Around 300 million people share a devastating collection of conditions: ultra-rare disorders. Each individual condition affects less than 1/50,000, but in some cases are fatal. Until now, the state of scientific knowledge, regulation and economics made treating these conditions impossible – controlled trials with thousands of participants couldn’t work where, in some cases, only one individual had the condition. Yet this month Britain’s Medicines and Healthcare products Regulatory Agency (MHRA) approved a new sort of clinical trial where 10 patients would receive a unique antisense oligonucleotide (ASO). These ASOs work by ambushing the faulty RNA messenger molecules caused by the disorder, then either neutralising or altering them to mitigate their effects.
This treatment is a breakthrough, but 10 patients out of a possible 300 million shows the scope for progress. For one, the costs of producing these individual treatments are eyewatering – EveryONE Medicines (the firm tailoring the ASOs to each patient) estimates that it costs between $2-3mn and 2-3 years to develop these therapies. Further, skipping the normal clinical trial process will magnify any scrutiny if the treatment proves unsuccessful.
Exclusivity for individualised products
Mila’s Miracle Foundation (a charity dedicated to the promotion of individualised medicines) has advocated for improving the efficiency of the sector by promoting greater private sector involvement. One of the key challenges for the private sector however will be protecting their initial investment in customised medicines. Patents – typically the lifeblood of the life sciences industry – face two main challenges in this context:
Firstly, the idea of an individualised medicine would not, in of itself, be patentable in the UK. Patents may not be granted for methods of medical treatment (), which the concept of treatment would clearly fall within. Potential developers will therefore need to frame their claims more narrowly; for example, by patenting a particular ASO for a particular use in the medical context (see EPO Guidelines, Part G-IV 4).
Secondly, any patent claim must make a plausible technical contribution (see Warner-Lambert v Generics [2018] UKSC 56). Given the present uncertainty around individualised genetic medicines, the relatively high threshold adopted by the Court in Warner-Lambert –there must be something that causes the skilled person to believe that there is a reasonable prospect the claim is true – is likely to increase the pressure on drug makers to keep any individualised medicines confidential until at least the end of a successful use in a clinical trial.
Genetic mutations
Individualised medicine developers face further risks if the treatment ultimately proves harmful; notably the Consumer Protection Act 1987 s.3(1) requires that the safety of a product is ‘generally such as person are entitled to expect’. To mitigate this risk developers should:
- Focus on patients who face the greatest risk without any medical intervention. The legal assessment of any product’s safety will consider the risks, benefits and opportunities involved (Wilkes v Depuy International Limited [2016] EWHC 3096). For patients who are otherwise facing certain death (as many with rare genetic conditions are) a court is less likely to conclude that a risky method of treatment failed to meet the standards required.
- Communicate the risks clearly to all relevant stakeholders. As the judge made clear in Pollard v Tesco Stores Ltd [2006] AER 186, the focus of the CPA is on the ‘actual’ expectations of the consumers involved. Addressing with parents at the outset the experimental nature of individualised medicines – along with being good practice – will safeguard against liability.
Medically discharged
Individualised medical treatment present a moment of hope for patients, practitioners and parents. The best way to develop these treatments is with further private sector involvement; for which issues of patentability and future negligence claims warrant careful consideration.
