In health care, “access” can be defined as the opportunity or right to receive health care. Accessibility has many aspects e.g. infrastructure, reimbursement, health workforce and others. One of the major success factors of treatment is access to the right drug therapy at the right time for the right patient and finally reimbursement of the drug. A series of innovative treatments alongside ongoing discussions on costs and access to these treatments have been accelerated. In times of escalating budgetary pressure, access to innovative therapies also remains critical in many countries. Physicians are increasingly limited in prescribing expensive drugs and the influence of payers and budget holders continues to increase. In the case of prescribing cost intensive therapies, physicians are increasingly requested to justify their decision based on high clinical evidence and economic considerations.
At the market launch of innovative therapies, Health Technology Assessments (HTA) and Value Dossiers gain importance in payers decisions on reimbursement of new therapies. Due to the need for data transparency and the current lack of data available, the preparation of submission dossiers for reimbursements is challenging, and often associated with information gaps leading to subsequent negative effects on reimbursement discussions. The IPSG Access Expert Working Group was formed in 2018 with the aim to collect information from IPSG Members, and interdisciplinary experts within the international community to identify deficits and needs in health care provision and access to new therapies, and to develop strategies for overcoming potential reimbursement barriers.
After licensing of a drug, payers require information on long-term safety, benefits in terms of outcomes, and costs for standard of care and innovative therapies in different patient groups for a reassessment of reimbursement conditions. In this context, discussions are ongoing whether data collected in routine clinical practice, so-called real-world data (RWD), could be considered for market licensing, pricing and reimbursement discussions. RWD in research are sub-classified as primary and secondary data sources. Primary data are collected with a research intention; for example, registries. Secondary data has not been primarily collected for research purposes, but instead for other purposes, such as reimbursement in the case of claims data. Presently, RWD has earned greater visibility, especially in rare diseases, by complementing sparse study data, and filling evidence gaps.
As a result, the objectives of the Access Expert Working Group include the following:
- To inform clinicians and health care professionals on major trends and needs that will have impact on the provision of haemophilia care and the access (clinicians and patients) to therapy
- Developing strategies how to maintain / achieve optimal patient care
- Feed back to other IPSG groups on identified needs
- Summary of statement, consensus papers to be submitted for publication in peer reviewed journals