From 2010 to 2019, inpatient departments of the Heidelberg University Hospital incurred average annual costs of € 45.1 million (SD = ±7.6 million) for drugs and pharmaceutical products. In 2019, the highest costs ever of € 60.5 million were recorded. Figure 1 shows the annual expenditure on drugs in the five departments that generate the largest annual costs for pharmaceuticals at Heidelberg University Hospital.
Since 2016, the strongest increase in drug expenditure took place in the Center for Child and Adolescent Medicine with annual progressions of up to 84 percent. In 2019, € 14.46 million were spent on pharmaceuticals in the field of pediatrics. Compared to 2010, this represents an increase in expenditure by a factor of 5.3, essentially generated by the Department of Pediatric Neurology (74 %) (Figure 1).
A detailed investigation of the Department of Pediatric Neurology identified in particular two drugs for the treatment of SMA as the main cost drivers: Nusinersen and Onasemnogene Abeparvovec. 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.(14) The conditional marketing authorization of Onasemnogene Abeparvovec by the EMA was granted in May 2020. However, already in the first half of 2020, three more patients were treated with Onasemnogene Abeparvovec with total drug expenditures of € 5.835 million plus import VAT, more patients will follow in the course of the year.
The incidence of SMA is estimated at 1:7,500 for Germany. It represents the most common genetic cause of infant and childhood mortality.(15, 16) Most patients suffer from subtype 1 (Werdnig-Hoffmann) and are diagnosed in infancy. In our survey 1,097 patients were included. Of these patients, 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 next 12 months, confronting payors with total drug costs of € 178.94 million (corresponding to € 212.94 million gross). According to the survey results, the number of patients with Nusinersen therapy will decrease from 867 during the last 12 months to 761 in the coming 12 months (Supplementary Table 1).
All 41 respondents state that the application of innovative SMA therapies in standard care create new challenges, 28 (68.3%) expect challenges half medical and half non-medical in character. A total of 82.9 % (n = 34) of those responsible for the organization in their centers report an additional workload of more than 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 infrastructural 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, there is a strong need for improvement and more precise standardization of follow-up care, 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) even stated that compensation was insufficient, and 95.2 % (n = 39) saw the reason for this in the existing outpatient remuneration systems. 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 and analysis of the outcomes of innovative therapies should be carried out in international registries, 94.4% (n = 39) also support that this should be done independently of the pharmaceutical industry. There was also broad consensus concerning financing of clinical trial registers, 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). Since international recommendations for the implementation of registry work are missing so far, a modified model of quality management and price-finding based on the dynamic evidence price will be presented in the discussion.