From 2010 to 2020, inpatient departments of the Heidelberg University Hospital incurred average annual costs of € 48.7 million (SD = € ± 14.4 million) for drugs. In 2020, the highest cost ever of € 88.1 million was recorded. Table 1 shows the annual expenditure on drugs of the five departments with the highest annual costs for drugs at Heidelberg University Hospital.
The trend of rapidly rising costs for medications can be illustrated by the figures from the Center for Pediatric and Adolescent Medicine. Since 2016, there have been annual increases in expenditures here. Most recently in 2020, there were costs of € 34.05 million and an increase of 135 % compared with the previous year (Table 1 and Figure 1 A). Compared to 2010, 12.5 times the amount was spent on medications in 2020, while the number of prescriptions filled remained constant throughout the observation period. The majority of these costs (92 %) can be attributed to the field of pediatric neurology (Figure 1 B).
A detailed investigation identified in particular two drugs for the treatment of SMA as the main cost drivers: Nusinersen, responsible for an increase of drug costs from 2017 on and Onasemnogene Abeparvovec with a single dose applied in 2019 and a total of 10 doses applied in 2020. In 2019, 92 doses of Nusinersen were applied at the Centre for Children and Adolescent Medicine Heidelberg with a total expenditure of € 8.44 million. The average drug cost of a single application was € 91,739.13. In the same year, a first patient was treated with Onasemnogene Abeparvovec, the net drug cost amounted to € 1.945 million.(19) The conditional marketing authorization of Onasemnogene Abeparvovec by the EMA was granted in May 2020 which has led to a rapid increase in demand for the drug. During 2020, a total of 10 patients were treated with Onasemnogene Abeparvovec, resulting in total drug spending worth € 19.45 million plus import VAT.
To put these findings in a broader context, we concluded a survey of 41 German SMA treatment centers caring for a total of 1,097 patients. Within the patient cohort, 646 (59 %) were younger than 18 years of age at the time of the survey. Based on our survey, in Germany, 92 patients are expected to qualify for start of treatment with Onasemnogen Abeparvovec during the following 12 months, confronting insurance companies with total drug costs of € 178.94 million plus VAT. Compared to other drug groups, such as TNF-alpha inhibitors (Adalimumab: € 468.1 million) or modern anticoagulants (Apixaban: € 475.3 million)(20), this amount appears to be small, but it must be emphasized that the number of patients treated with Onasemnogen Abeparvovec is much lower and it remains unclear whether recurrent drug administration will be necessary for individual patients. According to the survey, the number of patients with Nusinersen therapy will decrease from 867 during the most recent 12 months to 761 in the coming 12 months (Supplementary Table 1).
All 41 centers state that the application of innovative SMA therapies in standard care creates new challenges, 28 (68.3%) centers expect challenges of medical and non-medical character. A total of 82.9 % (n = 34) report an additional workload of more than at least 5 hours per week. New tasks will primarily arise in the areas of case management, pre- and post-clinical care, and administration of reimbursement or invoicing. The need for additional human resources (even more so than infrastructure or financial resources) is thus perceived as the biggest challenge in the overall process of applying innovative therapies (Figure 2).
Those responsible for the SMA treatment centers see the need for standardization of processes. While both, the indication process as well as the pharmacy process were reported to be well regulated, a strong need for improvement and more precise standardization of follow-up care was reported by 39 (95.1 %) respondents. At the time of investigation, only 25 (61 %) of the centers reported to have standard operating procedures (SOPs) related to SMA treatment. While reporting an increase of workload, 80.5 % (n = 33) of the respondents do not consider the reimbursement for the healthcare providers of innovative therapies to be appropriate. With regard to follow-up care, 87.8 % (n = 36) stated that compensation was insufficient, and 95.2 % (n = 39) saw that the reason for this is in the existing outpatient reimbursement system. When respondents were given the opportunity to prioritize different areas of optimization, the compensation for aftercare was ranked highest.
All respondents indicated that structured documentation of the outcome of innovative therapies should be carried out in international registries. A majority of 94.4% (n = 39) stated that this should happen independently of the pharmaceutical industry. There was also broad consensus concerning financing of clinical trial registries, where 80.5 % (n = 33) of the respondents were in favor of a fund to be administered in trust by the Federal Joint Committee, into which the pharmaceutical company would have to pay an amount based on the price of the drug after approval. Only a small proportion of those surveyed saw responsibility for structured documentation with the insurance companies (12.2 %, n = 5) or with the health-care providers (7.3 %, n = 3). With few exceptions,(21) international recommendations for the implementation of registry work are missing so far.