PHARMACOVIGILANCE AND RISK MANAGEMENT CONSIDERATION FOR RARE DISEASE GENE THERAPIES

July 17, 2019

The development of a pharmacovigilance and risk management system to support a gene therapy for a rare genetic disease requires consideration of the specific challenges and nuances that novel therapies present throughout the lifetime of the product.

Spotlight

Drugs & Diagnostics for Tropical Diseases

Our mission is to develop treatments and diagnostics for neglected tropical diseases that affect the most impoverished social groups. An urgent gap in treatment technology has left millions in sub-Saharan Africa at risk of debilitating and deadly parasitic diseases--but we're developing a novel diagnostic device that will end this. There is an urgent need for a simple, rapid, and quantitative diagnostic test to detect loiasis, an infection of the parasitic roundworm Loa loa (L. loa). Even though treatments exist, there are no commercial diagnostic tests available to identify the disease and monitor treatment efficacy.

OTHER WHITEPAPERS
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A BLOCKCHAIN TO ENSURE COUNTERFEIT FREE PHARMACEUTICAL SUPPLY CHAIN

whitePaper | February 5, 2022

Essential medicine access is one of the main objectives of healthcare systems in and pharmaceutical supply chains ensure that the right amount of medicine, with acceptable quality, will reach the customers in need

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Pharma 2020: Marketing the future. Which path will you take?

whitePaper | August 8, 2021

If Pharma is to create a new marketing and sales model that is fit for 2020, it will have to begin by analysing its own value chain to identify opportunities for working more closely with healthcare payers and providers. It will, for example, have to collaborate much more closely with payers (be they governments, health insurers, employers or patients) to ensure that it develops medicines which have real social and economic value.

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Paving the way for pharma R&D to use FAIR data

whitePaper | December 16, 2019

The use of FAIR (Findable, Accessible, Interoperable and Reusable) data in drug discovery and development could be transformational. But the pharma industry has been slow to embrace FAIR data — partially because of difficulties in establishing the necessary infrastructure and because of internal culture.

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How Cellular is Enabling the Digital Supply Chain

whitePaper | January 12, 2020

Supply chains grow more and more complex every day. In response, manufacturers and shippers have been eagerly exploring digital tools that provide actionable insight into the location and condition of in-transit goods. Although there are many technology options when it comes to building a digital supply chain, one type of solution stands out as the most reliable and cost-effective: cellular.

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Access to medicinal products

whitePaper | January 3, 2022

There are many factors that can prevent a patient from being able to obtain the medicine they need, ranging from selective marketing decisions by companies to products being too expensive or pharmacy stock-outs

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WILL PHARMA COMMIT TO DELIVERING AFFORDABLE THERAPEUTICS AGAINST COVID-19?

whitePaper | April 8, 2020

EPHA is a change agent – Europe’s leading NGO alliance advocating for better health. We are a dynamic member-led organisation, made up of public health civil society, patient groups, health professionals, and disease groups working together to improve health and strengthen the voice of public health in Europe.

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Spotlight

Drugs & Diagnostics for Tropical Diseases

Our mission is to develop treatments and diagnostics for neglected tropical diseases that affect the most impoverished social groups. An urgent gap in treatment technology has left millions in sub-Saharan Africa at risk of debilitating and deadly parasitic diseases--but we're developing a novel diagnostic device that will end this. There is an urgent need for a simple, rapid, and quantitative diagnostic test to detect loiasis, an infection of the parasitic roundworm Loa loa (L. loa). Even though treatments exist, there are no commercial diagnostic tests available to identify the disease and monitor treatment efficacy.

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