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.


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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CDF to save NHS £140m over five years

The Cancer Drugs Fund (CDF) is expected to save the U.K. National Health Service (NHS) a minimum of £140 million over the next five years. Medicines that are recommended to the CDF by NICE (National Institute of Health and Care Excellence) have their prices negotiated lower than the manufacturer’s initial listed price. Over 15,000 patients […]

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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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Generation Bio pioneers revolutionary gene therapy delivery system

The biotech venture capital firm Atlas Ventures has announced the creation of Generation Bio, a company that has developed a system by which the dosage of a gene therapy can be varied to maximise the potential benefit. The company has started on the back of a $25 million Series A funding round from Atlas. The […]

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Spark Therapeutics Announces $850 000 price tag of gene therapy treatment Luxturna

Spark Therapeutics has announced its recently approved Luxturna (voretigene neparvovec-rzyl), a gene therapy treatment for biallelic RPE65 mutation-associated retinal dystrophy will cost $425 000 per eye, or $850 000 in total, in the U.S. This is below the $1 million price tag that was originally speculated. Spark Therapeutics is also working with Harvard Pilgrim, a […]

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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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