[Reporter's View] Concerns about new drugs for rare diseases

Duchenne muscular dystrophy is a rare muscular disorder caused by the lack of dystrophin, primarily affecting boys.

 

The symptoms typically begin before the age of 3 and rapidly deteriorate, eventually leading to the loss of ability to walk before the age of 10 and above.

 

To date, various genetic targeted therapies for Duchenne muscular dystrophy won FDA approval, including U.S.-based Sarepta Therapeutics' 'Amondys 45,' 'Exondys 51,' 'Vyondys 53,' and 'Elevidys,' the American subsidiary of Japan's Nippon Shinyaku NS Pharma's 'Viltepso,' and Italfarmaco's 'Duvyzat.' Since these new drugs have not been introduced to South Korea, so patients with Duchenne muscular dystrophy are relying on steroids.

 

The Korea National Enterprise for Clinical Trials (KoNECT) ranked 'Amondys 45' as the No.1 new drug for domestic entry candidates through the '2021 Report priority ranking international new drugs not yet introduced in South Korea.' However, it has not been introduced to South Korea to date.

 

New drugs have been introduced to various rare diseases, not only Duchenne muscular dystrophy but also metachromatic leukodystrophy and hemophilia, but patient access to these drugs is still limited.

 

Rare diseases have limited treatment options and often patients have limited treatment opportunities.

 

Fortunately, innovative new drugs for rare diseases have emerged in recent years.

 

Introducing new drugs for rare diseases provides not just simply new drugs but opportunities for patients to improve their quality of life substantially.

 

Notably, new drugs such as genetic therapy offer the possibility of fundamental treatment.

 

Patients who have already received treatments give favorable reviews that their quality of life has significantly improved.

 

However, the discussion on whether new drugs can be provided to Korean patients remains a crucial issue.

 

Many pharmaceutical companies face challenges to commercialize new drugs for rare disease due to the lack of treatment infrastructure and limited treatment targets.

 

Eventually, to promote domestic entry of new drugs for diseases with high unmet needs and limited treatments, supportive policies are essential, including government funding and insurance reimbursement.

 

Until now, most evaluations indicate that the existing supports and policies toward patient access to new drugs for rare diseases have been inadequate.

 

Clinical trial opportunities for Korean patients with rare disease must be discussed in depth.

 

If the Korean market is not considered, there is no need for pharmaceutical companies to conduct new drug clinical trials subjecting Korean patients.

 

If clinical trial opportunities involving potential new drug candidates are unavailable to patients without further treatment option, patients have no other resorts.

 

It is of utmost importance to broaden patient access so that pharmaceutical companies would consider the Korean market.

 

It is also essential for Korean pharmaceutical companies to pursue new drug introductions and make R&D investments.

 

Importantly, pharmaceutical companies and the government must collaborate to establish ways to introduce effective new drugs.

 

Fortunately, Korean pharmaceutical companies strive to introduce new drugs for rare diseases.

 

Pharmaceutical companies like Dong-A ST and Boryung are pursuing new drugs in collaboration with KoNECT.

 

Also, many Korean pharmaceutical companies like Handok have been distributing new drugs for rare diseases.

 

No disease is unimportant, from rare diseases, cancer, brain tumors, severe diseases, and chronic diseases.

 

However, patients should not have to travel abroad for domestically unavailable treatments.