With the increasing use of accelerated regulatory tools like the Food and Drug Administration’s breakthrough designation, there are mounting challenges for European health technology assessors (HTAs) to make an accurate assessment of the long- term value and performance of many new therapies. Data presented in evidence is often extrapolated from outcomes six months or less, and what’s good enough for regulatory approval often doesn’t meet the needs for comparative effectiveness, value, and outcomes measures for HTAs. Vital Transformation’s recent BMJ publication demonstrates a novel, practical approach to harnessing electronic health records and RWE to help solve this problem. However, access to robust deidentified patient records in Europe is key.

According to research from the US Health and Human Services, US tax payers pay 1.8 times the international price for medicines and are ‘subsidizing’ global healthcare. In order to combat this imbalance, the Trump administration is proposing the “International Pricing Index” (IPI) payment model to reduce reimbursement levels for the Centers for Medicare & Medicaid Services (CMS). Following Vital Transformation's research on investment success factors for biotech companies released at the European Health Forum Gastein, Vital Transformation presented original research modelling the potential impact both short and long term for patients, payers, and industry for such proposals, and show how the IPI can impact access and innovation broadly. As well, the panellists placed the IPI into the context of other models currently being investigated internationally such as value based payments, managed entry agreements, and adaptive pathways.

Publish date: February 28, 2019 By Sharon Worcester The high cost of chimeric antigen receptor (CAR) T-cell therapy largely limits its use to the sickest patients and prohibits experimentation in “less-diseased” populations, outcomes data suggest.

By Duane Schulthess - 26 December 2018

It’s odd that a city like Brussels managed to carve itself out a niche as one of the major political centres of the world. The medieval site of a notoriously typhus infested bog, one of the main streets in downtown, ‘Rue du Marais’, literally means, “Street of the Swamp” and the name of the city itself comes from the old Flemish word Broekzele, which roughly translates to ‘settlement in the swamp.’ So, when one talks politically of draining the swamp, at least in Brussels, it carries a literal interpretation that is often distant from the current political climate in DC.

What goes without question is that both Brussels and DC are currently heaving under waves of increasing populist threats to drain political swamps. With the Brexit referendum followed by the election of President Trump, we also now have the Yellow Vest protests in France coupled with a letter accusing President Macron of treason signed by a dozen French generals and a former Minister of Defense. There is an air of anger against political institutions on both sides of the Atlantic, and increasingly, the heated rhetoric is being pointed at the price of medicines.

Vital Transformation released its newest research at the European Health Forum Gastein on October 4th, 2018. Our project analysed 116 EU biotech start-ups from the period of 2001 – 2007 through September 21, 2018 from the UK, Netherlands, Belgium, and Spain. We focused on companies developing medicinal products for human consumption. In this work, we compared the impact of IPOs (stock market listings), private investors, and direct EU funding on their success or failure.

24th June 2018, Boston, USA

Duane Schulthess, Managing Director of Vital Transformation, and Luca Pani, former Director General of the Italian Medicine's Agency and currently a Faculty Member at the University of Miami, will run a 3 hour and 15 minute Short Course outlining how best to harness RWE to better understand the price, value, and impact of next generation therapies.

30th December, 2017

The objective of this paper is to identify the extent to which real world data (RWD) is being utilized, or could be utilized, at scale in drug development. Through screening peer-reviewed literature, we have cited specific examples where RWD can be used for biomarker discovery or validation, gaining a new understanding of a disease or disease associations, discovering new markers for patient stratification and targeted therapies, new markers for identifying persons with a disease, and pharmacovigilance.

21st October, 2015

After nearly a decade of discussion, analysis, and development, the Medicines Adaptive Pathways to Patients (MAPPs) initiative is beginning to see acceptance from regulators, industry, patients, and payers, with the first live pilot project initiated under the guidance of the European Medicines Agency in 2014.