Pharma firm holds the key to costly trial therapy for Hong Kong boy with rare disease

PUBLISHED : Thursday, 14 September, 2017, 4:59pm
UPDATED : Thursday, 14 September, 2017, 10:06pm

I refer to the report on the young child waiting for subsidised access to an expensive drug to treat his rare condition (“Boy with rare disease suffers as drug cost debated”, September 4).

The parents of the seven-year-old child, who is suffering from MPS (Mucopolysaccharidosis) Type IV A, are appealing for the Hospital Authority to support his ultra-expensive drug treatment. The treatment is an enzyme replacement therapy with Elosulfase alfa, a drug registered in Hong Kong in 2015 for treating MPS IV A. The annual treatment cost ranges from

HK$2.7 million to HK$9 million per patient. As in most advanced countries, there are patients here suffering from uncommon diseases which cannot be covered by conventional therapies.

With a designated recurrent government funding of

­HK$75 million, the authority is providing enzyme replacement therapy to 22 patients with six types of lysosomal storage disorder, within the highly subsidised standard fees and charges.

It is now evaluating the safety, efficacy and cost-effectiveness of Elosulfase alfa, following an evidence-based approach.

The Hospital Authority is ready to consider trial drug therapy if BioMarin Pharma can agree on a compassionate arrangement

One important consideration is international practices. The authority has reviewed the reimbursement status of the drug in various countries. It is rejected for reimbursement by public funds in Scotland and is not listed in Taiwan’s National Health Insurance Programme. In England and Australia, confidential risk-sharing arrangements have been reached with the drug company for provision of this drug for MPS IV A patients.

The authority is ready to consider trial drug therapy for this child if the pharmaceutical company, BioMarin Pharmaceutical, can agree on a compassionate drug arrangement.

It is not uncommon for drug companies worldwide to offer compassionate programmes with long-term drug arrangement for individual patients. Some even offer free access to drugs for specific patients, with lifelong coverage.

The authority believes establishing a long-term drug arrangement agreement with the drug company is a necessary precondition for starting drug therapy. The reason is that if Elosulfase alfa is proved to be of clinical benefit to the patient, he would need to undergo long-term treatment with this drug.

Therefore, the offer of short-term, limited duration drug arrangement would not benefit the patient.

The authority hopes the drug firm will give favourable considerations for the benefit of the MPS IV A patient, rather than putting profit as the foremost objective. Upon reaching an agreement with the company, the authority could start drug treatment for the patient within months.

Dr W. L. Cheung, director (cluster services), Hospital Authority