In today’s rapidly evolving healthcare landscape, navigating the reimbursement pathway for high-cost highly specialised therapies presents a formidable challenge. These treatments, including groundbreaking innovations like cell and gene therapies, offer significant hope for patients with previously untreatable conditions. However, the financial burden they place on public hospitals often exceeds traditional healthcare budgets, making their adoption difficult for state and territory healthcare systems.
In Australia, the funding and reimbursement processes for these therapies are intricate, involving multiple stakeholders such as the Pharmaceutical Benefits Advisory Committee (PBAC), Medical Services Advisory Committee (MSAC), and state-based Health Technology Assessment (HTA) bodies. HTA for high-cost highly specialised therapies has several challenges, including:
- A smaller evidence base due to trial designs and smaller patient populations
- High uncertainty regarding long-term health outcomes
- Uncertainty about cost-effectiveness
- Potential for individualised therapy
A multifaceted approach that includes early engagement with HTA bodies, robust evidence generation, and strategic advocacy is essential to successfully navigate these complexities.
Criteria and Funding Process
High-cost HSTs are funded through the National Health Reform Agreement (NHRA). The NHRA defines high-cost HSTs as TGA-approved medicines and biologicals delivered in public hospitals where:
- The therapy and its conditions of use are recommended by MSAC or PBAC.
- The average annual treatment cost at the commencement of funding exceeds $200,000 per patient (including ancillary services), as determined by MSAC or PBAC with input from the Independent Hospital Pricing Authority.
- The therapy is not otherwise funded through a Commonwealth program or the therapy’s costs would be appropriately funded through an existing pricing classification.
The decision of whether a therapy meets the NHRA criteria is jointly made by the MSAC and PBAC chairs, along with representatives from each state and territory. Figure 1 outlines this decision-making process.
Figure 1. Decision tree for determining if a health technology should be treated as a potential NHRA high cost, HST
If a reimbursement application meets the criteria for an HST, it follows the MSAC assessment pathway. If it does not, other funding programs, such as the Pharmaceutical Benefits Scheme (PBS), may be considered. The information required for MSAC consideration is consistent with other submissions but includes input from state and territory health departments. Patient and consumer perspectives are also considered through existing MSAC consultations.
After a supportive MSAC recommendation, a Deed of Agreement is negotiated between the Commonwealth and the sponsor company, with states and territories notified on the same day the company agrees to MSAC’s recommendations (Figure 2). Each state and territory then decides when and where the therapy will be provided. The assessment timeframe for HSTs is the same as for other health technologies considered by MSAC. However, as with MBS listings, there is no specific timeframe for funding after MSAC advice.
Figure 2. Reimbursement process for high cost highly specialised therapies
The following high-cost HSTs are currently funded under the NHRA:
Case Study: CAR-T Cell Therapy
Chimeric Antigen Receptor-T (CAR-T) cell therapies are among the most promising and high-cost treatments in the Australian healthcare system. By reprogramming a patient’s immune cells to target and eliminate cancer cells, these therapies offer potentially life-saving solutions for certain cancers. However, the reimbursement process is highly complex, and this case study is presented here to guide sponsors in navigating the reimbursement pathway.
- Pre-Submission Stage
Before lodging an MSAC application (also known as applicant developed assessment report, ADAR), sponsors are invited to attend pre-submission meetings with the MSAC Secretariat and representatives from key state and territory jurisdictions. These meetings help jurisdictions understand the disease area and review preliminary data, aiding in budget management. Importantly, they allow discussion around which qualified and accredited centres could deliver the new CAR-T cell therapy.
While pricing isn’t the focus of these discussions, understanding expected patient numbers and financial implications is crucial. Pre-submission meetings also allow sponsors to identify key stakeholders in state and territory health departments, a vital step for navigating subsequent stages in the funding and implementation process.
2. Advocacy
Patient advocacy plays a crucial role in the reimbursement process for high-cost highly specialised therapies like CAR-T. Engaging patients, carers, and clinicians can bring real-world perspectives that complement clinical and economic data, helping to highlight the therapy’s broader impact. By involving advocacy early in the process, sponsors can shape public and governmental opinion, strengthen their case for reimbursement, improve post-implementation monitoring, and ensure long-term access to these therapies.
3. PASC Meeting
An inhouse analysis of the seven CAR-T cell therapy ADARs lodged to MSAC indicates most ADARs (5/7 = 71%) successfully bypassed a PICO Advisory Sub-Committee (PASC) meeting. Further, ADARs required an average of two MSAC meetings to receive a positive recommendation and took an average of 1.8 years from lodgement date of application form to receive a positive MSAC recommendation (Table 1).
Table 1. Summary of CAR-T cell therapy ADARs
4. MSAC Submission and Evaluation Stage
The Department of Health and Aged Care commissions an external HTA group to evaluate the ADAR and provide a critique, which is then shared with the sponsor for feedback. The Evaluation Sub-Committee (ESC) reviews the ADAR, the critique (Commentary), and the sponsor’s responses.
Simultaneously, jurisdictional Drug and Therapeutics Committees (DTCs) assess the therapy’s clinical and cost-effectiveness. This feedback plays a crucial role in refining the submission and addressing concerns raised by the ESC and jurisdictional representatives. Timely and thorough engagement with this additional evaluation process can significantly improve the chances of a positive recommendation.
5. MSAC Consideration
MSAC can recommend funding for a therapy even if state and territory representatives raise concerns about its cost. Early alignment between stakeholders can prevent conflicts later in the process, and clear communication is essential for market access professionals to balance differing priorities between the payers.
6. Supply Agreements and Implementation
Once MSAC recommends funding, the sponsor enters into a Commonwealth Deed, followed by state-level or hospital-level supply agreements. The implementation process can be lengthy, making it essential for sponsors to leverage the relationships built during pre-submission meetings with key contacts in state health and hospital departments to ensure smooth progress.
7. Post-Monitoring
Sponsors must plan for extensive post-implementation monitoring, including ongoing data collection to confirm that the therapy continues to meet its expected outcomes. Consistent communication with HTA bodies is crucial to ensure the long-term value of the therapy is maintained.
Conclusion
Navigating the reimbursement process for high-cost, highly specialised therapies can be complex. This newsletter highlights key strategies for market access professionals, including early engagement with HTA bodies and leveraging patient and clinician advocacy.
We hope the case study on CAR-T cell therapy will help guide sponsors through the intricate process of securing reimbursement of high-cost, highly specialised therapies in Australia’s healthcare system.
