Islamabad: Thalassemia is no longer a disease of the developed world but a growing public health crisis in Pakistan, where between 6,000 and 9,000 new cases are reported every year, and the country must now find its own solutions for prevention, treatment, and management, senior health officials said on Wednesday.
Speaking at the National Consultation on Repositioning Drugs in the Management of Transfusion-Dependent Thalassemia, Drug Regulatory Authority of Pakistan (DRAP) Chief Executive Officer Dr. Obaidullah said Thalassemia is a problem of low- and middle-income countries (LMICs) like Pakistan, and its solution must come from within the country, from our own physicians and pediatric hematologists who have decades of experience treating these patients but are working in isolation.
The dialogue was organized by the Health Services Academy (HSA) in collaboration with the Blood and Bone Marrow Transplant Society of Pakistan (BBMT) and other national and international partners, including the Thalassemia International Federation (TIF) and the World Health Organization (WHO).
Offering the Central Research Fund (CRF) for research on thalassemia prevention and treatment, he urged clinicians and scientists to develop collaborative, high-quality proposals that meet international standards. “DRAP is ready to support such efforts, but we need joint, coordinated research instead of fragmented publications that make little impact,” he said.
Acknowledging that off-label use of drugs such as thalidomide and hydroxyurea has shown promising results in Pakistan and abroad, he stressed the need for “rigorous, evidence-based studies to confirm their safety and effectiveness in Pakistani patients.” He also called on physicians using these drugs to report any adverse reactions to DRAP to ensure drug and patient safety.
“The onus of off-label use of drugs lies with physicians, so they must be extremely careful and cautious,” he warned. “We cannot rely on anecdotal evidence. Evidence-based research and shared reporting are essential to formalize treatments and safeguard patients,” he said, adding that preventing new births through screening and public awareness must remain a top priority.
Renowned pediatric hematologist and bone marrow transplant surgeon Dr. Saqib Ansari said his late mentor, Prof. Tahir Shamsi, had encouraged him to research the off-label use of thalidomide and hydroxyurea 25 years ago. “Our research shows that about 40 to 50 percent of thalassemia patients can live without blood transfusions when treated with these drugs, either individually or in combination,” he said.
He noted that bone marrow transplant remains the gold standard treatment, though it is not suitable for all due to cost, limited donor availability, and clinical risks. “Over 100,000 Pakistanis are living with thalassemia, and thousands more are born each year. Many depend on repeated transfusions that expose them to infections such as hepatitis B and C,” he said. “It’s time to adopt unified, evidence-based national guidelines so these therapies can benefit more patients safely.”
Sharing his story, Raja Adnan, an adult thalassemia patient, said he had not required a blood transfusion for 13 years due to supervised use of thalidomide. “This medicine changed my life. I appeal to doctors to develop national guidelines so others can also benefit safely,” he said.
Event co-organizer and WHO Blood Safety Advisor Prof. Hasan Abbas Zaheer said the consultation aimed to consolidate local and international evidence on repositioned therapies and to develop standardized treatment protocols for Pakistan. “We need structured national collaboration to move from informal, individual practices to formal, regulated, and evidence-based care,” he said.
Consultant hematologist Prof. Samina Amanat said countries such as India and Bangladesh had already developed national guidelines for repositioned therapies and were seeing positive outcomes. “Pakistan must move in the same direction through scientific collaboration and consensus,” she said.
In a message shared with participants, HSA Vice Chancellor Prof. Shahzad Ali Khan said the academy would support the development of national guidelines and training programs based on expert recommendations. “Pakistan needs a unified scientific front to manage and ultimately eliminate thalassemia. The Health Services Academy will facilitate this process,” he said.
The consultation, attended by leading hematologists, pediatricians, researchers, regulators, and patient representatives, marked a significant step toward developing national clinical guidelines and protocols for managing transfusion-dependent thalassemia in Pakistan.
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