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.


Future-proofing 2019 plans: P4A’s 6 Orphan Drug, Cell & Gene Therapy trends to watch out for

2019 is expected to a strong year for the orphan drug industry with an expected 10% surge in sales growth. Contributing to this growth will be new treatment launches particularly from cell and gene therapies. As these novel therapies are geared towards commercialization, the industry is witnessing a change in approach by manufacturers, regulators and […]

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Rare Diseases: A New Purpose

How drug repurposing could help drive orphan drug access if pharma can accept the risks By Nader Murad, Senior Analyst Email: The US National Institutes of Health describes drug repurposing as “studying drugs that are already approved to treat one disease or condition to see if they are safe and effective for treating other […]

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Partners4Access Briefing: November 2018 update on the European Commission regulation proposal on health technology assessment

By Christina Poschen, Consultant Email: Background Since 2004, the European Union’s centralised drug approval is in place, allowing manufacturers to submit just one application to gain marketing authorisation in all European Union Member States. Although there were voluntary agreements and working groups to further harmonize health technology assessment, so far, there are 28 different systems […]

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Innovative Access Agreements: A potential payer solution for Cell & Gene Therapies

A US and EU perspective By Daniele Severi Bruni & Sophie Schmitz What’s the idea? Payer concerns over increasing cost of therapies and uncertainty of clinical data has led to a growing interest in market access tools. Experts Daniele Severi Bruni partner at MKO and Sophie Schmitz from Partners4Access discuss this potential payer solution for […]

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CAR-T Therapies, the future for cancer patients? A market access perspective

Chimeric Antigen Receptor T-cell (CAR-T) therapies, which use a patient’s own immune cells to fight cancer, are one of the most exciting breakthroughs in cancer treatment for decades. Research into this technology has been ongoing since the early 1990s, but only in the last few years have these treatments been successfully used to treat cancer […]

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What does the new FDA accelerated regulatory pathway mean for gene therapies?

U.S. Food and Drug Administration (FDA) commissioner Scott Gottlieb has announced that the drug agency is developing a system by which innovative gene therapies could be sped through the regulatory process. In a speech to board members of the Alliance for Regenerative Medicine, Dr. Gottlieb spoke about the challenges associated with reviewing cell and gene […]

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Biopharma 2018 Success Kit: 11 months to go, 6 rare disease trends to watch!

For the rapidly developing orphan drug market, change is a constant. From new technologies to discussions over pricing, the estimated $137 billion market will witness new challenges for biopharma companies in the rare disease space. In a bid to unravel these barriers and with 11 months more to go in 2018, the team at Partners4Access […]

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ICER finds Roche’s Hemlibra cost-effective despite high price tag

Roche’s new haemophilia treatment, Hemlibra, is has a high estimated price tag of $482,000 for the first year of treatment and $448,000 for every subsequent year. Despite this, an analysis by the U.S. Institute for Clinical and Economic Review (ICER) has revealed that the treatment is still cost effective. The report stated that the Hemlibra […]

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Sanofi to buy Bioverativ in $11.6 billion deal

French pharmaceutical giant Sanofi have agreed to acquire Bioverativ, a U.S. hemophilia specialist for $11.6 billion. This deal is the biggest for Sanofi in seven years, with the aim of achieving a stronger position in the rare disease space. After initially falling due to investor speculation that Bioverativ was overvalued, shares in Sanofi have since […]

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UK regulator attempts to ease fears of impending access abyss

The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) announced that there would be no sudden changes to the UK regulatory framework in the event of a no-deal following Brexit negotiations. It went on to confirm that it would provide “adequate notice to industry and ensure that companies had sufficient time to implement any changed […]

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