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.


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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Cancer treatments: A brief history

February 4 was World Cancer Day and here at Partners4Access, we are having a look back at how cancer treatments have evolved through the years and where they are likely to go in the future. First came surgery In the 18th century, surgery was hailed as the first effective cancer treatment. The discovery of anesthesia […]

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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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Though there is some way to go, NICE is moving in the right direction on HTAs

  From 1 March 2000 to 31 December 2017, NICE (National Institute for Health and Care Excellence) published over 800 healthcare technology recommendations. In total, 81% of those decisions were recommended, optimized or recommended for use in the Cancer Drugs Fund (CDF). Why then is NICE considered such a tough cookie when it comes to […]

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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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Orphan Drug Pricing: Is Eurordis call to reduce prices by 3-5 times the answer to unequitable access?

Partners4Access comments on the new Eurordis Position Paper Earlier this month Eurordis published its position paper on orphan drug access. Whilst the report is commendable in pushing to drive equitable access for patients; their call to reduce orphan drug prices by 3 to 5 times by 2025 is no guarantee of full patient access. Affordability […]

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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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