Orphan Drug Market, Large Pharmaceutical Companies Advance to Heavy Weights

Recently, when interviewing several CEOs of multinational pharmaceutical companies, the reporter found that in their future development strategies, they all mentioned that more resources will be devoted to the development of rare drug products. The rare drugs known as "orphan drugs" have never been valued by pharmaceutical companies. Today, this "past" is really going to be a thing of the past - very few people dare to ignore the development of "orphan drugs" because they often contain extremely high scientific and technological energy and commercial value. Some "orphan drugs" even have tannins that become "bombshell" drugs (with annual sales of more than $ 1 billion).

Unexpected R&D "hot"

"Orphan drugs" refers to drugs used for the prevention, treatment, and diagnosis of rare diseases. Because rare patients have a small group of patients, there is less market demand, and research and development costs are high, there are very few pharmaceutical companies concerned. Therefore, these drugs are vividly called " Orphan medicine."

However, in recent years, “orphans” have not been isolated, and the development of “orphan drugs” market has been very rapid. According to the report of FiercePHarma, a small number of patient groups can also bring huge market opportunities. Orphan medicine is already one of the hottest areas in the pharmaceutical industry. Reuters research shows that the rapid development of "orphan drugs" will open the next era and create new product groups. In the past 10 years, sales of “orphan drugs” have grown at an average annual rate of 25.8%, which is better than the average annual growth rate of 20.1% of other “orphan drugs”. Reuters experts speculated that the current global market value of "orphan drugs" has reached 50 billion U.S. dollars, and will erode other drug markets. The rapid development of “orphan drugs” is due to the protection of special market exclusive rights, the large investment in research and development, and relatively short time and development costs in clinical research and new drug approval.

Oncology accounts for most of the "orphan drugs." About 40% of "orphan drugs" are used to treat cancer, which also reflects the extraordinary development of genetic research - the identification of specific groups that divide the patient population into groups. The economic analysis of "orphan drugs" is very important. Of the 86 "orphan drugs" analyzed by the Reuters research team, 1/3 can be considered to have grown into "bombshells." "The number of drugs approved during the period 2000-2010, which have grown into "blockbusters" in the market, is stable every year. This shows that the "blockbuster" business model is not out of date, but shows from another perspective. The importance of R&D in the pharmaceutical industry is even more crucial in capturing emerging market opportunities, such as rare diseases,” said one researcher.

Analysts have analyzed and analyzed globally selected rare disease treatment systems and pointed out that the rapid development of "orphan drugs" has not only saved the lives of millions of patients worldwide, but also promoted the evolution of existing drugs. This explains why Biomarin, which specializes in orphan drugs, has a higher market value than other companies in the industry.

According to the annual report issued by the American Pharmaceutical Research and Manufacturers Association (PHRMA) in 2011, there are currently 491 rare drug drugs in the clinical and later research and development state. Interestingly, over 20% of products (eg, Infliximab, Rituximab, Bevacizumab, Everolimus, etc.) were redeveloped as rare drugs. For example, Pfizer, Novartis, Sanofi, GlaxoSmithKline etc. all began to line up in the field of rare medicine.

The World Health Organization has defined rare diseases as diseases that account for 0.65% to 1% of the total population. China has not yet defined them clearly. Patients with a patient population of 20 or less (United States) and patients with an oral status of 5/10,000 or less (Europe) are classified as rare. There are five or six thousand rare diseases recognized by the world, accounting for about one-tenth of human diseases.

Although the total number of patients suffering from rare diseases in the world can be fully recorded with a thin record book, the high-end benchmarks displayed in the field of scientific and technological commerce are obvious: For example, a brain cancer drug was approved in 1998 for rare treatment. The form of adentocellular carcinoma adenoma, and later this drug has been approved for the treatment of other brain cancer diseases. The drug's sales in 2009 reached more than $1 billion. Another example is soliris, the single most expensive drug in the world. The annual cost of treatment is up to 409,500 US dollars to treat a rare disease called paroxysmal nocturnal hemoglobinuria. At present, 8,000 people in the United States suffer from this disease. Last year, sales of soliris was 295 million U.S. dollars.

Among the five or six thousand rare diseases currently identified, the vast majority are medicine-free, and only a small portion of them can be cured. However, the price of this orphan medicine is very expensive. In the case of drugs for the treatment of "Gayse disease", the treatment cost of the disease for one year is about 200,000 US dollars. The small population of patients and the large market opportunities are the most significant features of “orphan drugs”. Gleevec, produced by Novartis, is undoubtedly representative on the road where “orphan drugs” have become “bombshell” drugs. The initial target population of Gleevec was slow-growing type of myeloid cancer, with a population of about 50,000. Currently, about 120,000 patients benefit from the drug's treatment. Gleevec's annual sales are nearly 5 billion U.S. dollars.

The miracle under special care To some extent, the rapid development of the “orphan medicine” market has a lot to do with the special policies it enjoys. In order to encourage the development of drugs for rare diseases, the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have established incentives for pharmaceutical companies to develop drugs for rare diseases, including assistance in the reduction of test tax and the provision of approved franchise rights, etc. . For example, according to the American Rare Drugs Act, a biopharmaceutical company can get a 50% tax credit on clinical development costs, as well as various grants and contracts. The most important of these is the acquisition of exclusive sales rights for seven years, while other drugs are only five years old. Another benefit is that after the expiry of exclusive sales rights, competitors will not flock because the process for producing such drugs is complicated.

In 1993, Japan formally implemented the "Regulatory System for Rare Diseases and Medications." The entire process of Rare-disease Medication Research can enjoy fund grants, tax reductions, priority approvals, extended re-examination of drugs, and national health insurance payments.

The incentive laws and regulations for the R&D and sales of rare drugs in countries around the world have greatly accelerated the listing of rare drugs. In the United States, before the implementation of the Rare Drugs Act in 1983, there were fewer than 10 rare diseases listed. By December 2008, there were 1,951 rare drug drugs registered with the FDA, and 325 rare drugs had been approved for marketing. Before the implementation of the rare drug legislation in 1999, only eight kinds of “orphan drugs” were approved in the European Union. By February 2009, 619 rare disease products had been identified and 47 orphan drugs were approved.

When it comes to medical insurance, "orphan drugs" also have special advantages. Some people find it strange that "orphan drugs" are expensive and why insurance companies do not evade it? Some experts pointed out that a brand of medicine for the mass market can treat millions of patients. Even if each patient uses it for only a few dollars a day, the cumulative amount is surprising and the insurance pressure is huge. For an orphan drug that is used by only 200,000 patients, even if a single dose of treatment costs $1,500 or more, the cost per month is even lower if it is spread over a large number of insured people. To a few cents.

In marketing, the cost of "orphan drugs" is much lower than that of ordinary drugs.

A blue sea that cannot be ignored

China also has "orphan drugs", such as the long-term urine collapse in the treatment of diabetes insipidus, the detoxification of organophosphorus pesticides, such as clotrimyl chloride. In the era of planned economy, China has implemented fixed-point production, special reserves, unified distribution, and expired drug retirement systems for these drugs. However, with the advent of a market economy, drug production and circulation have long been fully market-oriented. The "protection" is also difficult to sustain, and new compensation and incentive mechanisms have not been established, thus inhibiting the enthusiasm of enterprises to produce "orphan drugs." At the same time, due to the uncertainty of the time and population of special diseases and rare diseases, the use time and quantity of “orphan drugs” are also uncertain, and the related companies are unable to bear the risks of expired drug retirement, resulting in “orphan drugs”. Shortages often do not guarantee supply.

It is for these reasons that domestic companies and drug developers are paying more attention to the development of common and frequently-occurring drugs, and the development of “orphan drugs” has been neglected; on the contrary, the “orphan drugs” of foreign pharmaceutical companies have been driven straightforwardly. Many patients with rare diseases can only choose expensive imported drugs, or no drugs are available.

Some experts pointed out that at the same time as the lack of "orphan drugs," our country's clinical knowledge of rare diseases needs to be strengthened. There are still many patients who have been missed and misdiagnosed for a long time. For example, "porcelain doll" (brittle bone disease) may be misdiagnosed as calcium deficiency; "freezing people" (motor neuron disease) or being misdiagnosed as cerebral palsy, but in fact, muscle atrophy caused by motoneuron disease is not like cerebral palsy. Influencing intelligence, like the well-known generation of theoretical physics masters, science master Hawking is a "gradually frozen person."

According to the current situation, experts in the industry pointed out that expanding the indications for new products and releasing new drugs are important means for the development of many rare diseases. 80% of rare diseases are genetic diseases, and genetic biopharmaceuticals are the most widely used category. "Orphan medicine" is regarded as an emerging "blue ocean". Domestic companies should not be absent from their development. The mechanism of some rare diseases is already very clear. There are even patent expired products. Many domestic companies have full strength to research and development.