How to treat rare diseases "drug price disparity"

  According to a report from China News on August 4, a recent news of "seeking medicine" from the media has attracted attention. According to the article, the infant in Hunan who has just turned 1 year old this year is suffering from a rare disease called spinal muscular atrophy (SMA) and is in urgent need of a special medicine, but “700,000 yuan per bottle”, and the price of the medicine in Australia is “41 US dollars".

  In terms of price alone, there is a big difference. This "seeking medicine" news highlights the difficulty of taking medicine for rare diseases, which is thought-provoking.

  To discuss this topic, we must first clarify that the payment methods of "700,000 yuan" and "41 U.S. dollars" are different-"700,000 yuan" is the self-paid price of domestic patients, and "41 U.S. dollars" is for Australian medical card holders to be included in the local The amount paid after the drug benefit plan. So simple comparison is not scientific.

  From a positive perspective, this "seeking medicine" message reminds us that we must work hard to find solutions to the high prices of rare medicines. We can learn from the experiences and practices of other countries.

  First of all, my country's huge population base has created favorable conditions for drug price negotiations. Some rare-disease drugs are expensive because the number of users is small, and after sharing the research and development costs, the prices are pushed up. In fact, although the incidence of some diseases in newborns is only a few thousandths or even one-tenthousandths, the population base of our country is relatively large, and the total number of sicknesses is not small. Prior to this, many imported anti-cancer drugs that cost tens of thousands or even hundreds of thousands of dollars a bottle have achieved price cuts of more than 90% in the negotiation of volume-for-price. So, whether the price negotiation of rare disease drugs can replicate this model is worth studying.

  Secondly, multiple financing measures and channels work together, and the cumulative effect cannot be underestimated. In recent years, the "1+N multi-party co-payment model" has been widely mentioned-"1" refers to medical insurance, and "N" refers to multiple parties, such as special funds, policy-based commercial insurance, and serious illness. Everyone is gathering firewood and the flames are high, and Australia’s "$41" is based on a special fund. If we combine N kinds of measures together, it will undoubtedly also form a strong synergy, lower prices, and benefit patients.

  In the past few years, my country has significantly accelerated the pace of resolving the difficulty of taking drugs for patients with rare diseases, and has also introduced a number of policies. For example, in May 2018, the five ministries and commissions jointly formulated the "First Batch of Rare Diseases Catalogue"-the rare diseases were first rectified before subsequent protection measures such as being included in the medical insurance coverage; in February 2019, "guarantee 20 million rare diseases "Patient medication" was put on the agenda, and the State Council also decided to grant value-added tax concessions for rare disease drugs. In addition, the Zhejiang model, which focuses on special rescue projects, and the Foshan model, which focuses on commercial insurance, also have their own advantages and are worthy of reference.

  In terms of policy formulation and guarantee system construction for rare disease drugs, my country started late and has many debts, but there is no lack of thinking and planning. It is hoped that relevant parties can promote this work with a stronger sense of urgency and resolve the difficulties of patients with rare diseases in medication as soon as possible.

  Luo Zhihua