Casgevy access in Pakistan: the DRAP named-patient pathway

Last reviewed 2026-05-16 by Reserve Meds clinical and regulatory team.

Quick orientation

Patients in Pakistan access Casgevy (exa-cel) for sickle cell disease and transfusion-dependent beta thalassemia through the DRAP named-patient mechanism, filed by a PMDC-licensed physician for a specific named patient. Because Casgevy requires REMS-certified administration, the realistic path often combines named-patient sourcing with coordinated travel; Reserve Meds handles US sourcing, logistics, and timeline planning end to end.

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How Casgevy reaches patients in Pakistan

Casgevy (exagamglogene autotemcel, exa-cel, the first FDA-approved CRISPR-Cas9 gene-edited autologous cell therapy, FDA-approved December 2023 for sickle cell disease and January 2024 for transfusion-dependent beta-thalassemia in patients 12 years and older) is not visible on the DRAP registered-product verification portal at dra.gov.pk/e-services/online-data-verification as of 2026-05-31. Pakistan does not currently host a Vertex Pharmaceuticals authorised treatment centre (ATC), so Casgevy access for Pakistani patients runs through a hybrid route: travel-to-treatment at an authorised ATC abroad (UK NHS at Manchester Royal Infirmary, KSA at King Faisal Specialist Hospital and Research Centre, UAE at Abu Dhabi Stem Cells Center, or US Vertex-authorised ATCs) combined with DRAP coordination for the apheresis-collection-to-infusion timeline. Where any component is imported into Pakistan (for instance, a cryopreserved cell product for institutional administration), the Special Permission for Institutional Import of Unregistered Therapeutic Goods under the Drugs Act 1976 applies, filed through dra.gov.pk/therapeutic-goods/import-export/special-permissions. The Casgevy-specific clinical justification must document: confirmed homozygous or compound heterozygous sickle cell genotype or transfusion-dependent beta-thalassemia, age 12 years or older, and HLA-matched related allogeneic stem cell transplant unavailable or inappropriate per the FDA label. Conditioning regimen (busulfan-based myeloablation) and post-infusion long-term follow-up commitments must be coordinated with the receiving ATC. DRAP processing for institutional import is several working days when documentation is complete.

Where Casgevy infusion patients are seen in Pakistan

Because Casgevy infusion happens at an authorised ATC abroad, the Pakistani role is haematology workup, eligibility confirmation, post-infusion surveillance, and long-term follow-up. The Pakistani tertiary haematology and BMT centres positioned for these phases include the Aga Khan University Hospital (AKUH) Department of Haematology and Bone Marrow Transplant Service in Karachi at hospitals.aku.edu, which runs Pakistan's longest-standing allogeneic BMT programme and is the most-frequent point of contact for haemoglobinopathy patients seeking gene-therapy referral; Shaukat Khanum Memorial Cancer Hospital and Research Centre (SKMCH and RC) BMT Service in Lahore at shaukatkhanum.org.pk; the National Institute of Blood Diseases and Bone Marrow Transplantation (NIBD) in Karachi, the dedicated tertiary haemoglobinopathy and thalassemia centre; the Children's Hospital and Institute of Child Health in Lahore with its Paediatric Haematology service for paediatric Casgevy candidates (eligible from age 12); and the Indus Hospital Haematology service in Karachi. NIBD specifically runs a national thalassemia registry and a sickle cell disease cohort and is the most appropriate point of entry for haemoglobinopathy genotype confirmation and donor-search workup before the ATC referral.

What Casgevy costs in Pakistan

The US reference WAC for Casgevy is USD 2.2 million per single-administration treatment course per Vertex Pharmaceuticals published pricing and Drugs@FDA at accessdata.fda.gov/scripts/cder/daf. This is a one-time cost rather than an annualised cost, but it does not include the apheresis, myeloablative conditioning, transplant admission, and ICU-capacity-equivalent post-infusion support, which add a further multi-hundred-thousand-USD component at any ATC. No public local-currency benchmark for Casgevy in PKR is observed; no Pakistani patient organisation has published a Casgevy quote; do not estimate. Price snapshot: 2026-05-31. Cost layers for a Pakistani-resident patient: the drug-product cost (USD 2.2M) plus the ATC's institutional treatment package (typically USD 200,000 to USD 500,000 depending on geography), travel and accommodation in the receiving country for the patient and a caregiver for 4 to 6 months (a defining cost component for cross-border gene therapy), DRAP institutional-import administrative fees if any logistic step touches Pakistan, and the Reserve Meds concierge fee for the multi-jurisdictional coordination. State Bank of Pakistan outward remittance is the binding constraint for staging payments.

Funding and access barriers for Casgevy in Pakistan

Pakistan has approximately 100,000 patients with sickle cell disease and a transfusion-dependent thalassemia population estimated by NIBD in the high tens of thousands; demand for curative gene therapy is large but funding remains the binding constraint. Public-sector funding for Casgevy at any scale is not observed as of 2026-05-31. The Sehat Sahulat Programme inpatient cap is not commensurate with the cost. Pakistan Bait-ul-Mal at pbm.gov.pk grants are case-by-case but historically capped well below the USD 2.2M list; the same is true of provincial Sehat Card Plus. Major private insurers (Jubilee Life, EFU Life, State Life, Adamjee Life) do not list gene therapy on standard retail policies and have not published an unlicensed-drug formulary inclusive of Casgevy; named-individual catastrophic-illness riders on corporate group policies are theoretically capable but no Pakistani Casgevy claim adjudicated by a private insurer is publicly documented. SKMCH Zakat and Indus Hospital charity funds support oncology and BMT case load but not at the USD 2.2M ATC list. Out-of-pocket plus diaspora-family contribution is the only documented funding pattern; the Pakistani out-of-pocket share at above 50% per World Bank data compounds the affordability gap. The DRAP institutional-import permit step is procedurally straightforward; the binding barrier is funding, not regulation.

Recent regulatory and access news for Casgevy

Vertex Pharmaceuticals continued the global ATC network expansion through 2025 with public additions in the UK (NHS England 7 commissioned centres), Saudi Arabia (KFSHRC, Riyadh), and UAE (Abu Dhabi Stem Cells Center); the live ATC roster is published at casgevy.com. The NHS England national commissioning of Casgevy for transfusion-dependent beta-thalassemia at GBP 1.65 million per patient (reported August 2024) established the first national-payer precedent and is a frequent reference point in cross-border affordability conversations; source NICE TA1031. No DRAP-specific bulletin on Casgevy or gene therapy class has been observed over the last 12 months. The DRAP Federal Cabinet exemption for the import of unregistered drugs in defined public-interest cases (under Section 23 of the Drugs Act 1976, reported mid-2025 at Express Tribune) may apply procedurally to cell-therapy components routed through institutional import.

Operational timeline for a Pakistani Casgevy case

End-to-end, a Pakistani Casgevy case typically runs 16 to 28 weeks from first physician contact to post-infusion discharge from the receiving authorised treatment centre. The phases break down as follows. Phase 1 (weeks 1 to 4): clinical eligibility confirmation by the Pakistani haematology service, genotype documentation, donor-search workup completion to confirm allogeneic HSCT unavailability or inappropriateness, ATC selection and acceptance of the named patient, and family financial scoping. Phase 2 (weeks 4 to 8): documentation packaging, DRAP institutional-import filing where applicable, ATC intake paperwork, State Bank of Pakistan outward-remittance preparation, and travel-and-visa preparation for the patient plus one or two caregivers. Phase 3 (weeks 8 to 12): apheresis collection at the ATC, transition to the manufacturing window, and bridging supportive care including transfusion management for thalassaemia patients or vaso-occlusive crisis management for SCD patients. Phase 4 (weeks 12 to 20): Vertex manufacturing window. Phase 5 (weeks 20 to 24): myeloablative conditioning with busulfan-based regimen, Casgevy infusion, engraftment monitoring, and acute post-infusion care. Phase 6 (weeks 24 to 28 and beyond): post-engraftment monitoring at the ATC with progressive transition to outpatient care, eventual return to Pakistan, and long-term follow-up at the Pakistani referring haematology service.

Documentation set for the case

The composite Casgevy case documentation set includes the prescription naming the specific patient with the Casgevy single-administration plan, the clinical justification letter from the treating Pakistani haematologist covering diagnosis confirmation by genotype, severity assessment (transfusion burden for TDT, vaso-occlusive crisis frequency and end-organ status for SCD), prior therapies and their outcomes, donor-search documentation, and the rationale for autologous gene-edited therapy over allogeneic HSCT; the Pakistani prescriber's PMDC licence verification; the receiving ATC's Vertex-qualified status letter and acceptance of the named patient; the apheresis facility and manufacturing chain-of-custody plan from collection through cryopreservation, transport, and dosing; the institutional import permit application under the Drugs Act 1976 if any component touches Pakistani territory; the financial readiness attestation, since the case is cash-pay or sponsor-funded; and the long-term follow-up commitment, since Vertex requires up to 15 years of follow-up data on every Casgevy patient. Reserve Meds packages the kit, the prescribing haematologist signs the clinical components, and a registered Pakistani importer or the receiving ATC's overseas-coordination office files the import-side documentation.

Operational risk register

Four risks recur across Pakistani gene-therapy cases. The first is funding execution. The USD 2.2 million drug-product line plus the USD 200,000 to USD 500,000 institutional treatment package plus several months of family travel and accommodation puts the case in a USD 2.5 million to USD 3.0 million envelope. The State Bank of Pakistan outward-remittance approval process for sums of this magnitude is the binding execution constraint and is sequenced into the case at the start, not at the manufacturing window. The second is disease progression during the case, particularly for SCD patients with high vaso-occlusive crisis frequency. The bridging plan with the local haematologist is set