The Role of HEOR in Expanding Market Access for Orphan Drugs

The role of Health Economics and Outcomes Research (HEOR) is extensive. Helping to guide decisions about how to provide healthcare, who should receive it, and how it should be delivered, it is the cornerstone of the healthcare sector. And it plays a pivotal role in expanding market access for orphan drugs. With orphan drugs only being needed by a small population, their development is often sidelined in favour of treatments with greater commercial viability. Using economic principles alongside clinical and social evaluation, HEOR is helping to further market access for many orphan drugs.

HEOR’s Role in Expanding Market Access for Orphan Drugs

Economic value assessment

Orphan drugs are often expensive to develop and manufacture. This is due to the typically small numbers of patients that need to use them, which means limited economies of scale. HEOR evaluates whether the cost of an orphan drug is justified by its health benefits. If a drug only offers minimal benefits to the end user, it can’t be said to offer value for money.

HEOR also implements budget impact modelling to estimate the financial impact each new orphan drug is likely to have on healthcare budgets. And assesses the comparative effectiveness of each new orphan drug against existing treatments or no treatment.

Clinical outcomes and patient impact

From the patient’s perspective, this is the number one measure of whether a drug carries value. And it can often seem that healthcare authorities fail to take this into account when making financial decisions. But the reality is that HEOR assesses both patient survival rates and quality of life improvements when evaluating the effectiveness of orphan drugs.

Monitoring quality-adjusted life years (QALYs), HEOR assesses the value of drugs based upon broader impact on patient well-being. While using real-world evidence to gather insights into a drug’s performance in the widest possible patient populations.

Regulatory decisions

The majority of orphan drugs require regulatory approval before release to market. HEOR data can be used in regulatory dossiers to show that orphan drugs provide sufficient benefit to justify their approval. This can be particularly beneficial for orphan drugs, where it is often not viable to conduct a large-scale clinical trial.

Reimbursement support

A positive reimbursement decision from a range of payers—including government programs, private insurers, and healthcare providers—is integral to the release of new orphan drugs. This decision process is heavily reliant on HEOR studies, which provide evidence of a drug’s value. This supports negotiations for price and access.

Social and ethical value

A life-threatening or debilitating illness can have a profound impact on far more than the individual afflicted. Families, friends, colleagues, schools, and other caregivers can all be impacted. HEOR can help to highlight unmet needs by demonstrating the lack of or limited treatment options for a specific condition. And demonstrate the broader societal benefits that a particular drug could bring.

Stakeholder education

By providing data-driven insights, HEOR can help to educate healthcare professionals and decision-makers about the clinical and economic value of orphan drugs.

Market expansion

HEOR supports manufacturers in devising strategies to gain market entry and expand market reach. This can be done through the identification of target populations, negotiating with payers, or ensuring regulatory approvals.

The role of HEOR in expanding the market access of orphan drugs is the provision of evidence. Financial, social, and ethical. Without this evidence, the process of taking orphan drugs to market would be considerably slower and less effective. Leaving patients with rare diseases and other health conditions waiting for far longer than they should be for potentially life-saving or life-altering treatments.

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