New Models for the Evaluation of Specialized Medicinal Products: Beyond Conventional Health Technology Assessment and Pricing

New specialized therapeutics coming to market, such as advanced therapy medicinal products (ATMPs) and orphan drugs, differ from traditional therapies in terms of how they are manufactured and administered, as well as the potentially transformative benefits they may provide. The current health technology assessment (HTA) process that has been used for traditional therapies, such as small molecule drugs and antibodies, does not work adequately for specialized therapeutics, with a key issue being the generation of sufficient evidence to adequately capture the full long-term benefits. The objectives of this article are to discuss why the current HTA process is inadequate for evaluating these new therapies, how evidence should be continuously generated and presented to regulators and payers to support their use, and to propose new approaches to pricing models. This will enable payers to have an affordable, risk-mitigated means of funding new therapies in a timely manner, thus guaranteeing patient access to new, potentially life-saving therapies, while providing manufacturers with a return on their investment. Without new approaches or adaptation of existing frameworks, certain ATMPs may not reach patients in some or all countries or be at risk of withdrawal from the market.