Early Access to Medicines Scheme and Medical Innovations (Saatchi) Bill

Two exciting developments of particular interest to people with mesothelioma and other life threatening conditions for which there is no cure at the moment, are the Early Access to Medicines Scheme and the Medical Innovation Bill, aka The Saatchi Bill. These were both discussed in the House of Commons last Tuesday (25 March).  

Earl Howe is the Minister responsible for quality at the Department of Health who spoke at the meeting.  The full text of his peech is set out below.  Worth reading!  

I am very pleased to be here today to talk about this important subject. I do, of course, recognise the concerns raised by the other speakers before me regarding access to new and promising medicines for these terrible conditions.

I would like to assure those present that our priority is to ensure that patients, including those with rare and life threatening or limiting conditions, have access to new and effective treatments on terms that represent value to the NHS and the taxpayer.

Early Access to Medicines Scheme

I would like to spend a few minutes setting out the aims of the scheme, which I hope you will agree is an important development.

Through the scheme, the Medicines and Healthcare products Regulatory Agency will provide a scientific opinion on promising new, unlicensed or off-label medicines to treat, diagnose or prevent life threatening or seriously debilitating conditions which do not have adequate treatment options.

Our hope is that the scheme, which will operate within the current regulatory structure, could give patients with these conditions access much sooner to medicines that represent a significant advance in their area of unmet need.

MHRA is responsible for managing the scientific aspects of the scheme, which will follow a two-step process.

Step one includes giving a new medicine a promising innovative medicines designation.

This will provide an early indication that a product may be a possible candidate for early access, based on the available clinical data. The designation will be issued following an MHRA scientific meeting and could be given several years before the product is licensed.

Companies who wish to move to step two must hold a promising innovative medicines designation and provide further relevant data on their product’s quality, safety and efficacy.

At step two, the MHRA will produce a scientific opinion describing the benefits and risks of the medicine, based on information submitted by the applicant after sufficient data have been gathered from the patients who will benefit from the medicine.

The trigger for an Early Access to Medicines scientific opinion does not necessarily have to be the submission of a dossier for marketing authorisation application, but the availability of a sufficiently compelling case based on the total data and evidence collected to date as assessed by the MHRA.

This is, of course, conditional on data from the development process of the product which indicates that the benefit:risk profile of the medicine is positive.

The scientific opinion will be made available on the MHRA’s website to assist clinicians and patients in making treatment decisions, and to support informed consent by patients to the risks and benefits of the product.

The scheme will be launched and ready to receive applications in April 2014. I understand that full details, together with guidance, will be published on the MHRA website in due course.

Conclusion

This is an exciting initiative and just one way in which the Government is supportive of improving access to new medicines.

We are also consulting on a draft Medical Innovation Bill which aims to encourage responsible innovation in certain circumstances, and to discourage irresponsible innovation. The consultation paper was published on 27 February and the consultation period runs until 25 April. I would encourage anyone with views on the proposals to respond to the consultation.

We continue to support the existing incentives offered at EU level to encourage the development of medicines for small numbers of patients (‘Orphan’ medicines), and the development of the Commission’s plans for adaptive licensing, including their launch of the EMA adaptive licensing pilot on 19 March.

In giving patients access to the next generation of medicines before they are licensed, I hope you will agree that this scheme could help seriously ill patients in areas of unmet clinical need to have earlier access to new and effective medicines.