Empowering Rare Disease Innovation in Europe
Explore our curated resources to gain the knowledge you need to successfully launch and commercialize your rare disease therapy.

The Unseen Force

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…

The Rare Disease Market in APAC: Challenges and Opportunities

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…

US Biotech in Europe

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…

Hurdles to Harmonization

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…

The Global Ambition

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…

EU As A Launch Pad

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…

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…

Hope for Rare Diseases: Drug Repurposing for Better Patient Care in Europe

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…

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…

ENSURING PATIENT ACCESS: The Crucial Role of Market Access for Drugs

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…

Navigating Gene Therapy Commercialization in Europe: A Q&A with Novartis’ Joshi Venugopal

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…

RARE DISEASE CLINICAL TRIALS: A Collaborative Approach to Design and Execution

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…

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

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…

Expanding into Europe

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…

The Ultimate Guide to EMA Approval for Rare Disease Therapies: Unlocking European Access

The Ultimate Guide to EMA Approval for Rare Disease Therapies: Unlocking European Access

Your Ultimate Parter for EMA Approval of Rare Disease Therapies Unlocking European Access Developing a therapy for a rare disease is a noble endeavour, but the path to market is complex, especially…

The Geopolitics of Hope

The Geopolitics of Hope

The Geopolitics of Hope A Flourishing Market, Yet Millions in Europe Lack Access to Rare Disease Treatments Did you know that over 30 million people in Europe live with a rare disease,…