Previously we have discussed the shifts which take place in the regulatory centers of the world specifically London and Maryland, which present an opportunity to the industry to respond to expressed need. Perhaps the greatest concern of every individual is access to health and quality of life.
This implies a strong directive for drug developers to respond to the identified needs of the patients and patient communities. But amongst those with therapeutic needs are some with more unaddressed medical requirements: patients with ‘orphan’ diseases.
Orphan diseases are rare and frequently untreated diseases. The previously referred shift towards patient driven drug development made a few voices heard and legislators in the European Community and United States started to act on behalf of the population. Here the legislators and regulators provided incentives to drug developers by reducing fees, providing exemptions and protection from competitors.
Alongside drug development, legislation and regulation have been amenable to a patient driven model. And far from proving themselves lumbering bureaucratic behemoths, these organisations (FDA, EMA, US Senate) are facilitating the creation of an unprecedented environment of co-operation, insight and cohesion between the regulatory bodies, the legislative councils, drug developers and the patients. Proving the strength and influence of formal and informal patient organisations worldwide.
The Orphan Drug Act (1983), could be seen as the first such legislative response to acute patient need, and now serves to encourage drug developers to tackle rare diseases by offering incentives in the form of discounts and fast track advice and applications. The fact that the US model for orphan drugs has been adopted and rolled out to most jurisdictions globally is testament to its practical and important role in drug development.
We are now beginning to see further positive benefits from this global roll out. Recently, the FDA and EMA announced the commencement of a programme whereby data from orphan drugs, disorders, filings and applications are shared quickly and openly between the agencies. This simple response to need has further streamlined the process of delivering treatment to those who have a clear, unmet medical need.
And acting to address the importance of rare and untreated conditions is not confined to regulators.
Through consultation with patients, physicians, specialists and patient associations the porphyrias have been recognised as diseases of national importance in the US and funding allocated for their research. Such consideration is not born in a vacuum and is a clear move to respond to patient need
Aside from the benefit of regulatory incentives for developing orphan drugs and the potential to deliver treatment to those in a dire need of it, orphan diseases present a pharmaceutical opportunity.
Rare and untreated conditions are often the most severe and extreme. By researching, understanding and potentially treating them, we gain a deeper insight and uncover knowledge about drugs, molecules, diseases and human biology.
Proving and delivering therapeutic benefit in extreme conditions promotes the very real possibility of applying knowledge, expertise and treatment to more common ailments, while being able to deliver potentially life changing treatment to patients who need it the most.
Drug development and regulatory science will continue to evolve as we learn more about diseases and potential therapeutic molecules. The fundamental drive behind the development of such molecules and therapies, however, may undergo a shift as we enter an era of patient focused drug development.
Image reference:
[...] This post was mentioned on Twitter by Clinuvel News. Clinuvel News said: Orphan drug development legislation and regulation landscape http://bit.ly/dCep0d New post now live on the Clinuvel blog [...]