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.


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. These tools have many names and definitions, often referred to as innovative (IAA) or alternative access agreements (AAA). […]

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NICE balks at Spinraza price, rejects reimbursement in its draft guidance

By Christina Poschen, Consultant Email: Biogen’s Spinraza, a treatment for the rare genetic disease spinal muscular atrophy (SMA) is currently making headlines for its high price and promising outcomes.  Various European countries including Spain, Italy as well as The Netherlands and Belgium just made the product available to their patients or confirmed they will […]

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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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Seven months to Brexit, does the biopharma industry have the tools to be ready?

Everything you need to know about the latest Brexit White Paper By Joanna Fernandes, Analyst Email: As the countdown to Brexit draws near, a flurry of activities has emerged from the UK government, the EU and the pharma/biotech industry. But central to the conversation is the recent white paper released by the UK government […]

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Slow Brexit proceedings frustrates biotech industry

By Aparna Krishnan, Corporate Affairs Lead As the UK Prime Minister’s office is preparing for the cabinet meeting at Chequers this Friday, British businesses including the biotech industry are none the wiser on what lays ahead after March 29, 2019 when the UK exits the European Union. The current discussion is focussed on the Brexit […]

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Faster route to access in Scotland for rare disease patients requiring ultra-orphan drugs

By Alison Kneen, Vice President International Operations Fantastic news for Scottish patients who suffer from very rare diseases. As of October 1, 2018, patients will receive faster access to new treatments as a result of changes being made to the Scottish Medicine Consortium (SMC), the health technology assessment (HTA) body pathway which is used to evaluate […]

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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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Cancer diagnosis – a step forward for England

New rapid diagnostic and assessment centres are being piloted in ten areas across England in a focused approach to identify cancer early.  These new centres are part of the NHS plan to meet the new faster diagnosis standard, where patients with suspected cancer should receive a diagnosis or given the all clear within 28-days. The […]

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PRIME – two-year assessment – was it good for biopharma?

The PRIority Medicines Scheme (PRIME) of the European Medicines Agency (EMA) was started two years ago to support and optimise medicine development. The scheme provides early and enhanced scientific and regulatory support to medicines that have the potential to significantly address patients’ unmet medical needs. The EMA has published their two-year review and not surprisingly more than […]

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Irish patients continue to be denied vital new drugs

Todays’ article in the Irish Times[1] highlights again that patients in Ireland continue to be denied nine drugs which are already approved for use by the National Centre of Pharmacoeconomics (NCPE). They have been waiting on average for 2 years for the Health Service Executive (HSE) to approve them for reimbursement. The drugs concerned range […]

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