Beijing, China: According to an article in a sister publication of Sixth Tone, a woman living in China who has been treated during the past fourteen years for paroxysmal nocturnal hemoglobinuria (PNH) could no longer receive treatment due to the COVID-19 pandemic.
PNH is an extremely rare disease affecting one or two people out of one million. Symptoms include blood clots, eradication of red blood cells, and diminished bone marrow function all pointing to a life-threatening disorder.
For the first ten years after diagnosis, the subject was treated with blood transfusions and hormone therapies, and she remained in stable condition.
However, in 2018 she began ordering a drug called eculizumab (Soliris) that was developed by Alexion specifically for PNH. Although the drug was approved by Chinese authorities that same year, it never appeared on the market for commercial use.
About the 2019 Compassionate Use Policy
The Compassionate Use mechanism is not well known in China. In fact, the woman in this article is the first patient in China to have access to an unapproved medication under China’s 2019 policy.
Her doctor learned of a new drug being tested by Novartis in a Phase 3 trial, but she was not eligible to enroll since she had received prior treatment.
Then doctors at Peking’s Medical College applied for the new drug under the 2019 compassionate use policy. The policy stipulates that if patients have a life-threatening disorder but no effective treatment, as long as there are no violations of ethical principles, the patients may be eligible to receive the drugs for which they apply.
The application process took over two months but finally, the Swiss drugs arrived at the hospital in Beijing.
Hope for Others
The approval raised many questions among caregivers and patients with rare diseases who claim they have never heard about the compassionate use policy. They now have hope that they may also gain access to new medication abroad that has not yet been approved by Chinese authorities.
One person especially interested is Yao Yansuo who has a son with Duchenne muscular dystrophy (DMD). Yao is executive director of Beijing’s Zhiai DMD Support Center. He commented that although certain drugs designed to treat DMD are available abroad, they are not yet on the market in China.
Yao points to his center’s records which show that without adequate medication, children the Center has cared for have since died. Yao explained to Sixth Tone that children who have DMD are unable to walk by the time they are in their teens. They are affected by cardiorespiratory failure causing death between the age of twenty and thirty.
Generic Drugs From South Asia
The pandemic has also left hundreds of thousands of cancer patients in China facing unreliable delivery of the products they have ordered from India. It is estimated that each year about five hundred thousand people in China are diagnosed with liver cancer. The majority will be dependent upon affordable generic drugs from South Asia, the largest producer of generic drugs in the world.
Deliveries of drugs in the past had taken about ten days. Now, not only does it take months for receipt of the products, but the delivery of life-saving affordable drugs has become entirely unpredictable.
Part of the problem lies in the travel restrictions caused by the strained relations between the two countries. So far there have been very limited signs of recovery.
Patients are urged to use caution when purchasing drugs as the counterfeit market is taking hold. Fake generic drugs exist in China and India.
