Patients just want to be cured

Date: 08/05/1997

IN THE last 12 months, members of patient support groups - under the aegis of the European Alliance of Genetic Support Groups (EAGS) - have been a common sight in the European Parliament and the offices of the Commission.

Representatives of patient support groups from the UK, the Netherlands, Denmark, Germany, France and almost every other country in the EU have been in touch with their elected MEPs to educate and inform them of their needs - and about the hope that recent scientific advances in the understanding of human genetics have offered for the development of cures for many currently incurable diseases which blight families and span generations.

For individuals and families trying to cope with the needs of a close relative suffering from a disabling genetic disorder, European legislation can seem very remote from the day-to-day pressures.

If you are trying to ensure that a young child with cystic fibrosis has had his or her physiotherapy and taken all the medication necessary to stay well, whilst at the same time meeting the needs of other family members and arguing with your local services department for more support, even thinking about intellectual property - much less getting actively involved with an EU directive on it - can come a long way down the priority list.

So why has the draft Directive on the Legal Protection of Biotechnological Inventions got patients beating a path to the European Parliament's door? Why is it that this legislation has stirred genetic support groups into committing time, energy and scarce resources to try to ensure that the directive is adopted, and sooner rather than later?

And why have they made such an impact on a debate which has been characterised hitherto by antagonism between a noisy environmental lobby with an anti-science agenda and commercial interests insufficiently adept at presenting their case to ensure their arguments carry the day?

For patients with genetic diseases, the idea of becoming a genetically-modified organism (GMO) is not a threat but a goal. Genetic research is beginning to reveal that this is no longer a distant dream - at least for some.

Gene therapy and other forms of genetic medicine will lift the shadow of fatal diseases being transmitted from generation to generation, enabling children currently facing a greatly reduced life expectancy to enjoy an existence that most of the rest of us can take for granted.

The immediacy and the relevance of this need, and of the moral imperative to do what can be done to meet it, have created a recognition amongst legislators that they have a responsibility this time to get the directive right, ensuring European scientific research is supported by investment and developed into medicines available to all who need them.

Most patients are not fundamentally concerned whether the cures they hope for come through public or private sector spending, or are brought by aliens from outer space. They just want the cure.

Patient support groups, consulting their members as they see fit, have taken a view that the vast sums of money necessary to enable the promise of genetic medicine to be realised (about 300 million ecu per individual product) are unlikely to come from the public sector in a Europe where nations are struggling to meet the convergence criteria for economic and monetary union.

Alien space craft seem an unacceptably risky bet too, so the most likely route to success will be through encouraging the private sector to invest in biotechnology and genetic medicines in the hope of making a profit (as well as of alleviating intractable diseases and improving human health and well-being).

And if that profit is seen to be too great, then legislators might like to consider a hypothecated tax, to be used to improve services for those whom research has yet to help.

Although scientific knowledge is in itself morally neutral, the applications that this knowledge makes possible are clearly not and many have the potential for abuse, as well as a possibility of delivering dramatic benefits for mankind - not just in the health arena, but also in terms of our ability to improve food supplies and tackle environmental threats.

Patient support groups, in lobbying for progress, are not prepared to ignore the ethical issues raised by the new genetics and are arguing for the creation of a new body, accountable to the European Parliament as well as the Commission, to establish the ethical framework within which biotechnology can flourish whilst at the same time preventing unethical developments and abuses of knowledge.

Clearly, it is not enough to monitor the outcome of research and decide after the event whether or not it is ethically acceptable. To maintain public confidence, anybody must be capable of reviewing trends in research and offering guidance as to whether or not the outcome is likely to prove acceptable.

Any ethical authority must be adequately resourced in order to undertake its work promptly and effectively, and have the capacity to be proactive.

It must not create bureaucratic or other stumbling blocks which will impede beneficial progress or dissuade investment. It must also be in a position to respect academic and commercial confidentiality where appropriate, and avoid compromising research and development. At the same time, it must be transparent, open and rigorous.

Although it would not itself have authority to regulate, it is a sine qua non that the opinions produced by such a body would be regarded as a prima-facie case for control, and that those regulating the introduction and application of biotechnological inventions in society would have to justify any decision to disregard an opinion that the proposed development would be likely to be unethical.

On this body there should be academics, clinical geneticists, ethicists and those with religious authority, patients, lawyers, and representatives of European institutions, industry and commerce, consumers and environmentalists. Members would be appointed because of their disinterested expertise and their external credibility.

Possible terms of reference for such a body might be:
to investigate trends in the emerging science of human genetics to establish if these are likely to create new ethical dilemmas and to clarify whether they are appropriate;
develop consensus about the possible impact of such ethical issues;
report to the European Parliament, the Council of Ministers and the Commission about the possible adoption of regulations appropriate to preventing unethical applications of genetic technology;
publish reports on opinions resulting from the deliberations of the institutions, and to oversee the organisation of conferences and other events to publicise them;
produce reports for debate in the European Parliament and Council describing its work in progress and work completed.

Failure to recognise and respond to legitimate anxieties about the responsibility for abuses that scientific advance could create will undermine public confidence and possibly create a backlash against progress in the search for cures for many currently fatal diseases. Equally, overreaction and pandering to technophobia and ill-informed anti-science propaganda will do no one any favours either.

Creating a regime which seeks to eliminate any possibility of abuse will result in a framework so rigid that beneficial uses of new knowledge will be strangled too.

This would not only blight the hopes of patients and their families, but would also damage European industry, eliminate jobs and increase health-care costs as potentially curable conditions remain incurable.

The fall-out from such a failure would irreparably damage the credibility of the European institutions and the reputations of those who work in them.

Alastair Kent is president of the European Alliance of Genetic Support Groups (EAGS), the European umbrella group for voluntary organisations, charities and support groups for those affected by genetic disorders. He has been director of the Genetic Interest Group (the British national alliance) since 1993.