Abstract
BACKGROUND: Patients with hard-to-treat or rare cancers and those not responding to standard-of-care (SoC) treatment have unmet needs. Limited access to novel drugs is an increasing additional challenge. In 2003, the Danish government adopted a Health Act to ensure that treatment of patients with life-threatening disease could be reevaluated by independent experts. The Danish Health Authority (DHA) set up an Expert Advisory Panel to provide advice on possibilities for further treatment of patients, including treatment not approved nationally. A few years later, clinical units were established that could offer unestablished treatment to patients by referral from the Panel. The treatment was first reimbursed by the Government and later by regional authorities. MATERIALS AND METHODS: We present the structure, workflow, and impact of the Health Act for 21 years for patients with cancer. Annual reports from the DHA were the primary data source. RESULTS: 11 034 cases from 9603 cancer patients were evaluated by the Panel from 2003 to 2023, representing a median of 372 unique cases yearly. In 53%, the Panel advised on further treatment in Denmark, and of these, 56% were recommended nationally nonapproved treatment, 21% SoC treatment or workup, and 19% clinical trial participation. In 4.5% of cases, advice was given on treatment abroad. A significant decline in admissions to the Panel from a peak of 1167 patients in 2008 to 3-400 yearly from 2012 to 2017 followed the conversion of nonapproved treatments to SoC practice. A shift in drug reimbursement, independent of Panel advise, reduced the clinical impact and explained the further decline observed in admissions lately to only 51 patients in 2023. CONCLUSIONS: This unique national scheme provided early access to treatment for patients with no further SoC options and facilitated the introduction of new cancer treatments, initiation of clinical trials, and establishment of trial units in the country. The scheme may be adapted to other countries with a public healthcare system. Results of the current report indicate that impact is dependent on delivering clinical units and reimbursement associated with the recommended treatment.