The Swedish Dental and Pharmaceutical Benefits Agency (TLV) has introduced new procedures to enhance access to medications for patients with rare health conditions, details of which were outlined in a recent report 1. This initiative stems from a government assignment received in February 2024 which set out to strengthen access to drugs for rare conditions without increasing overall state pharmaceutical expenses, and analysis showing that other countries in Europe and elsewhere have implemented similar schemes.
Small patient populations may mean pharmaceutical companies are disincentivised from launching drugs if there are so few patients in a market that it is infeasible to achieve a cost-effective price. TLV recognise this dynamic and, as such, will now consider a drug’s sale value and its potential patient population when making decisions about drug pricing and subsidies, with the goal of improving treatment availability for individuals with severe and rare diseases.
TLV will now accept a higher price relative to the benefit of a drug for patients with rare (or very severe) diseases, under certain conditions: severity will be measured using a mix of quantitative and qualitative variables, and ‘rare’ is currently defined as fewer than 100 patients in Sweden. Although this threshold is perhaps too low, and the changes will be implemented gradually to ensure the healthcare system is able to easily adapt, it indicates a broader change of mindset which can only benefit patients with rare diseases moving forward. The development of a new system for case management and pricing negotiations for these medicines is an additional promising step.
Of course, incorporating patient population and sales values into pricing decisions will also affect drugs for large patient populations. In order to minimise the budgetary impact of this policy shift, drugs with a high sales value may be subject to reductions in price, even if cost-benefit analysis indicates otherwise. This will not only be implemented at the point of initial reimbursement: TLV have proposed the development of a framework to monitor drug sales and patient numbers, with further pricing reductions possible if sales volumes are high.
The immediate impact of these policy changes should not be overstated. The threshold for defining a rare disease under this new framework is quite high, and high ICERs are still unlikely to be accepted where there is significant uncertainty embedded in a model. It is also clear that any price increases for drugs with low sales volumes must be accompanied by price decreases for drugs with high sales volume, something which may be difficult to implement.
However, even the suggestion that sales volume should be considered in pricing marks a significant shift in TLV’s thinking on this issue, and thus bodes well for further policy changes that might benefit rare disease population. Pharmaceutical companies, and the industry more widely, should therefore welcome these changes and commit to continue working towards improving access to life-changing drugs for patients with rare diseases.
