Treatments intended for rare diseases may not be profitable for you to develop under typical circumstances.
To encourage the development of such vital medicines, regulatory agencies like the European Medicines Agency (EMA) offer orphan drug designation (ODD). This designation provides a range of benefits to incentivise the research and development of treatments for rare diseases. But what does ‘orphan drug designation’ mean?
Orphan drug designation is a status granted by regulatory agencies to drugs and biologics that are developed to treat rare diseases — often referred to as ‘orphan diseases.’ In the European Union, a disease is classified as rare if it affects fewer than 5 in 10,000 people. The ODD is granted to encourage the development of treatments for these rare conditions, as the market for these drugs is typically small and may not be financially viable without additional support. Once a drug receives ODD, it qualifies for various regulatory incentives, including market exclusivity, financial incentives and assistance in the regulatory approval process.
By understanding the criteria, benefits and the EMA’s new drug approval process for orphan drug designation in the EU, you can better navigate the regulatory landscape and contribute to addressing unmet medical needs in rare disease treatment.
What are the criteria for obtaining orphan drug designation in the EU?
The EMA has specific criteria for granting orphan drug designation. These criteria are designed to ensure only those drugs that address true unmet medical needs for rare diseases receive this status. The criteria include:
- The rarity of the disease. The drug must be intended to treat a rare disease (defined by the European Union as a condition affecting no more than 5 in 10,000 people across the EU).
- Unmet medical need. There must be no satisfactory treatment available for the disease, or the drug must provide significant benefit over existing treatments. This demonstrates that the orphan drug will fill a gap in the market and provide meaningful improvements in patient outcomes.
- Scientific justification. The application for orphan drug designation must include scientific data signifying the drug has the potential to treat the rare disease. The data could include pre-clinical results, early-phase clinical trials or compelling evidence of how the drug can address the underlying mechanisms of the disease.
- Potential for market exclusivity. The EMA requires evidence that the orphan drug has the potential to be a viable treatment option for the disease, even with the limited patient population. This involves providing detailed plans for clinical development and indicating how the drug may be marketed successfully despite the small market size.
What are the benefits of orphan drug designation in the EU?
Achieving orphan drug designation comes with several significant advantages. These benefits are designed to mitigate the financial challenges involved in developing treatments for rare diseases, accelerating drug development and the new drug approval process.
Market exclusivity
One of the key benefits of receiving ODD is market exclusivity for a period of 10 years from the date of marketing authorisation. During this period, no similar medicine can be approved for the same indication, providing you with a competitive advantage. Market exclusivity helps incentivise investment in rare disease research by offering a degree of protection from competition.
Reduced regulatory fees
Treatments with orphan drug designation are eligible for reduced fees for regulatory submissions and evaluations. These cost reductions help ease the financial burden of clinical trials, regulatory filings and other activities required to bring a drug to market.
Accelerated development and approval
Orphan drug designation facilitates accelerated new drug approval processes through priority review. This means that the EMA may prioritise your drug’s review, allowing for faster approval compared to non-orphan drugs. In certain cases, your drug may qualify for conditional marketing authorisation, which allows it to be approved based on less extensive clinical data if the drug addresses an urgent medical need.
Scientific advice and support
Orphan drug designation grants you access to scientific advice from the EMA. This support can help guide the drug’s development process, ensuring clinical trials are designed efficiently, and the regulatory requirements are clearly understood. The EMA provides assistance with trial design, endpoints and even potential collaborations with the European Commission to support the drug development process.
Tax incentives and grants
In some cases, you may be eligible for additional financial incentives, such as tax credits or grants from the EU or individual member states, when developing orphan drugs. These funds help offset the high costs of orphan drug development, making it more feasible for you to invest in research for rare diseases.
Access to EU-wide market
Once the drug is approved, you can market the drug across all EU member states. This broad market access is crucial for orphan drugs, as it allows the product to reach as many patients as possible, even within a small population.
What does the EMA orphan drug designation application process involve?
The orphan drug designation process in the EU involves several steps. Here’s an overview of the process:
- Submitting an application to the EMA. The first step for obtaining orphan drug designation is to submit an application to the EMA’s Committee for Orphan Medicinal Products (COMP). The application should include detailed information about the rare disease, the drug and the evidence supporting its potential benefits for treating the condition.
- Review of the application. The COMP reviews the application to assess whether the disease qualifies as rare and whether the drug meets the scientific, medical and market-related criteria. The review process takes about 90 days. If the COMP finds that the application meets the necessary criteria, they’ll issue a positive opinion for orphan drug designation.
- EMA decision. Once the COMP issues a positive opinion, the EMA issues its decision on whether to grant your drug the orphan drug designation. If approved, you’ll receive the official ODD status, which opens the door to the associated benefits.
- Ongoing obligations. Even after receiving orphan drug designation, you must continue to fulfil certain obligations, including submitting progress reports and complying with regulatory requirements for clinical trials and marketing authorisation applications (MAA).
Secure orphan drug designation with TMC
For patients with rare diseases, orphan drugs are often their best — and only — hope for effective treatment. So, obtaining orphan drug designation is a valuable step in the development of treatments for rare diseases.
The EU’s orphan drug programme, spearheaded by the European Medicines Agency (EMA), offers a range of benefits designed to help you overcome the challenges of developing treatments for small patient populations. The incentives of market exclusivity, reduced fees, scientific support and accelerated new drug approval processes make it easier for you to bring these innovative treatments to market.
You can significantly improve your chances of securing orphan drug designations and gaining market authorisation approval with TMC’s regulatory submission services. Our expert team can guide you through obtaining the appropriate scientific advice and agency meetings.
We can also act as your marketing authorisation holder, if you don’t have a presence in Europe, to ensure compliance with all local regulations. We take on all the legal and regulatory accountability to ensure your product is manufactured, marketed and distributed according to the terms and conditions laid out in the marketing authorisation.
As an established EU/EEA-based marketing authorisation holder with SME status, TMC can be your EU/EEA representative — simplifying new drug approvals and helping you secure substantial discounts on EMA fees.
Find out more about TMC’s regulatory services or contact our team today at regulatory.services@tmcpharma.com to see how we can help you gain ODD status and support your marketing authorisation application.