Scarcely a day goes by that the Clinuvel team is not contacted regarding the latest conference: everything from monitoring to manufacturing to marketing is analysed, discussed and disputed across the globe in conference form. It’s impossible – and unwise – for small companies to try to attend all these forums, but it is equally impossible to work in the space without looking outside the office door: there is much to be learned from what others have done or not done to succeed in an industry fraught with failure.
And indeed there was much to take on at the recent World Orphan Drug Summit, held in Frankfurt last week.
As highlighted late last month in a CEO Blog post, ‘orphan’ drugs – those for rare diseases – are gaining more and more of the pharmaceutical spotlight as big companies move in. Indeed, many of these organisations were represented in Frankfurt and presented interesting case studies, yet it was recurring themes from those working around the industry – in regulation, reimbursement and research – which reinforced the challenges faced by orphan drug developers.
The raw stats behind the orphan drug program are interesting, but don’t tell the full story. It’s estimated that 6,0000-8,000 rare diseases exist, affecting 27-36 million individuals across Europe. Since the introduction of orphan legislation in Europe in 2000, 850 orphan drug designations (ODDs) have been granted by the EMA with 63 orphan products approved for marketing up to 20101. Prices for these products vary, but some genetic therapies for life threatening disorders can cost in excess of €100,000 annually per patient (pricing itself being one of the most contentious issues in the space).
None of these products’ stories could be considered identical, but it was clear that those working in the orphan drug space face similar challenges in getting their drug to market.
The frameworks for drug development were one of the key issues. In short, regulatory and payer frameworks have been developed for indications or diseases which have larger patient populations and/or better understood symptoms or causes. Drugs are evaluated to ensure they are safe and effective for a given patient population. For most ‘mainstream’ drugs this evaluation is able to rely on wide-ranging data or existing medical literature which can help determine the impact of symptoms either in isolation or in comparison with other diseases. Often there are even existing measures for specific diseases or disease categories which can help determine the efficacy of a treatment or comparative treatments.
These factors allow regulators and others to effectively and objectively review the merits of a treatment and recommend on its approval for use. Yet, for orphan indications little, if any, of this information exists or can be easily generated throughout the life of a program. As a result orphan developers must better understand their target indication(s), work more closely with their patients and work harder to educate regulators on the impact of both disease and drug. Framework doesn’t really exist to evaluate whether a drug is safe and effective in an indication when much of this information is lacking, making the development process and regulatory question even harder. The rarer the disease, the more difficult the process.
Fortunately, there are programs underway which help expand upon the orphan drug legislation to improve both evaluation and eventual patient access. Unfortunately, there is yet to be any true consensus at an international level on how best to approach many of these issues with evidence that national interest may sometimes stand in the way of improved international access, even once the regulators give an approval. What was clear from those who have overcome these issues, however, is that no two orphan developers are the same and a flexible, unique and collaborative approach to rigid drug evaluation framework is essential to success in the orphan drug space.
1 Note that some ODDs may be for multiple diseases for a single product, or multiple products for a single disease
“Green leaf of a bio plant in nature” posted to Flickr.com on August 31 2008 by epSos.de <http://www.flickr.com/photos/epsos/3384297473/>