Though a single-dose gene therapy for treating spinal muscular atrophy (SMA) has already been brought to the market, the access to the new drug is hindered by its expensive price. Prof Hsiao Fei-Yuan, the Director of the Graduate Institute of Clinical Pharmacy of the NTU and a member of the Pharmaceutical Benefit and Reimbursement Scheme Joint committee meeting, reckoned that, under the current healthcare and regulatory systems, the emergence of innovative gene therapies will continue to pose a major challenge to the NHI.

Prof Hsiao expressed that empirical data generated from pharmacoeconomic studies provide valuable information to support the decision making process concerning new medical technology and new drugs.  Pharmacoeconomics is about using the healthcare resources efficiently.    The key point is to evaluate whether spending more on new treatments would provide better clinical benefits in comparison with existing treatments.

Taking new cancer drugs for example, pharmacoeconomic studies calculate how much the extra expenses would be when replacing the existing treatment with a new drug in order to prolong a patient’s life by one year.  In the case of drugs for children with rare diseases, the estimation models have to be modified to take account of the benefits of longer healthy lifespans.

This one-course gene therapy for SMA is expensive; however, it will keep children from receiving injections for the rest of their lives.   The new drug could be cost-effective when spreading the treatment cost over a patient’s lifetime and taking into account their long-term health benefits.  This argument is supported by many foreign academic literatures.

The NHI financial problems have to be addressed.  The authority should thoroughly examine the situations and formulate corresponding measures without crowding out the access to treatments for rare diseases.

 【2022-05-24 / United Daily News】