By Erfan Akbraian, Analyst
In recent years there have been discussions to shift to a multi-stakeholder approach and increase early communication amongst key stakeholders to ensure appropriate access of advanced therapy medicinal products (ATMPs). With an increasing number of ATMPs for orphan diseases in the pipeline, there is a need for health technology assessment (HTA) authorities to adapt their traditional assessment methods. The traditional assessment and pricing analyses have been developed mainly for chronic therapies addressing diseases with relatively large population. They do not address the clinical and financial uncertainties associated with ATMPs for orphan diseases.
Rare disease patient advocacy and patient involvement also fall under the category of multi-stakeholder communication. Many times, the payers involved in rare disease assessments will not be too familiar with the disease, and this is a big challenge when ensuring a balanced HTA assessment. Appropriate input from patient representatives can provide first-hand experience of the associated clinical, social and economic burden of the disease on patients and their families. Also patient input can help inform how a novel therapy could impact their quality of life on a day-to-day basis.
Patient input in HTAs is widely acknowledged as important to ensure patient centricity. During ISPOR Europe 2020, we presented a poster where we identified and classified the varying opportunities for patient involvement and their impact within the HTA appraisal and decision-making process across 12 countries (see below for weblink to download the poster). This work highlighted the need to develop more efficient patient input pathways, train patient representatives to provide relevant payer inputs, and for HTA bodies to be more transparent around how the patient voice will be incorporated within the decision-making process. This is critical to ensure HTAs for ATMPs in rare diseases are indeed patient centric
1. https://www.ispor.org/conferences-education/conferences/past-conferences/ispor-europe-2020/program/plenary-sessions [Plenary session: Patient and public involvement in healthcare decision making – are we maximizing opportunities?