The Development of Orphan Vaccines: An Industry Perspective.
The development of vaccines (Orphan Vaccines) against rare emerging infectious diseases is hampered by many disincentives. On the one hand, there is the humanitarian need to develop niche (orphan) vaccines for rare diseases not preventable or curable by other means. On the other hand, the development of orphan vaccines is a precarious venture for any pharmaceutical company planning to market them because, by their very nature, the money that has to be invested may not be recovered by the sales of the product…
In fact, most pharmaceutical companies, in the face of growing in-house expenditures associated with research and development projects in a complex legal and regulatory environment, prioritize their projects and streamline their product portfolio.
Therefore, there is legislation, both in the U.S. and Europe, which provides financial and practical incentives for the research and development of drugs to treat rare diseases.
Other key factors in overcoming the complexity of orphan status with its inherently and financially unfavourable limited need for vaccine include updated epidemiologic information from experts in the field of emerging diseases; increased disease awareness among health professionals, patients, and the general public; a list of priority vaccines; and the emergence of a dedicated organization with strong leadership able to document the long-term pharmacoeconomic viability of orphan products.
Other obstacles to the development of orphan vaccines are cost, patent protection, and specific developing country issues.
Cost: contrary to popular opinion, the number of sales of vaccines is not directly linked to the extent of the epidemic (number of subjects that may be immunised), but rather to the cost of the vaccine dose. The price of the vaccine must be calculated by minimising the research and development, production, marketing and distribution costs in order to break into a limited market.
A tiered pricing strategy has been endorsed by the World Health Organisation (WHO), in which high-cost but low-volume vaccines in industrialised countries could subsidize the low cost and larger volume of sales in developing countries. Unfortunately, this may not be feasible if the quantity of vaccines needed in developing countries is low.
Patent Protection: Development of new drugs relies heavily on the respect of intellectual property rights, represented by the patent, which reassures investors that drugs will provide a fair return on invested funds. The lack of patent protection or legal framework for intellectual property rights in some developing countries prevents the long-term viability of a vaccine.
Developing Countries: It is difficult to develop new vaccines in these countries, because the optimal use of existing vaccines, considered as a priority, first need to be ensured.
Within the framework of WHO, the Children’s Vaccine Initiative (CVI), set the development of vaccines with commercial prospects as a priority. This cost-oriented definition reflects the difficulty of developing drugs for tropical diseases, even the most prevalent ones such as malaria.
Finally, other non-economic factors could justify an industry’s decision to develop and market an orphan vaccine, such as the desire to enhance the company’s ethical profile, by fulfilling a medical or social need, the capacity to develop, product and market a drug, or a larger company strategy (e.g. extension of a product range).