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A Framework for Assessing the Value of Rare Disease Treatments


Difficulty in making pricing and reimbursement decisions for rare diseases has prevented patients from having universal access to new medicines despite increased investment in their research and development. In Europe, a group has proposed 9 principles to guide value assessment, pricing, and funding.

Difficulty making pricing and reimbursement decisions for rare diseases has prevented patients from having universal access to new medicines despite increased investment in their research and development.

The European Working Group for Value Assessment and Fund Processes in Rare Diseases (ORPH-VAL) has proposed 9 principles to guide value assessment, pricing, and funding processes for rare disease drugs, while also contributing to the sustainability of both healthcare systems and innovation. These principles were published in Orphanet Journal of Rare Diseases.

The 9 principles fall into 4 areas: orphan drug decision criteria, orphan drug decision process, orphan drug sustainable funding systems, and European coordination.

“Rare diseases are an important public health issue and it is important that patients with these diseases have access to safe and effective therapies in the same way as others suffering from more common diseases,” the 15 authors on the paper wrote.

The principles are:

Principle 1 (decision criteria)

Orphan medicinal product (OMP) assessment should consider all relevant elements of product value in an appropriate multi-dimensional framework. This includes considering the OMP value from perspectives of patients, the health system, and society as a whole.

Principle 2 (decision criteria)

Pricing and reimbursement decisions should be founded on the assessment of OMP value and adjusted to reflect other considerations beyond product value. Reimbursement decisions should be based on principle 1, and the price should be informed by considering the product value in light of price-value precedents.

Principle 3 (decision process)

Those making pricing and reimbursement decisions about OMPs at a national level should take into account all official regulatory and health technology assessments of OMPs undertaken at the European level.

Recommendations from the following should be considered: the European Public Assessment Report and Summary of Product Characteristics from the European Medicines Agency; relative effectiveness assessments undertaken by the European network for health technology assessment; and assessments on significant benefit and prevalence from the Committee for Orphan Medicinal Products

Principle 4 (decision process)

The assessment and appraisal of OMPs to inform national pricing and reimbursement decisions should incorporate rare disease expertise, including both the healthcare professionals’ and patients’ perspectives.

Principle 5 (decision process)

To accommodate uncertainty, value assessment and pricing and reimbursement decisions should be adaptive subject to the need and availability of information over time. Payers should consider the uncertainty around product value for rare diseases in light of: disease prevalence; disease severity and unmet need; amount of prior research conducted in the disease; and the extent to which the manufacturer has taken reasonable steps to minimize uncertainty.

Principle 6 (decision process)

All eligible patients within the authorized label of an OMP should be considered in the national pricing and reimbursement decision although different decision on access may apply to different subpopulations.

Principle 7 (sustainable funding systems)

Funding should be provided at the national level to ensure patient access to OMPs. This will avoid disparities in access between regions and regional and local funding bodies should work with national authorities to prevent inconsistencies and inequalities.

Principle 8 (sustainable funding systems)

Evidence-based funding mechanisms should be developed to guarantee long-term sustainability. Collaboration between manufacturers, payers, and health technology assessment agencies on horizon scanning can help budget holders plan for expenditures.

Principle 9 (European coordination)

In the future, there should be greater coordination on OMP value assessment processes at a European level. This coordination can help ensure more consistency among members in the definition and assessment of clinical value and provide opportunities for systemic collection and assessment of data while reducing duplication of efforts.

“The need for greater consistency, clarity, and certainty will become ever more important as the number of OMPs grows,” the authors concluded. “Yet, while there are still many thousands of rare diseases for which no effective treatments exist, all stakeholder—payers, policy makers, and manufacturers—will need to take a long-term perspective and build frameworks and processes that are capable of addressing the scale of the challenge.”

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