In January 1983 the United States government passed an Act that has had a profound impact on pharmaceutical development and the individuals whom it was intended to benefit. The Orphan Drug Act (ODA) was lobbied for successfully by the National Organisation for Rare Disorders (NORD) and provides incentives for researching and developing treatments for rare or ‘orphan’ diseases.
Orphan diseases are conditions that affect a very small number of people. The numbers necessary to be considered rare vary from region to region, but a disease with an incidence of 1 out of 1,500 – 2,500 is said to be orphan. Interestingly, this can sometimes include diseases that have a higher prevalence in developing regions, but are rare in industrialised nations. Orphanet (a database of information on rare diseases and orphan drugs) currently lists over 5,600 orphan conditions.
Orphan conditions are frequently genetic and therefore often also affect children, making them more urgent to treat, but also more problematic to develop treatments for (due to the ethical issues involved in running clinical trials on children).
As we’ve discussed previously on this blog, pharmaceutical development is a very complex and expensive process, with the cost of getting a single drug to market estimated to be between $500 million and $1 billion US. If, at the end of that process, there is not adequate demand for the drug then the treatment may not be financially viable, or it must be charged at an extremely high price in order to recoup the costs of research and development, manufacture, clinical trials regulatory applications, etc.
To address this shortcoming, the ODA was enacted to provide incentives and support for drug companies developing treatments for conditions previously ignored due to the lack of a viable return on investment. An Orphan Drug Designation (ODD) is a formal recognition by the regulatory agencies, following review of supporting documentation, that the treatment being developed is a likely therapeutic advancement for a rare medical condition.
Drugs that receive ODD can receive clinical trial assistance, free scientific advice, financial and tax incentives and in some cases a fast track review and approval process.
The ODA, spearheaded by the US and since adopted in different forms by most industrialised nations has seen more than 250 new treatments for rare diseases approved (out of over 1,100 applications), while the decade prior to 1983 saw fewer than ten. Orphan diseases have garnered increasing support and awareness since the ODA, and the benefit kick-started by the ODA continues to this day.
2008 saw the first ‘Rare Disease Day’ in Europe, while a similar national day was observed in the US in February this year. There is also an important piece of legislation currently progressing through the US senate which, if approved, will see substantial funding allocated to the further research of rare disorders.
Clinuvel’s proprietary drug afamelanotide has been granted ODD as a potential treatment for Erythropoietic Protorporphyrias (EPP and CEP) by the EMEA (Europe), FDA (US) and SwissMedic (Switzerland), and solar urticaria (SU) by the EMEA.
For more information on afamelanotide’s regulatory status, see here.
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