Awards provide real world evidence that UK pharma remains at its innovative best
London, 21st September, 2016. The first new treatment for heart failure in 15 years and Europe’s first approved stem cell therapy have scooped top prizes at the 2016 UK Prix Galien Awards.
Novartis’ Entresto and Chiesi’s Holoclar won two of the three major accolades awarded on a historic evening at London’s House of Commons.
The 2016 Awards also saw the UK become the first country in the world to issue a Prix Galien medal for Real World Evidence – won by Dificlir from Astellas.
Announcing the results, Professor Sir Michael Rawlins, Chair of the UK Prix Galien judges, said: “Every year we wonder if the new entrants will surpass those seen previously in both quality and number. We were not disappointed in 2016. We received a record number of entrants, with 16 of these at a high enough standard to be shortlisted. Congratulations must go not only to the winners, but to all entrants for putting up such a strong showing.”
Innovative Product Award
The Innovative Product Award was won by the breakthrough heart failure treatment, Entresto. The win gives Novartis its fourth UK Prix Galien medal since 2002 and its second in succession, having won the last Innovative Product Award with Bexsero in 2014.
“Entresto, our only small molecule entry, offers a unique approach to the treatment of patients with heart failure,” said Professor Sir Michael. “The PARADIGM-HF study demonstrated key benefits for Entresto over and above current standards of care for patients with reduced ejection fraction including being cost effective when compared to comparators. The judging panel recognised that this is the first new treatment for heart failure in 15 years with the potential to provide a better outcome to around 300,000 people in the UK.”
Commendations were awarded to Entyvio (Takeda) and Opdivo (Bristol Myers Squibb).
Orphan Product Award
The Orphan Product Award, first introduced as a UK Prix Galien category in 2006, recognises innovation in conditions that affect very small numbers of patients. “Many of these conditions are either untreatable or treated inadequately,” said Professor Sir Michael. “Pharmaceutical companies face a number of hurdles in developing drugs for these conditions, particularly the likely size of the market and the small number of patients available for clinical trials.”
The 2016 Orphan Product Award was won by Holoclar, which in 2015 became the first stem cell therapy to receive European marketing approval. The therapy is used to treat moderate-to-severe forms of limbal stem cell deficiency (LSCD), a rare eye condition that can lead to blindness. “LSCD may affect one or both eyes; left untreated it results in chronic pain, photophobia, inflammation, corneal neovascularisation and the reduction or complete loss of vision,” says Professor Sir Michael. “Holoclar consists of a sheet of viable autologous human corneal epithelial cells and is transplanted into the affected eye(s) after removal of the damaged corneal epithelium. The Judging Panel agreed that it was tremendous to see this level of innovation in an orphan indication.”
A commendation was awarded to Alexion for Strensiq, its treatment for childhood-onset hypophosphatasia.
Real World Evidence Award
Prix Galien Awards currently take place in 17 countries around the world. However, the UK is the first to introduce a category for Real World Evidence. Explaining this pioneering introduction, Professor Sir Michael said: “The UK Steering Group felt that the time was right, particularly as many organisations have begun to initiate important pieces of evidence collection following the launch of their products. There is no doubt that efforts made in generating high-quality RWE data is critical in facilitating reimbursement for new technologies – and we fully expect other award groups to follow suit.”
The inaugural RWE Award was won by Astellas’ treatment for clostridium difficile infection (CDI) Dificlir, which was launched in 2012 and received a commendation at UK Prix Galien 2014. Outlining the rationale behind its win, Professor Sir Michael said: “A unique series of local service evaluations were conducted in 2013–2014 to evaluate the impact of Dificlir introduction on the NHS. The evaluation, studied in real world settings, included investigating its effects on service delivery, the management of CDI and its costs, primarily to inform local decision-making. Results indicated the very significant contribution that Dificlir’s use can make to tackling the major public health problems of antimicrobial resistance through targeted antibiotic therapy and infection control.”
Karen Westaway, Chief Executive of ValueBase, the new owners of Prix Galien’s UK franchise, said: “UK Prix Galien has once again showed the depth and diversity of medical innovation in UK life sciences. In an Olympic year, it’s easy to see why UK pharma consistently remains at the top of the global medal table for innovation. We are particularly proud that our awards are themselves innovating, becoming the first Prix Galien nation to recognise excellence in Real World Evidence. We know that real world evidence will, alongside traditional RCT data, become crucial to the uptake of new medicines and the upward trajectory of patient outcomes. But as we reflect on a magnificent ceremony and prepare for the next one in 2018, the list of finalists for UK Prix Galien 2016 provides the most striking real world evidence of all: UK pharma is as strong as ever.”