Orphan Drugs in the USA

Orphan drugs are drugs that are exclusively developed and used for treating rare diseases. Orphan drugs are not researched and developed for widespread use, since by their nature, they are meant only for rare diseases. Since by definition, a rare disease is not likely to have too many patients; orphan drugs are limited in their research, development and eventually, the market.

Situation governing orphan drugs in the USA

Different countries and markets have their own rules for how orphan drugs are developed and marketed. Orphan drugs in the USA have rules designated as to their application. Three major aspects mark out the rules relating to orphan drugs in the USA:

Liberal rules for clinical research

One, there are sufficient leniencies relating to the conduct of the clinical research for the orphan drugs in the USA. The requirement that non- orphan drugs in the USA need to make in relation to sample size does not apply to orphan drugs in the USA. This is natural, considering that fixing a sample size for the clinical research makes no sense, since in many cases, the full number of people suffering from the rare disease, that is, the people for whom the drug is being developed, could be quite nominal. In such instances, it will not be possible for the study to gather a big sample trial size.

Stresses the role of governments

Secondly, research and development of orphan drugs in the USA requires the intervention of the government, not-for-profit organizations that fund such activities, and philanthropy for support. In the case of non-orphan drugs in the USA; there are sufficient incentives for full-scale development and monetization. This is not so in the case of orphan drugs, since as noted, the population needing the drug could be miniscule.

Unencumbered approval process

As an offshoot of these two factors, the FDA has made the approval process of orphan drugs in the USA extremely easy and simple. Again, this is also based on the same logic that governs the entire nature of orphan drugs in the USA: their need in people who do not belong to the general population. To bring about incentives for orphan drugs in the USA; the federal government has passed The Orphan Drug Act.

The Orphan Drug Act

As a means to concretize the role of orphan drugs in the USA; the US administration passed the Orphan Drug Act in 1983. Aimed at encouraging firms to take up development of drugs for products with a very limited market, the Orphan Drug Act has the following features:

• It makes manufacturers of orphan drugs eligible for incentives such as a seven-year exclusivity
• It offers tax rebates of up to half of all their costs spent on research and development, and several other tax incentives for clinical trials of orphan drugs in the USA.

Read More:https://www.linkedin.com/pulse/orphan-drugs-usa-ronald-gardner

Orphan Drugs in the European Union

Rules concerning orphan drugs in the European Union are largely inspired by those concerning orphan drugs in the US. Orphan drugs in the European Union are based on and built on the same premise, namely offering support to pharmaceutical companies that manufacture drugs to treat rare diseases.

Regulations on orphan drugs in the European Union

The European Medicines Evaluation Agency (EMEA) has made regulations for the conduct of orphan drugs in the European Union. The whole set of regulations was built on the need for developing rare drugs and making them available at the most affordable cost and in the quickest possible time for the patient.

January 1, 1995 can be considered the date from which rule concerning orphan drugs in the European Union came into existence across the board in the European Union. Till then, the EU was in the process of synchronizing and unifying the fragmented laws on orphan drugs in the European Union across its many countries. From this date, the rules for orphan drugs in the European Union have been uniform across the bloc.

Regulation n° 141/2000

Regulation n° 141/2000 was the first and most important regulation that the EU passed as part of regulation of orphan drugs in the European Union. Passed by the European Parliament in December 1999; its salient features include the following:

Read More:https://www.linkedin.com/pulse/orphan-drugs-european-union-ronald-gardner?trk=prof-post

Rule relating to orphan drugs in Japan

Orphan drugs are those that are developed purely to treat rare diseases. The nature of orphan drugs is a little piquant: on the one hand, rare diseases affect very few people, but these cannot be ignored. On the other hand, orphan drugs, since they are so few in number, are not taken up usually by profit-driven organizations because they offer very less scope for large-scale production and monetization.

Different countries such as the US and Japan, and blocs such as the European Union have their own unique rules regarding orphan drugs. Rules relating to orphan drugs in Japan too, have their unique and salient features.

A look at the uniqueness of rules relating to orphan drugs in Japan

Orphan drugs in Japan are governed by five incentives:

• Subsidies
• Consultation
• Tax preferences
• Priority review
• Reexamination period review

Briefly, this is how each of these is carried out in practice:

Subsidies:The Japanese government has a fund of close to a billion yen that is used to subsidize orphan drug applicants. This grant, given through the National Institute of Biomedical Innovation (NIBIO), is aimed at helping manufacturers to ease the costs associated with development of orphan drugs in Japan.

Consultation:Under this system, the Japanese government offers priorities for manufacturers of orphan drugs in Japan. Called the Priority Consultation System; this process is offered in two ways:

One, when a sponsor of orphan drugs in Japan approaches the PMDA; the application is never rejected. Clinical data is discussed with each sponsor, at which all the aspects of orphan drugs in Japan are considered. Areas taken up for discussion include the number of patients, the intensity of the disease, the integrity and safety of the data, and so on.

Two, the fee category is also considerably lower for orphan drugs in Japan, with these manufacturers given about 25 percent discount. An application made for developing orphan drugs in Japan is put for consolation with a team of Technical Experts, who are selected from the Office of New Drug Review team, the Office of Cellular and Tissue-based Products Review team, and the Office of Medical Devices Review team. In addition, fee categories are drastically lower for orphan drugs in Japan when they are being developed by small companies or research institutes. These categories have a whopping 90 percent discount.

Tax incentive: Manufacturers involved in developing and manufacturing orphan drugs in Japan are given a 12 percent reduction as tax credits for expenses incurred during the NIBIO subsidy payment period.

Priority review:Manufacturers of orphan drugs in Japan are offered a priority review by the PMDA. While the median standard review time is one year for non-orphan drugs, that for orphan drugs in Japan is nine months.

Reexamination period review: The Reexamination period review for orphan drugs in Japan is also relaxed. It is fixed at eight years for orphan drugs in Japan, while this period is ten years for non-orphan drugs in Japan.

Read More:https://www.linkedin.com/pulse/rule-relating-orphan-drugs-japan-ronald-gardner