---
title: Volv Global Blog | Regulation
description: Regulation | Blog on the topic of rare and orphan diseases and other related topics
---

# Volv Global Blog

Select Category Data Science Innovation Patient Finding Predictive models Rare Diseases inTrigue Real World Evidence patient experience inClude AI democratising the data Regulation Case Study Cell & Gene Therapy Machine Learning RWE

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Posts about:

## Regulation

![Hands in the air](https://blog.volv.global/hubfs/Hands%20in%20the%20air%20-%20ratio%202%20to%201%20-%20Stock-157312044.jpg)

[Real World Evidence,](https://blog.volv.global/tag/real-world-evidence) [Rare Diseases,](https://blog.volv.global/tag/rare-diseases) [Innovation,](https://blog.volv.global/tag/innovation) [Regulation,](https://blog.volv.global/tag/regulation) [Data Science,](https://blog.volv.global/tag/data-science) [Predictive models,](https://blog.volv.global/tag/predictive-models) [Patient Finding,](https://blog.volv.global/tag/patient-finding) [inClude,](https://blog.volv.global/tag/include) [Machine Learning](https://blog.volv.global/tag/machine-learning)

### [The Path to Rare Disease Clinical Trial Innovation](https://blog.volv.global/the-path-to-rare-disease-clinical-trial-innovation)

Credit Photo by RichLegg on iStock

### Executive Summary

For decades, the pharmaceutical industry has faced the same recurring problems with clinical development: the struggle to fully recruit and retain enough patients, meet target timelines, and have trials conclude on time.

Certainly, the industry does overestimate its ability to recruit, but a bigger issue is that study designs and protocol development seemingly fail to truly reflect patients’ lives, or account for the reality in the clinic. In fact, data shows the probability of success for any clinical development effort is 6.2% for orphan drug trials, compared with 13.8% overall, which translates to a 93.8% failure rate for orphan drug development efforts.

Download the Blog here:  
[![Download PDF](https://hubspot-no-cache-eu1-prod.s3.amazonaws.com/cta/default/9428630/f73f8cc9-0352-4c0d-9b97-9d86bfcbc6c4.png)](https://hubspot-cta-redirect-eu1-prod.s3.amazonaws.com/cta/redirect/9428630/f73f8cc9-0352-4c0d-9b97-9d86bfcbc6c4)

[Volv Global SA and WODC EU contributors](https://blog.volv.global/author/volv-global-sa-and-wodc-eu-contributors) 

[Read More](https://blog.volv.global/the-path-to-rare-disease-clinical-trial-innovation)

![Picture of the World Orphan Drug Congress hosted a webinar titled, “How can AI impact industry?”](https://blog.volv.global/hubfs/RWE.png)

[Real World Evidence,](https://blog.volv.global/tag/real-world-evidence) [RWE,](https://blog.volv.global/tag/rwe) [Rare Diseases,](https://blog.volv.global/tag/rare-diseases) [Innovation,](https://blog.volv.global/tag/innovation) [Regulation](https://blog.volv.global/tag/regulation)

### [Driving patient-centricity through real-world evidence](https://blog.volv.global/real-world-evidence-round-table-session)

#### Executive Summary

> To acknowledge Rare Disease Day and the struggles facing patients with rare diseases, the [World Orphan Drug Congress](https://www.terrapinn.com/conference/world-orphan-drug-congress-usa/index.stm) hosted a webinar titled, “How can AI impact industry?” The session, which explores the role of real-world evidence (RWE) to improve orphan drug development and access, brought together leaders in their field with deep knowledge of rare diseases and the importance of RWE in helping to identify the right solutions for the right patient.
> 
> During the discussion, several important and often overlooked themes were brought to the fore. The purpose of this paper is to explore these in greater depth and share the unique insights from the panel. These include the integral role of the patient in owning, managing, and deciding when, how and where to share their data. Certainly, a patient-focused approach that safeguards the individual’s privacy and ensures consent, is paramount if researchers and drug developers are to make full use of RWE to find therapeutic approaches and cures for rare diseases. In addition, the industry will need to address data sharing issues and legislative barriers and ensure they fully engage the regulators to bring therapies to patients in need. The paper provides a thoughtful and balanced discussion of these topics and aims to seed further insights as drug developers, clinical research experts, regulators, artificial intelligence specialists and, crucially, patients and their carers, seek to leverage tools such as RWE to help the millions of rare disease patients in need worldwide.
> 
> [![Download PDF](https://hubspot-no-cache-eu1-prod.s3.amazonaws.com/cta/default/9428630/cececdf7-85cc-4adb-8fdd-4d40a49128da.png)](https://hubspot-cta-redirect-eu1-prod.s3.amazonaws.com/cta/redirect/9428630/cececdf7-85cc-4adb-8fdd-4d40a49128da)
> 
>  

[Christopher M de M Rudolf](https://blog.volv.global/author/christopher-rudolf) 

[Read More](https://blog.volv.global/real-world-evidence-round-table-session)