Publications/White Papers

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P4A experts regularly share their unique perspectives that inform and challenge the norm through articles and white papers.

FDA approves gene therapy for rare form of vision loss

The U.S. Food and Drug Administration (FDA) has approved the use of Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl) for the treatment of a rare inherited form of vision loss and blindness. This marks the first approval by the FDA of a directly administered gene therapy for the treatment of a disease caused by mutations […]

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FDA issues new draft guidance for Orphan Drug development

The U.S. Food and Drug Administration (FDA) published its recommendation for a new approach to drug development for rare pediatric diseases. The new approach supports the FDA’s commitment to increase the efficiency and speed of developing drugs, as well as supporting the sponsors of the research. The draft guidance, ‘Pediatric Rare Diseases: Collaborative Approach for […]

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The EMA has updated its practical guidance for pharmaceutical companies for procedures related to Brexit

From 30th March 2019, when the United Kingdom’s (U.K.) is expected to withdraw from the European Union (EU), all EU law will no longer apply to the U.K. The European Medicines Agency (EMA) and the European Commission have therefore published additional practical guidance to help pharmaceutical companies legally prepare for this transition. This guidance will […]

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Amsterdam will be the new host of the EMA

Amsterdam has been selected as the new post-Brexit home for the European Medicines Agency (EMA). It was chosen in a draw of lots between it and Milan, after three rounds of voting had failed to pick a favourite. The EMA has been located in London since its formation in 1995, where it employs around 900 […]

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EMA and EUnetHTA publish a 3 year collaboration plan to improve efficiency and quality

The European Medicines Agency (EMA) and the European Network for Health Technology Assessment (EUnetHTA) have finalised “a joint work plan outlining key areas of collaboration for the next three years”. The main areas of collaboration are information exchange at market entry, data generation post approval and early dialogue. These areas are expected to also provide […]

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European P&MA Consolidation: A Paradigm Shift and Implications for Pharma

Earlier this month, Italy’s Health Minister, Beatrice Lorenzin, announced that two additional countries- Slovenia and Croatia- had signed up to the Valletta Declaration. Of the 28 EU member states, over a third (10 countries*) are now part of the initiative which aims to strengthen their bargaining power over pharmaceutical companies through collective price negotiations and […]

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Hot topics in pharma and orphan drugs

I attended this year’s pharma pricing and market access conference in London with attendees across the pharmaceutical industry, payer and HTA bodies and patient representatives. While following the talks, panel discussions and roundtable debates, a few overarching key topics crystallised: –          Germany – AMNOG changes AMNOG, the German pharmaceutical law is regularly adapted to changes […]

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AMNOG and the German Reforms: What Will Change?

Germany is typically prioritized by the pharmaceutical industry: it remains a market with relatively high drug prices, offers extensive commercial opportunities and has an important position as an international reference country to 17 markets. The implementation of AMNOG in 2011 sought to reduce drug prices and relieve the healthcare budget, yet financial pressure remains, particularly […]

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