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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.

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Editorial support in the form of initial preparation of the outline based on input from authors, and collation and incorporation of author feedback to develop subsequent drafts, copyediting, and referencing was provided by Lisa Auker, PhD, of Fishawack Communications Ltd, part of Fishawack Health, which was funded by Boehringer Ingelheim International GmbH.

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Correspondence to Luca Pani.

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Boehringer Ingelheim International GmbH funded editorial support in the form of initial preparation of the manuscript outline based on input from authors, and collation and incorporation of author feedback to develop subsequent drafts, copyediting, and referencing.

Conflicts of Interests/Competing interest

LP is a part-time employee of the Universities of Modena and Reggio Emilia, Italy and Miami, USA and acts as Chief Scientific Officer for the EDRA-LSWR Publishing Company and the Inpeco SA Total Lab Automation Company; he is also VP for Regulatory Strategy and Market Access Innovation at VeraSci, Durham, USA. He has previously acted as a scientific consultant for AbbVie USA, Acadia USA, BCG Switzerland, Boehringer Ingelheim International GmbH, Compass Pathways, Ferrer Spain, Johnson & Johnson USA, NeuroCog Trials USA, Otsuka USA, Pfizer Global USA, PharmaMar Spain, Relmada USA, and Takeda USA. Karin Becker is a full-time employee of Boehringer Ingelheim International GmbH.

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Pani, L., Becker, K. New Models for the Evaluation of Specialized Medicinal Products: Beyond Conventional Health Technology Assessment and Pricing. Clin Drug Investig 41, 529–537 (2021).

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