발행일_2014.3.24발행처_한국보건산업진흥원 발행인_정기택

“주요국의 신약재창출(Drug Repositioning)동향과 전망”

- 전세계적으로 신약 개발 환경이 변화하면서 신약재창출은 제약사들에게 신약 개발의 리스크를 줄일 수 있는 새로운 사업 전략으로 역할하는 한편,신약 개발을 앞당겨 의료 서비스에 대한 수요를 충족할 수 있는 방법이 되고 있음.
- 신약개발 여건이 충분하지 못한 우리나라에서 글로벌 시장에서 경쟁할 수 있는 신약을 개발하는 전략 중 하나가 신약재창출임이 입증되고 있기 때문에 신약 재창출의 성과를 거두기 위해서는 정부,제약사, 학계 및 연구기관들의 다양하고 중층적인 노력과 지원이 필수적인 시점이라고 할 수 있음.

“1975년부터 1995년 사이에 시장에 출시된 1393개의 신화학 물질 중에서 열대성 질병 및 결핵과 관련된 것은 16개뿐이다”는 연구결과를 실은  2002년 6월 22일자 <랜셋(Lancet)>의 article 초록입니다.

그 아래 article은 2008년 11월 6일자 의 열대성 질병 및 결핵 등 연구에서 간과된 질병에 대한 약물 개발에 대해 미 FDA가 바우저 제도를 도입한 것에 대한 리뷰 논문의 전문과 파일입니다.
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Drug development for neglected diseases: a deficient market and a public-health policy failure.

 출처 : Lancet. 2002 Jun 22;359(9324):2188-94.

Trouiller P, Olliaro P, Torreele E, Orbinski J, Laing R, Ford N.

Centre Hospitalier Universitaire, BP 217, 38043 Grenoble cedex 9, France. PTrouiller@chu-grenoble.fr

There is a lack of effective, safe, and affordable pharmaceuticals to control infectious diseases that cause high mortality and morbidity among poor people in the developing world. We analysed outcomes of pharmaceutical research and development over the past 25 years, and reviewed current public and private initiatives aimed at correcting the imbalance in research and development that leaves diseases that occur predominantly in the developing world largely unaddressed. We compiled data by searches of Medline and databases of the US Food and Drug Administration and the European Agency for the Evaluation of Medicinal Products, and reviewed current public and private initiatives through an analysis of recently published studies. We found that, of 1393 new chemical entities marketed between 1975 and 1999, only 16 were for tropical diseases and tuberculosis. There is a 13-fold greater chance of a drug being brought to market for central-nervous-system disorders or cancer than for a neglected disease. The pharmaceutical industry argues that research and development is too costly and risky to invest in low-return neglected diseases, and public and private initiatives have tried to overcome this market limitation through incentive packages and public-private partnerships. The lack of drug research and development for “non-profitable” infectious diseases will require new strategies. No sustainable solution will result for diseases that predominantly affect poor people in the South without the establishment of an international pharmaceutical policy for all neglected diseases. Private-sector research obligations should be explored, and a public-sector not-for-profit research and development capacity promoted.

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Drug Development for Neglected Diseases —The Trouble with FDA Review Vouchers

Aaron S. Kesselheim, M.D., J.D., M.P.H.

출처 : NEJM  Volume 359:1981-1983		November 6, 2008		Number 19

content.nejm.org/cgi/content/full/359/19/1981

September 2008 marked the beginning of a new federal program intended to promote the
development of pharmaceutical products for so-called neglected diseases — infectious diseases that disproportionately affect poor populations in developing countries.

Implemented by the Food and Drug Administration (FDA) Amendments Act of 2007, this program will give the sponsor of a drug for a tropical disease a “voucher” entitling the company to expedited FDA review of a new drug application for any other product it makes.1

The need to encourage additional research in this field is clear. Diseases such as  tuberculosis, malaria, leishmaniasis, and trypanosomiasis affect millions of people each year, but these people live primarily in resourcepoor settings with underdeveloped health care systems. As a result, the for-profit pharmaceutical industry has invested little in treatments for these conditions.

One study found that of the 1393 new chemical entities marketed between 1975 and 1999,
only 16 were for such diseases.2 The new program links the development of drugs targeting
tropical diseases to accelerated approval of a company’s other, more profitable drugs for conditions prevalent in wealthier countries. A voucher obtained after the approval of a drug for a tropical disease can be used to require accelerated regulatory review (in 6 months or less) of a cholesterol-lowering drug or an antidepressant, for example, that the sponsor might sell in the United States for thousands of dollars per year of treatment. According to the arrangement’s proponents, vouchers could speed up FDA evaluation time by an average of 12 months, providing domestic patients with more rapid access to the latter types of drugs.3 A voucher could be worth more than $300 million, thanks to the earlier period of market exclusivity afforded by decreasing the time a drug spends in FDA review.

As enacted, however, priorityreview vouchers represent an inefficient and potentially dangerous way of encouraging research into tropical diseases. It is inefficient because the program does not directly connect the incentive with the innovation. Large pharmaceutical companies traditionally have not conducted effective research programs on tropical diseases.

These manufacturers will be unlikely to start such a program merely because of the prospect of earning a voucher some years in the future, since the voucher’s value depends on the success of potential “blockbuster” drugs that are currently in their pipelines, which is far from assured. In fact, tropicaldisease research is predominantly conducted by small pharmaceutical companies with limited drug portfolios. Such companies will often be unable to use their vouchers, although the law permits voucher rights to be sold to a large manufacturer. Relying on these sorts of transactions to spur innovation is speculative as well, and the deals between small and large pharmaceutical companies affecting agents of great
importance to global health will lack transparency. Such deals may include other payments or exchanges of intellectual property that raise the cost or restrict the future availability of the products. 

Another source of inefficiency is that a voucher’s value will bear no relation to the usefulness of the drug whose development it is intended to reward. For example, the law stipulates that no voucher will be earned for a product whose “active ingredient” was previously approved. As a result, an effective novel antimalarial