Measures for the reimbursement of advanced therapies

Advanced therapies have the potential to cure severe diseases…perception of' high price' needs to be reconsidered

The existing ICER threshold cannot adequately assess the value of new drugs … flexible application is necessary

The release of the CAR-T treatment ‘Kymriah (tisagenlecleucel)’ in Korea was met with both anticipation and concerns.

 

The drug itself is undeniably an innovative drug that can offer new opportunities to blood cancer patients with short life expectancy.

 

However, the challenge lies in how this innovative new drug that costs 500 million won per dose should be listed for reimbursement in Korea’s healthcare system.

 

Clearly, these advanced drugs cannot be evaluated under the same standards as existing drugs.

 

The development trend of new drugs has already shifted towards the treatment of severe or rare diseases, and gene and cell therapies that combine new science and technology occupy a large proportion of development.

 

Therefore, a new model that may reasonably evaluate the price of ultra-expensive drugs is necessary to accommodate the changing environment.

 

Then, what limitations and changes has the industry seen and desired in Korea’s reimbursement environment?

 

To hear the voices in the field, Dailypharm held its 41st Pharmaceutical Industry Future Forum, "Finding the correct solution for the ultra-expensive drug listing system," at its Moonjeong-dong office.

 

Dr.

 

Hyung-Ki Lee, Professor of Clinical Pharmacology and Therapeutics at the Seoul National University Hospital, chaired the event.

 

Also, various industry and government officials including Kyung-Ho Choi, Deputy Director of the Division of Pharmaceutical Benefits at the Ministry of Health and Welfare; Min-Young Kim, Director at Korean Research-based Pharma Industry Association (KRPIA); Hyeon-Seok Na, Senior Manager at JW Pharmaceutical Corp.; and Jae-Ho Jeong, Department Head at Novartis Korea attended the event to share their views on the matter.

 

(from the left) Dr.

 

Hyung-Ki Lee, Professor of Clinical Pharmacology and Therapeutics at the Seoul National University Hospital; Kyung-Ho Choi, Deputy Director of the Division of Pharmaceutical Benefits at the Ministry of Health and Welfare; Min-Young Kim, Director at Korean Research-based Pharma Industry Association (KRPIA); Hyeon-Seok Na, Senior Manager at JW Pharmaceutical Corp.; and Jae-Ho Jeong, Department Head at Novartis Korea attended Dailypharm’s 42nd Future Forum.

◆'Advanced therapies that cost hundreds of millions of won may deliver value beyond their cost = Kymriah was tagged as an ‘ultra-expensive' drug as its single injection costs 500 million won.

 

However, Jae-Ho Jeong, Department Head at Novartis Korea, expressed his opposition to the view.

 

Unlike conventional drugs that should be taken annually or on a regular cycle, one can expect a full cure with a single injection of Kymriah, which ultimately would reduce the total cost invested in treatment.

 

“We need to consider whether Kymriah is really an ultra-expensive drug from the financial aspect,“ said Department Head Jae-Ho Jeong.

 

“Other orphan drugs that cost 400–500 million won per year are also listed as reimbursement.

 

The only difference is, with Kymriah, one can expect to reach complete remission with a single injection.” He added, “The value of Kymriah lies in that it can provide the return to daily lives for patients who were non-responsive to existing treatments and therefore left with only several months of to live at most.

 

We need to reconsider drawing the line just because a drug is expensive.” In fact, Kymriah achieved complete remission (CR) in 82% of the young adult and pediatric patients with acute lymphoblastic leukemia (pALL) within 3 months of its administration.

 

The patients were relapsed and refractory patients with a life expectancy of only 6 months.

 

Emily, a pediatric patient who participated in the first trial of Kymriah, is currently living a normal life after receiving Kymriah 9 years ago.

 

Like Kymriah, advanced therapies such as CAR-T cell therapies and gene replacement therapies allow patients to dream of the long-awaited ‘cure.’ ‘Zolgensma,’ a gene replacement therapy, can cure the rare disease spinal muscular atrophy (SMA) with a single dose.

 

◆The need for a personalized reimbursement system = Rather than introducing a completely new system, industry experts have suggested implementing measures that may allow for more flexible use of the existing system, in other words, a personalized reimbursement system.

 

KRPIA director Min-Young Kim referred to the U.K.

 

as an example.

 

For rare diseases that are chronic, severely disabling, and require life-long treatment, the U.K.’s Highly Specialised Technologies (HST) evaluation system weighs the quality-adjusted life-year (QALY) gain and allows a QALY index of 3 if the gain is 30 QALY or more.

 

Also, the Patient Access Scheme (PAS) and Confidential Commercial Arrangement (CCA) implements a kind of risk-sharing system for drugs that have not been able to receive an ultimate value judgment from NICE or drugs with a large difference in ICER due to different health benefits per drug or indication.

 

Also, a Cancer Drug Fund (CDF) system provides funding for oncology drugs that are not recommended by NICE for reasons of clinical uncertainty during the drug's data collection period “Like the U.K., Korea also needs segmentized, personalized policies so as to provide the opportunity for a full cure to patients with severe rare and incurable diseases,” said Kim.

 

He continued, “In terms of patient accessibility, we may consider using systems like the ‘pre-listing post-evaluation' system.

 

Financial uncertainty can be resolved by collecting information on the use amount based on real-world data during the pre-listed period, and then evaluating once the period is over.

 

Also, customized systems that price drugs by indication or the trade-off system may also be used.”