A robust agenda to decide the future of healthcare in Europe looms large over the upcoming elections to the European Parliament in May. In the lead up to election day, the current Presidency of the European Union, Romania, has already advocated for an ambitious healthcare plan – including efforts to ensure patients’ access to medicines across the continent.
The unprecedented pace of biomedical research continues to yield new medicines, renewing hope for European patients waiting for better therapies to treat their unmet medical needs. The European Medicines Agency (EMA) is approving more novel drugs every year: in 2018, 42 new active substances were approved by the EMA, an increase from 35 in 2017 and 27 in 2016. These new approvals hold promise for patients in treatments ranging from gene therapies to monoclonal antibodies.
Despite this progress, some patients in Europe are unable to access the medicines they need due to post-approval market access delays throughout the EU. Some medicines can be delayed by an average of 400 days after EMA approval, blocking access to critical medicines for months or even years. In countries where manufacturers command lower prices, such as Poland and EU candidate member Serbia, patients can sometimes wait as long as 1,000 days to receive medicines that have already been approved by the European regulatory authorities.
After securing EMA approval, manufacturers must then negotiate with each individual EU member state to reach pricing and reimbursement agreements. Since pricing procedures differ widely among national health authorities, there are often vast disparities in the time required for a new medicine to enter each European country. Complicating matters, countries with smaller markets often delay negotiations with companies until similar negotiations have been completed in larger markets, whose pricing schemes can provide a ceiling for price negotiations. Given some of the higher prices commanded by new treatments, resource-restricted smaller countries may delay uptake of new drugs in order to defer large payments, despite the impact on patients.
Previous reform efforts have fallen short. In 2012, the European Commission proposed a strict 120-day window for reimbursement determinations to be enforced across all member states – an effort that was supported by European drug manufacturers. But when it was finally adopted, the EU Transparency Directive instead used a 180-day window. This ignores the fact that even a two-month delay can result in unnecessary suffering for patients or death, especially those with life-threatening rare diseases that have no other effective treatment options.
Moreover, the inability of reform efforts to sufficiently address this problem is inconsistent with Europe’s Healthy Ageing goals, which include strengthening European health systems to meet the changing needs of an ageing population and ensuring equal access to quality healthcare, within and between countries.
In recent months, these challenges have only intensified due to the uncertainty around market access for novel medicines that treat rare conditions, called ‘orphan drugs.’ Due to the considerably higher prices commanded by many ‘orphan drugs,’ biopharmaceutical companies and payers have begun to implement value-based payment models to amortise payments and tie them directly to medical outcomes. However, these payment models often lead to prolonged and demanding negotiations, exacerbating delays.
For example, a breakthrough treatment for cystic fibrosis received EMA approval in November 2015 but is still not available in the UK, France, and Spain, and did not enter the German market until December 2016. In England, delays went on for so long that nearly 8,000 packets of the treatment exceeded their 2018 expiry and had to be destroyed, leaving more than 2,800 cystic fibrosis patients waiting to receive treatment.
Similar delays face a different medication to treat spinal muscular atrophy (SMA), which was approved by the EMA in June 2017. By October 2018, few EU markets had reimbursed access for all SMA patients, even though this is the only existing treatment for the disease.
These examples highlight the real-word impact of blocking access to life-altering treatments for patients. EU health authorities must answer a critical question: how can companies and payers negotiate to capture the most value for patients and adhere to cost-effectiveness standards without impeding patient access?
In the absence of an all-encompassing government solution, payers are turning to other strategies to shorten negotiations. In particular, real-world evidence collection has been used to inform payment agreements tied to patient outcomes – a positive first step. This would allow for companies to utilize data from larger European countries to inform product negotiations in smaller states.
9 April marks European Patients’ Rights Day and with that, a reminder that access to high-quality, innovative care is fundamental to European values at the heart of a compassionate, democratic society. The breakneck speed of biomedical innovation and newly approved medicines can only achieve full value if we have unencumbered patient access. Negotiations between healthcare stakeholders should not impede the ultimate stakeholder – patients – from receiving the treatments they so urgently need.