Orphan designation of new medicines could help in fight against antimicrobial resistance, says expert

Out-Law Analysis | 22 Sep 2016 | 9:53 am | 2 min. read

FOCUS: Pharmaceutical companies could be incentivised to develop new drugs to fight antimicrobial resistance (AMR) if the medicines they produce are awarded with an orphan designation.

AMR is a growing problem which governments and scientists around the world are recognising. Many bacterial diseases are evolving in a way which makes them resistant to existing medicines. If unchecked, this development will pose a health risk to the global population.

The problem is so significant that UN countries have newly signed a landmark declaration on AMR (4-page / 453KB PDF) pledging action. The UK earlier this month outlined steps it intends to take.

Developing new drugs is expensive. The costs involved in bringing drugs to market, from carrying out clinical trials, to meeting regulatory requirements, can be prohibitive. The economic case for developing those drugs can be undermined if the market for those drugs is fairly small. Often only a relatively small proportion of the population require access to new treatments for rare strains of diseases showing AMR.

Governments and regulators can help incentivise the development of these life-saving treatments by making it easier for new antimicrobials to obtain orphan designation.

What is orphan designation?

Orphan designation is a form of classification given to certain types of medicine as a means of incentivising their development.

In the EU, a medicine is given an orphan designation if it meets set criteria.

Firstly, the medicine must be intended for the treatment, prevention or diagnosis of a life threatening or chronically debilitating disease.

The condition to be treated also must not be prevalent in more than five of every 10,000 people or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development.

Lastly, to satisfy the criteria for orphan designation, there must be no satisfactory method of diagnosis, prevention or treatment of the condition concerned, or, where the drug had already been developed, the medicine must be of significant benefit to those affected by the condition.

Drugs with an orphan designation can take advantage of a number of benefits.

These benefits can include extended market exclusivity, meaning manufacturers of those drugs will not face competition from developers of copycat products even if they do not own a patent for those medicines.

Orphan designation can also help manufacturers navigate regulatory requirements more easily. The European Medicines Agency supports drugs with orphan designation through the regulatory and authorisation process.

With orphan designation, manufacturers can also benefit from subsidies towards the cost of clinical trials, and the very fact drugs companies are developing treatments that win orphan designation can help those businesses improve their profile and strengthen their brand.

Incentivising new treatments

The new UN declaration recognises that the onus cannot be put on pharmaceutical companies to foot the cost of developing new antimicrobials alone.

The declaration specifically envisages "innovative public private partnerships and incentives and funding initiatives" in the context of combatting AMR.

The UK government's recently released response (33-page / 782KB PDF) to a review it commissioned into AMR also touched on incentives for development of antimicrobial drugs.

The review, led by Lord Jim O'Neill, recommended that a global system of "market entry rewards" should be created to encourage pharmaceutical companies to invest in the development of new antimicrobial drugs.

In its response, the UK government said it is "leading discussions within the global finance and health community to create an innovative and sustainable global system that rewards companies that develop new, successful antibiotics and makes them available to all who need them". It said it is "committed to finding a successful solution".

One option the O'Neill review recommended, and which the UK government suggested it would discuss further at international level, was imposing a "small levy on the pharmaceutical sector, as one of the options to raise funding for the market entry rewards for new antibiotics".

Making more of the orphan designation system is another route that offers incentives. Some AMR diseases may fit the orphan disease criteria. Given the threat of AMR, this should be explored further by governments, regulators and pharmaceutical companies.

Asawari Churi is a trainee patent attorney at Pinsent Masons, the law firm behind Out-Law.com and a former research scientist in molecular biology.