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Salvador Payán Pernía on Equity Challenges in Access to Gene Therapy for Hemoglobinopathies
Salvador Payán Pernía/LinkedIn
Sep 29, 2025, 04:16

Salvador Payán Pernía on Equity Challenges in Access to Gene Therapy for Hemoglobinopathies

Salvador Payán Pernía, Member of the Board of Directors of the Erythropathology Group at Spanish Society of Hematology and Hemotherapy (SEHH), shared on LinkedIn:

”As with other advanced therapies, access to gene therapy in hemoglobinopatías will face, once funded, equity risks arising from its high cost and the authorization of only certain centers for its administration.

For example, patients could be induced to participate in a particular clinical trial rather than referred to the referral center for therapy, or “own” patients could be prioritized over referrals at the referral center.

These risks may be greater in haemoglobinopathies compared to other diseases, as they are rare diseases in which management expertise is limited to a few centres and because they preferentially affect, especially sickle cell disease, marginalised groups.

In addition, patients will have to “compete” for hospital beds with patients with haematological cancer, for whom there is already a lack of beds, with a greater vital urgency in terms of treatment and greater awareness of the disease among decision-making health teams.

In this context, the efforts of the pharmaceutical industry, patient associations, and the scientific community should not be satisfied with funding, but in parallel and in advance the equidad should be addressed.

The creation of a national committee of experts for the review of proposed patients, or the development of a national “prioritization protocol” like that of a North American institution published a few days ago are examples of feasible strategies.”

Read the full article in Blood Advances.

Article: An ethical allocation scheme for scarce gene therapies in sickle cell disease and transfusion-dependent β-thalassemia

Authors: Amar H. Kelkar, Maureen O. Achebe, Andrew Hantel

Salvador Payán Pernía on Equity Challenges in Access to Gene Therapy for Hemoglobinopathies

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Amar H. Kelkar Andrew Hantel Blood Advances Gene therapy Health Hematology Hemoglobinopathies Hemostasis Hemostasis Today Maureen O. Achebe Medicine Salvador Payán Pernía thrombosis
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