[Reporter’s view] Dilemma of compassionate use

Recently, certain global pharmaceutical companies have decided to discontinue compassionate use of its drugs, which permits patients access to investigational drugs, following the official approval of these drugs in Korea..

 

Consequently, patients without alternative treatment options are left with no choice but to obtain the drugs at a non-reimbursed price, despite the effectiveness of the treatment.

 

The Ministry of Food and Drug Safety (MFDS) permits an ‘approval system for compassionate use of investigational drugs,’ which is intended for patients with a serious or life-threatening disease or condition, particularly in cases where no alternative treatment options is available.

 

The system is designed to allow patients to access therapeutic drugs, often referred to as “off-label” (prior to receiving official approval for prescription) drugs, typically in situations where there are no other treatment options available.

 

However, there are cases where pharmaceutical companies discontinue compassionate use of drugs once the drugs are officially approved or receive expanded indications.

 

Recently, Norvatis announced that they are discontinuing the supply of two drugs, Rafinlar (dabrafenib) and Meqsel (trametinib), on a compassionate basis for patients with BRAF-positive solid tumors excluding lung cancer.

 

The two drugs were granted approval for the treatment of BRAF-mutation positive malignant melanoma and non-small cell lung cancer.

 

Until now, the two drugs were provided on a compassionate basis to patients with BRAF-mutation positive solid tumors that had no comparable or alternative treatment options available.

 

On the 15th of last month, the combination therapy of Rafinlar and Meqsel received an expanded indication for the treatment of unresectable or metastatic BRAF V600 mutation-positive melanoma.

 

Novartis has stated that due to this updated indication, they are no longer able to supply the drugs for compassionate use.

 

In response, some medical professionals have strongly lodged a complaint against Novartis.

 

Novartis has recently extended the supply of the drug for an additional 6 months, but they have not provided any information regarding the supply of the drug after the specified period ends.

 

There are more cases like Novartis’s.

 

Pfizer, for example, tried to discontinue compassionate use of Lorviqua (lorlatinib), which has shown effectiveness in ALK and ROS1 mutation-positive non-small cell lung cancer, following official approval in Korea in 2021.

 

However, in response to the continued requests from medical professionals, Pfizer reversed its decision to discontinue compassionate supply of the medication.

 

Medical professionals argue that it is unethical to stop supplying compassionate use of drugs to patients without alternative treatment options.

 

Since global pharmaceutical companies put emphasis on social responsibility and on serving the community’s best interests, they should continue providing these drugs as a moral obligation.

 

It's not entirely unreasonable from the pharmaceutical company's perspective.

 

Since pharmaceutical companies have provided the medication off-label, it's understandable that once the treatment is officially approved, patients should follow the approved procedures.

 

However, instead of abruptly transitioning to non-reimbursed administration, it might be more appropriate to establish a grace period through mutual agreement.

 

Corporate social responsibility to serve their community doesn't always involve grand gestures.

 

It's the behind-the-scenes efforts that can make a significant impact on fulfilling the needs of the patients.