Hemophilia patients around the world are getting new access to lifesaving drugs that help their blood to clot properly, thanks to an unusual effort by activists and pharmaceutical companies to not only give away the medication, but ensure it reaches families in need.
Biogen, based in Cambridge, Mass., has teamed up with the World Federation of Hemophilia to create a supply chain that is meant to ensure millions of donated units of the drug traverse the globe in cold storage, make it through customs, and arrive in clinics in Africa, Asia, and Central America without being diverted to the black market.
Biogen and its Stockholm-based partner, Sobi, are donating enough medicine over the next five years to treat tens of thousands of bleeding episodes and give prophylactic treatment to children under 5, which is the standard of care in the US but has rarely been done in developing countries.
A genetic condition in which blood fails to clot, hemophilia can result in excessive bleeding from internal or external injuries. People with hemophilia in developing countries often die before adulthood.
“When your child goes to school, you are afraid until they get home,” said Megan Adediran, a mother of two boys with hemophilia living in Nigeria. “There is the constant fear of them getting injured.”
Doctors are searching for new ways to prevent hemophilia. For now, however, the best treatment is giving patients the clotting factor their bodies lack. That’s been difficult in Nigeria, Adediran said: “There were a lot of hurdles to cross.”
In the developing world, scarcity of medicine is common. And if clinics do have a drug, it is typically the “run-off” — extra that pharmaceutical companies can’t sell commercially.
Clinics often hoard the medication, too, making tough choices as to which patients needed it most. That might mean that a child with the painful, swollen joints common in hemophilia wouldn’t get treated. Instead, the drug would be saved for a life-threatening emergency, such as a patient with a brain bleed. Elective surgeries for hemophilia patients were often put on hold indefinitely.
Before the Biogen donations began, “some of our patients got the factor,” said Dr. Haruna Muktar, who runs a medical center in Kaduna, Nigeria. “Many didn’t.”
But now, Nigeria is one of 16 countries receiving clotting factor through the Biogen-Sobi partnership with the World Federation of Hemophilia, known as WFH. The program there formally kicks off on April 16 with a celebration to mark the opening of 10 new treatment centers.
Other recipients include Kenya, the Philippines, the Dominican Republic, Pakistan, El Salvador, and Indonesia.
In Nigeria, Muktar said that the contributions allow his clinic to do more than treat incoming patients: It can actively reach out to try to diagnose the condition in new patients.
“We saw an average of two or three hemophilia patients a week before the donations,” he said. “Today, we see five to seven patients, and we have a drive to detect more.”
WFH is pressing other pharmaceutical companies to make similar multiyear commitments to ship hemophilia drugs overseas.
WFH already receives support from companies such as Pfizer, Novo Nordisk, and Bayer, whose newest clotting factor formulation received FDA approval last month. Bayer has not joined the multiyear program, however. Instead, the company issued a statement touting its “long-standing commitment to the hemophilia community,” and an upcoming, one-time donation of its clotting drug.
“We’ve received donations of from all of our corporate partners, but the frequency and amount varies from year to year,” said Assad Haffar, humanitarian aid director for Montreal-based WFH. “This program lets us do things in a more predictable way. We are trying to sell the concept of a multiyear agreement.”
