Advice on a potential candidate for conditional inclusion of rhPTH 1-84 (Natpar®) in chronic hypoparathyroidism
Zorginstituut Nederland recommends the minister of Medical Care that rhPTH 1-84 (Natpar®) be designated as a potential candidate for conditional inclusion. rhPTH 1-84 is registered for use as an additional treatment in adult patients with chronic hypoparathyroidism, whose condition cannot be sufficiently controlled with standard therapy alone.
Zorginstituut's advice
In 2020, the applicants submitted an application for the conditional inclusion of orphan drugs, conditionals and exceptionals to the Zorginstituut, for recombinant human parathyroid hormone 1-84 (rhPTH 1-84) (Natpar®). rhPTH 1-84 is registered for use as an additional treatment in adult patients with chronic hypoparathyroidism, whose condition cannot be sufficiently controlled with standard therapy alone.
Based on the data in the dossier and the advice of the Scientific Advisory Board (WAR), the Zorginstituut has concluded that treatment with rhPTH 1-84 in this group of patients with hypoparathyroidism meets the criteria for conditional inclusion, namely:
- rhPTH 1-84 has been granted marketing authorisation by the EMA and has ‘orphan drug’ and ‘conditional’ status.
- This is a case of an unmet medical need.
- The marketing authorisation holder is the dossier’s lead applicant. The co-applicants are an independent research institute, treating physicians, and patient associations.
- Based on the data it has collected, this study is expected to provide an answer to the ’package question’.
- The Zorginstituut anticipates that the ’package question’ will be answered within 3.5 to 4 years.
Based on these conclusions, we recommend that rhPTH 1-84 be designated as a potential candidate for conditional inclusion. Phase 2 of the procedure will commence when the Minister adopts this advice. We ask the parties to formulate their plans in greater detail and to draw up a covenant setting out the agreements needed to ensure that the CI process is conducted carefully and successfully. The Ministry of Health, Welfare and Sport will have to conclude a financial arrangement with the marketing authorisation holder.
Conditional inclusion
Whether a form of treatment or a medicinal product is worth including in the basic health care package depends on the availability of clinically relevant evidence of its effectiveness. Such information is generally not available (in sufficient quantities) at the moment of registration of medicinal products for severe - often less prevalent - diseases.
Conditional inclusion in the package will allow marketing authorisation holders an opportunity to carry out further research into the effectiveness and appropriate use in the Netherlands of these medicinal products.
Based on the research outcomes, the Zorginstituut will eventually be able to issue advice on whether a medicinal product complies with ‘established medical science and medical practice’, which means that its effectiveness has been scientifically proven.