US Company Awarded UK's First "Promising Innovative Medicine" Designation

The UK’s Medicines and Healthcare Regulatory Authority (MHRA) has awarded Northwest Biotherapeutics, Inc. (Bethesda, MD) the very first “Promising Innovative Medicine”, or PIM designation, for the company’s DCVax-L product.

A positive PIM is the first step towards an Early Access to Medicine Scientific Opinion as part of the UK’s Early Access to Medicine Scheme (EAMS).  What that means is that the MHRA will publish both a treatment protocol and their opinion on the benefits and risks of the product, based on the data available at that point in time, in a Public Assessment Report (PAR).

None of this replaces a formal marketing authorization; that will have to come once more data is available and with real world evidence expected to be collected whilst the product is used under EAMS.

But getting a PIM is not straightforward. (See MHRA’s gateway criteria here.)  The company has to convince MHRA that the condition is life threatening or seriously debilitating with a high unmet need. They also have to convince them that the product will offer a major advantage to methods already used in the UK, and that any adverse effects are likely to be outweighed by the benefits. MHRA needs to see the preliminary scientific evidence and will scrutinize it carefully.

Northwest Biotherapeutics’ DCVax-L is under development to treat Glioblastoma multiforme (GBM). The company describes GMB as the most lethal form of brain cancer. Prognosis isn’t good following diagnosis; around 30% of patients are still alive after a year. Surgery, radiotherapy and chemotherapy are the standard treatments but they aren’t curative. GMB also rare, affecting perhaps 2 or 3 in 100,000.

Next step: EAMS scientific opinion
To get the positive EAMS scientific opinion more needs to be done. A dossier needs to include information for the patient, physician, pharmacovigilance with a risk management plan and a registry. Plus an overview of the immature data available on the product. But much of that is what is already needed in any case for getting marketing authorization.

EAMS is part of a much bigger effort to boost life sciences in the UK.  It’s been trumpeted by the Life Sciences Minister, George Freeman. He said; “Making Britain the best place in the world for science, research and development is a central part of our long term economic plan.”  He’s also hoping that more companies want to take part.

What difference does a PIM make?
More PIMs can be expected, perhaps as many as 12 a year.  What we don’t know yet is how valuable getting a PIM is (although Northwest’s stock price did ‘surge’ following the announcement). It might signal to investors how important a breakthrough the product is, but it doesn’t automatically translate into immediate revenue. That’s because before formal licensing, manufacturers getting a PIM, or even the full positive EAMS scientific opinion, are expected to supply to the NHS at no charge.  Companies are also still expected to go through NICE and funding will then be dependent on getting a nod from them.

But all of that could be up to a year faster than would have been the case, adding valuable years to effective patent life.

Leela Barham (leels@btinternet.com) is an independent health economist and policy expert. http://leelabarhameconomicconsulting.blogspot.co.uk