In this text, Mary Rosenzweig, Country director Denmark at Quantify Research, gives her perspectives on the changing landscapes in market access and real-world evidence in Denmark and the keys to successful value demonstration of new innovative medicines.

Real-world evidence can reduce uncertainty and improve patient access

The Danish Medicines Council has been exploring new approaches to market access including managed entry agreements. One approach is where a new treatment is offered to a group of patients while its use is being monitored in the clinical setting for a period, allowing for real-world effectiveness data to be collected over time. As an example, in January 2018, the Medicine Council recommended this new approach for the medicine Spinraza (nusinersen) to treat a limited patient population with SMA type 1 and for pre-symptomatic infants.

Another relatively new approach in Denmark is outcomes-based price agreements, which are meant to guarantee that the regions only pay for the real-world health outcomes provided by the treatment. In 2020, this approach was applied to treatment with Luxturna (voretigene neparvovec), which is indicated for the treatment of vision loss due to inherited retinal dystrophy, caused by a genetic mutation. Although the follow-up period for the price agreement is unknown, the regions pay the manufacturer through instalments, and the cost is dependent on the observed effect of the medicine. The same approach was taken in a pilot project for prescription medicines that are collected at private pharmacies. This method ensures that the public health expenses only cover the costs of reimbursement for a limited patient population, while the company pays for those patients who do not meet the conditions for reimbursement. Two drugs were chosen for this pilot: The blood thinner Brilique 60 mg and Skilarence to treat severe plaque psoriasis. It will run until the end of 2021.

Each of these novel approaches to market access highlight the need for fit-for-purpose real-world evidence, which requires a combination of high-quality data and expert competence within this field. Real-world evidence will inevitably play a key role in the future, and many aspects of the necessary infrastructure are already in place by virtue of the unique health data available in Denmark and other Nordic countries. The Nordic registers have broad coverage, continuously improve in quality and digitalization allows for new types of data sources and information to be collected. Together, this information can provide crucial insights into the value of new treatments. Quantify has unsurpassed experience in generating the type of tailored real-world evidence that I believe will be increasingly demanded throughout all levels of healthcare. In doing so, we are able to support manufacturers meet the increasing evidence requirements set out by payers and regulators.