Approximately 30 million people live with rare diseases in Europe. In a drive to improve treatment for people with these complex conditions, a cross border cooperation via Directive 2011/24/EU “Patient’s rights in cross border healthcare,”1 has provided a framework for the development of European Reference Networks (ERNs).
Gaining reimbursement for rare and specialist diseases can be challenging, primarily because small patient populations restrict the size of clinical trial cohorts. This limits the clinical evidence available to support a drug through the health technology assessment (HTA) process. Other factors, such as diagnosis, capturing long-term data, and defining patient populations can also hinder market […]
The Republic of Ireland has become the first country in Europe to switch a whole population suffering from Haemophilia A and Haemophilia B from short-acting clotting factors to new generation, extended half-life (EHL) therapies. This news highlights the importance of a good tendering strategy to ensure market access.
What do your target product profiles (TPP) say about you? Are you the pragmatist? The dreamer? Or maybe the pessimist? Fortunately, we cannot gauge your personality type from the TPP, or at least we should not be able to. We should, however, be able to understand enough about your product to gauge the potential value […]
In 2015, Lord Carter published an independent report on operational performance and productivity for the UK national health service (NHS)1. Recommendations outlined how the NHS could save £5 billion by improving operational consistency between trusts. Over the last 18 months, the NHS has set about implementing these recommendations and, in January 2017, a new procurement […]
Recent times have shown that nothing influences the trajectory of healthcare innovation like the convergence of medicine and technology; but for now, the full potential of this pairing remains to be seen.
For decades, the pharma industry has been at the centre of an incessant debate: transparency in drug pricing. Pharma faces a perpetual paradox of compliance and standardisation on the one hand, and commercial sustainability on the other. Consequently, its drug pricing agenda rides a constant rollercoaster of scrutiny.
With rising treatment prices and healthcare spending, payer organisations are making efforts to contain costs, with the unintended consequence of putting up treatment access barriers. Hence, there is a growing interest in more innovative agreements, such as performance-based (reimbursements tied to conditions around producing outcomes or bridging evidence gaps), finance-based (rebates/discounts for excesses in agreed-upon […]
2017 saw the National Institute for Health and Care Excellence (NICE) board, with input from the NHS England Specialised Services Commissioning Committee, agree to a change that will have a substantial impact on both patients and the companies bringing ultra-orphan drugs to market. In this article we consider what is going to change and how […]
Biobetters are biologics that have been improved compared to the marketed originator, for example in efficacy, safety, tolerability or dosing regimen. For regulatory approval, biobetters must meet the same data requirements as all other originator products, whilst only a reduced clinical data package is needed for biosimilars (Table 1). This produces time pressure for biobetters […]