigating Gene Therapy Commercialization in Europe: A Q&A with Novartis’ Joshi Venugopal
Navigating Gene Therapy Commercialization in Europe A Q&A with Joshi Venugopal, General Manager of Gene Therapies and Rare Diseases, Europe Opinions expressed here are personal and not that of Novartis* Gene therapies hold immense promise for revolutionizing the treatment of rare diseases, offering potential cures where previously only symptom management was possible. However, the path […]
RARE DISEASE CLINICAL TRIALS: A Collaborative Approach to Design and Execution
Rare Disease Clinical Trials A Collaborative Approach to Design and Execution Patients who suffer from rare diseases need access to effective and safe medications. Given the low incidence of the diseases, the collaboration between regulatory authorities, producers, insurances, and market access teams is essential for pharmaceutical companies seeking to bring new medications to patients and […]
Demystifying the Maze: Centralized vs. National Procedures for Rare Disease Drug Registration in the EU
Demystifying The Maze Centralized vs. National Procedures for Rare Disease Drug Registration in the EU Introduction The European market presents a compelling opportunity for US pharma and biotech companies developing life-changing therapies for rare diseases. Engaged patient communities and sophisticated healthcare systems create a fertile ground for innovation. However, navigating the intricacies of the European […]
Expanding into Europe
Cracking the EMA Code Expanding into Europe Expanding a Rare Disease drug’s marketing authorization into Europe can potentially increase a company’s net worth, but it’s not a guaranteed outcome. Here’s a breakdown of the factors involved: Potential Benefits Increased Market Size Europe represents a significant market with a large population. Expanding authorization allows the company […]
