Some patients know there are drugs for their diseases but cannot use them.
In reality, however, most patients find it hardly imaginable to use expensive and uninsured new drugs when treatments for rare, incurable diseases are developed one after another, and their prices keep hitting record-high levels.
Are there no ways for Korean patients to use such unapproved and uninsured new drugs, then? Not necessarily.
In cases of unapproved drugs, ‘compassionate use system’ is recommended
Like the U.S. and Europe, Korea implements a system that provides and supports treatment opportunities for patients with life-threatening diseases without other treatment methods from a humanitarian aspect.
The most typical is the “compassionate use of drugs for clinical trials.”
Drugs for clinical trials are treatments that have not completely proved their safety and efficacy and must be used only for clinical trials. However, it is difficult for patients with life-threatening diseases to wait for the drug’s commercializing process. Therefore, the Ministry of Food and Drug Safety has opened the way for patients and doctors to use such drugs even before its approval.
One of the companies that provide treatment opportunities to patients based on this system is Pfizer Korea.
The company had supplied Lorviqua (lorlatinib), a ROS1/ALK (c-ros oncogene 1 and anaplastic lymphoma kinase) inhibitor developed by Pfizer, ince 2016, five years before the ministry approved its domestic sales.
The number of the ministry approves using Lorviqua for treatment purposes totals about 560.
Lorviqua is a target cancer drug used to treat ALK-positive non-small cell lung cancer (NSCLC). It won domestic approval in July last year. It began to receive insurance benefits for the second or later treatment of patients with progressive disease after taking ALK inhibitors this past September.
ALK mutation is a rare genetic mutation found in only 5 percent of total lung cancer patients. It is difficult to conduct large-scale clinical trials due to the small number of patients and the long period to produce positive results.
Therefore, the U.S. and European regulators approved the drug based only on the results of phase 2 trials, considering the severity of the disease and patients’ treatment access. However, the Korean authorities have put off its approval due to the lack of a conditional approval system here.
There are two ways to use it, however: Either the patient's doctor directly applies to the Ministry of Food and Drug Safety for approval of use for therapeutic purposes, or a pharmaceutical company prepares a usage plan for the use of many patients and applies for approval of use to the ministry.
Both need to win approval from the minister of food and drug safety, and the latter method should also win the IRB (institutional review board) of the medical institutions that conduct the trial.
‘Early access program’ can be considered in pre-reimbursement cases
Patients cannot use hyper-expensive drugs without insurance benefits. However, it takes a long time for regulatory authorities to register them on reimbursement because they must evaluate their effects and create a public consensus.
For this reason, pharmaceutical companies support patient treatment through various methods before their drugs receive insurance benefits. Biogen’s patient support program on Spinraza (nucinersen) is a case in point.
Spinraza is the first genetic therapy for spinal muscular atrophy (SMA) patients. In addition, Biogen has implemented early access program (EAP) for infant SMA patients in 17 European countries since 2017.
Korea also carried out a similar program for local patients by providing entire drug costs since February 2018.
The other is the patient support program, largely implemented via third-party agencies in supporting treatment costs or drug expenses for patients. The third parties are not necessarily nonprofit organizations, but 80-90 percent of patient support programs are now being made through the Korea Blood Diseases and Cancer Association (KBDCA). Pharmaceutical companies cannot intervene in patient selection and other matters in this case.