BioWorld International Correspondent

BRUSSELS, Belgium - Smaller European biotechnology firms have not responded with enthusiasm to changes in the European Union's system of support for orphan drugs. The European Medicines Agency has changed its instructions to applicants seeking orphan designation, but the modifications do not go far in matching the changes that smaller companies face in using the procedure, which is intended to offer incentives for the development of new treatments for rare conditions.

The Emerging Biopharmaceutical Enterprises grouping in Brussels had asked the commission to "remedy some of the concerns about the current European orphan system." It alleges that the current prevalence calculation requirements can create an obstacle to the development process for smaller firms. Because the burden of proof of the rare prevalence is with the sponsor, support should be provided - for instance, through publishing lists of conditions that already have been deemed not to qualify according to the prevalence criteria.

Translations also are a major burden for smaller firms: "What is the value of developing a translation for the name of a rare indication in all (even small) European languages at this early stage?" it asked. And it had requested that bureaucratic requirements should be reduced, and that access to the advice and financial assistance under the scheme should be streamlined.

The UK's BioIndustry Association also had asked for the new guidelines to take account of the impact on small and medium-sized companies of the increased cost and bureaucracy involved in meeting requirements to achieve orphan drug status.

EBE had urged more flexibility and allowed "documentation from relevant experts," where literature-based descriptions of the disease are not possible, or in official recognition that the mechanism of action of a medicine is not always known at the time of the application for designation. It also had requested additional guidance on the types of preclinical or clinical data that could be considered as sufficient justification for the designation, with specific examples of diseases. And it had called for acknowledgement of the costs of withdrawn applications in the calculation of "potential for return on investment."

But the modified guidance, which has just been published, avoids many of those requests. It clarifies some procedural matters - making clear, for instance, that applications must be made before a request for marketing authorization (except when it concerns a new indication for an already authorized product). It insists that applications for medicines with active substances already marketed in the EU will not be accepted unless the application concerns a new indication.

It also lays down that full justification must be supplied for any application for orphan status, which means demonstration of the life-threatening or debilitating nature of the condition to be treated, or the degree of prevalence of the orphan disease or condition in the EU. To allow the authorities to check the calculated return on investment, details must be provided of all grants and tax incentives, past and future development costs, production and marketing costs, and expected revenues.

The applicant also must provide details of any existing diagnosis, prevention or treatment methods, and arguments as to why they are not considered satisfactory, in order to demonstrate the innovative nature of any medicine for which orphan status is sought. And it is necessary to show justification of significant benefit from the proposed new treatment.

Meanwhile, the European Commission has initiated its threatened study on the prices of orphan medicines in the EU. The biotech industry's fear is that the EU authorities are aiming to find pretexts for premature withdrawal of the 10-year market exclusivity awarded to orphan drugs. A clause in the current legislation allows for withdrawal of orphan status if a product is judged "sufficiently profitable" after five years, but the provision has not yet been used. New definitions of profitability, which is what the study is intended to provide, are unlikely to be in the industry's favor, European companies say, insisting that the process must be handled with prudence.

GM Labeling Threshhold Vexing Environmentalists

The European Commission is planning to allow maize and oilseed rape seed to contain up to 0.3 percent of genetically modified organisms without requiring label information stating that it contains GMOs. The draft decision "establishing minimum thresholds for adventitious or technically unavoidable traces of genetically modified seeds in other products" is likely to be adopted this month. It is a recognition by EU authorities that some accidental contamination of non-GM crops is going to be virtually impossible to avoid - but that should not impose undue burdens on farmers and suppliers.

The proposal already has come under attack from the EU's environmentalist lobby.

"If this decision goes ahead, it could spell the end of freedom of choice for consumers regarding GMOs," said Eric Gall, of Greenpeace Europe.

Marco Schlüter, of IFOAM Europe, an umbrella organization for the organic movement, said, "EU member states, which are supposed to develop national legislation to protect conventional and organic farming from GMO contamination, will be left no room for maneuver."

Arnd Spahn from the European Federation of Food, Agriculture and Tourism trade unions added: "By undermining the GM-free market, the commission will destroy businesses and threaten jobs. Our sector has suffered too much from food scandals in the past to risk going against consumer opinion."

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