Orphan drugs

The global market for drugs that treat rare diseases is growing. Orphan drug products often sit at a complicated intersection of medical science and regulatory law. From orphan drug designation and authorisation, through to the legal protections that follow, our life sciences team has the specialist knowledge to advise on all legal aspects of the orphan drug product lifecycle.

Clear, commercial advice on orphan drug law

Orphan drugs are medicines developed for rare diseases: conditions that affect only small numbers of patients individually, but which collectively represent a significant area of unmet medical need. The cost of research and development into treatments for small patient populations may be hard to justify commercially, but the UK and the European Union offer incentives to encourage investment in this area, including an entitlement to a period of market exclusivity (orphan market exclusivity) once the product is authorised. During this time, other companies may not launch an equivalent or similar product for the orphan medical condition.

In practice, the precise scope of orphan market exclusivity is often unclear; in particular, regarding whether a new product is genuinely ‘similar’ to one that is already benefiting from orphan exclusivity, or whether an exception applies because the new product offers a meaningful clinical improvement. These questions sit on the boundary of regulatory requirements and clinical evidence and the conclusions can have significant commercial consequences.

Since Brexit, the UK and EU orphan regulatory regimes are gradually diverging, with further major changes expected soon. A product’s orphan status may now differ depending on which medicines regulator is involved. Companies operating across both markets need practical advice that reflects the current and evolving legal landscape.

The combination of expertise, lateral thinking and client-centric approach sets them apart from other firms.

Our life sciences lawyers have a range of expertise and experience that makes them particularly well suited to advising in this area. We have a high level of medical and scientific literacy, based on our prior experiences in clinical and research environments, as well as deep practical knowledge of, and familiarity with how the exclusivities actually work in the UK and EU. In addition, our experts possess an understanding and a practical, commercial appreciation of how businesses plan around exclusivity over the life of a product.

Members of our life sciences litigation team can provide advice on any related disputes, including enforcement of, and challenges to, both regulatory exclusivities and patent rights. This means that we can help clients see clearly where they stand, whether that is protecting a hard-won orphan market exclusivity, or working out if there is genuine room in the market for a competing product.

How we help our clients

Guidance on current status of UK/EU orphan exclusivities, and their likely scope

‘Freedom to operate’ advice concerning orphan drugs, patent rights, and market access strategies

Regulatory IP rights including data protection, market exclusivity, SPC and paediatric rewards

Practical UK and EU regulatory advice on marketing authorisations, pricing and reimbursement

NHS ‘net zero’ requirements for suppliers of orphan drugs

Patent and regulatory litigation

Recent work highlights

Inclusion of indications in SmPC

Advising a client on the appropriate non-orphan medical indications to include in the summary of product characteristics (SmPC) for a ‘skinny labelled’ medicine that was subject to orphan market exclusivity for another indication.

Orphan drug products due diligence

Providing detailed due diligence on potential orphan drug products from third-party developers, including advice relating to the strength of the patent protection around the molecule and the eligibility for protection by means of orphan exclusivity in the UK and the EU.

Orphan market exclusivity term limits

Assisting a client in liaising with a medicines regulator over the correct term of orphan market exclusivity and the limits of that exclusivity, especially in relation to post-Brexit interpretation of terms imported into UK law from EU legislation.

Commercial agreements and IP ownership

Advising on all aspects of commercial agreements with suppliers and client partners, including collaboration agreements and, in particular, IP ownership provisions arising from foreground and background IP.

Advice on NHS net zero requirements

Providing guidance to an orphan drugs client on how to comply with NHS ‘net zero’ requirements for suppliers, in line with its commitment to becoming a carbon net zero health system.

AI in orphan drug development and trials

Considering the implications of the use of artificial intelligence in orphan drug development and clinical trials, in particular the requirements of medicines regulators such as the MHRA.

Eligibility for awards following clinical trials

Advising on various aspects of eligibility for rewards (paediatric exclusivity and extensions to supplementary protection certificates) following the completion of clinical trials in children, including proactive monitoring of ongoing clinical trials being undertaken by third parties (competitive intelligence).

Eligibility for awards following clinical trials

Penningtons Manches Cooper has cultivated a very strong niche in the patent transactions space.

A flair for the fine print of IP-rich transactions. No patent-related agreement is beyond its capabilities.

They have deep experience in understanding the requirements of both investors and investees and developing appropriate legal frameworks to meet these.

An exceptional practice with highly qualified partners and associates. Trustworthy and consistently producing high quality work with very quick turnaround.

The entire team at Penningtons exhibits exceptional expertise in their respective specialisation. Regardless of the individual we interact with, we consistently receive exceptional service at a fair price.