Orphan diseases, affect not more than 5 in 10000 people, With some 29 million sufferers in the EU;

The EU offers the pharmaceutical industry some incentives to investigate rare illnesses. According to the General Director of LEEM, Philippe Lamoureux, European-backed research into drugs for the treatment of orphan diseases has led to 68 new medicines approved between 2000 and 2011. But there are between 6,000 and 7000 different rare diseases, so these drugs help just a fraction of sufferers.

Where does animal testing fit into the orphan drug equation? It is present in preclinical trials – as is the case with all drugs – but would it be right to ban the use of animals in research when patients have so few treatments to choose from in the first place? The Journal of Animal Ethics proposes that doctors tell patients, or their carers, the role that animals played in the development of their medicines. If sufferers of rare illnesses had access to this information, would they refuse treatment?

Stopping experimentation on animals in the EU probably wouldn’t stop European patients using drugs that have been tested on animals. The problem would simply be relocated, with testing taking place further away, in countries with less stringent regulations. Banning animal testing could also lead to a slowdown in research, as scientists consider alternative means of testing. The question remains: would sufferers of rare diseases be able to wait?

Some believe that not enough is being done to develop orphan drugs. According to work published by three Italian pharmaceutical researchers, a lack of testing on recommended animal species may have affected the investigations of 24 molecules, candidates for treatment of rare diseases. So when it comes to orphan drugs, should there be more, rather than less, research?

Sufferers of orphan diseases already face limited treatment options. Take animal experimentation out of the equation, and drug development options shrink even further.

Given my interest in issues around animal testing, these timely reminders of the burden of disease got me thinking about the contribution that research has made to human health.

Survival rates from breast cancer have been improving for 30 years due in no small part to basic research using animals. Cancer is still to be dreaded however outcomes are much more promising than in the past and anti-cancer treatments are becoming more targeted (and so have fewer side effects).

These days it’s not uncommon to find cancer patients who want to be enrolled in phase III clinical trials so they have a chance of benefitting from the latest innovation medical science has to offer.

They believe new anti-cancer treatments, like chemotherapy, are safe – because they’ve been tested on animals – and they hope the drug will help prolong their lives.

What about non-fatal diseases?

Okay, so cancer is often put forward as a case where the deaths of animals in the lab ultimately helped prevent the deaths of people on oncology wards, albeit not in equal number.

But what about diseases like rheumatoid arthritis (RA)? This is a disease which too often strikes women in their prime. Statistics of woman effected vary slightly but stand at about 75%, compared to 25% of men. Unlike cancer, diagnosis of RA is not received as a death sentence – but it is a prescription for misery, pain, and a dramatic decline in quality of life.

Here we’re talking about a disease which is not immediately life threatening but which can prevent a young mother from playing with her children or heap severe strain on entire families.

Less than a decade ago, a new class of so-called ‘biologic’ drugs – monoclonal antibodies – arrived in doctors’ arsenals. Early work in this field was done using antibodies from mice and rats, and even now some of the human antibodies used in these therapies are produced using transgenic mice.

These drugs changed people’s lives almost overnight. Men and women with RA who could barely move – who couldn’t make a fist let alone drive to work – were given back a large portion of their independence.

‘Something must be done’

When we hear patient stories during awareness days/weeks/months, the visceral reaction is often to say “something should be done about this”.

Indeed it should. But what do we mean when we demand action? That science should find a cure or a treatment to end the suffering? Do we presume that this will involve animal research and, if so, are we okay with that?

So, dear reader, the question to you is:

How do we weigh animal suffering against the burden of human disease?

Is it okay to sacrifice animals to cure cancer? What about arthritis? Or depression? Or restless leg syndrome?

There’s no easy answers – but what’s yours?