Bringing Cell Therapies to Rare Disease Patients in Europe

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

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

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 […]

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

PTJ Expanding into Europe A1

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 […]