flip, since the MHRA has already approved DCVax-L for compassionate use, all they would need to do is provide the funding for the patients to receive the treatment. There are new pathways in the UK if the MHRA is receptive. Here's a couple:
Conditional Marketing Authorization Applications The MHRA has introduced a national conditional marketing authorization (CMA) scheme for new medicinal products in UK effective from 1 January 2021. Eligibility criteria for this scheme, as that of the EU scheme, is intended for medicinal products that fill an unmet medical need and for serious and life-threatening diseases where no satisfactory treatment methods are available, or where the product offers a major therapeutic advantage.
The MHRA determines eligibility for a CMA at the time of MAA assessment. The MHRA does not have a specific application route for a CMA. e sponsor needs to file the MAA dossier for a full marketing authorization. At the completion of the MAA dossier assessment, the MHRA will determine whether to approve the application and grant a conditional MA or whether the benefit-risk ratio is negative and reject the application. e CMA may be granted where comprehensive clinical data is not yet complete and available. e sponsor must provide justification for a CMA, including the ongoing clinical studies’ status and timing of the availability of comprehensive clinical data. CMAs are valid for one year and can be renewed annually.
Exceptional Circumstances Marketing Authorizations From 1 January 2021, the MHRA’s existing scheme for applications under exceptional circumstances will continue to be available for medicines where a comprehensive data package cannot be provided because the condition to be treated is rare or because the collection of full information is not possible or is unethical. This scheme has the same eligibility criteria as the EU scheme (see Chapter 2). designation of a product as being eligible for an exceptional circumstances scheme by EMA or another jurisdiction may be taken into account by the MHRA, but the final decision on eligibility of the product for the GB scheme will rest with MHRA.
And perhaps this will help:
Patients to have earlier access to cutting-edge treatments on NHS The most promising medicines will be fast-tracked to NHS patients through the Innovative Medicines Fund. From: Department of Health and Social Care and The Rt Hon Sajid Javid MP Published 7 June 2022
* £340 million has been made available to purchase potentially life-saving drugs early * It builds on the success of the Cancer Drugs Fund, which has given tens of thousands of patients access to revolutionary treatments quickly * Improving access to treatment for patients most in need could alleviate pressure on the NHS, helping bust the COVID-19 backlog
NHS patients in England will have early access to potentially life-saving and cutting-edge treatments thanks to a new fund which has launched today.
Up to £340 million has been made available through the Innovative Medicines Fund to purchase the most promising medicines and fast-track them to patients to give adults and children the best chances of survival, recovery or a healthier, longer life.
The fund, which meets a manifesto commitment, will further support NHS England in offering patients potentially transformative new drugs while further real-world evidence is collected to inform a final decision by the National Institute for Health and Care Excellence (NICE) on whether the treatment is clinically and cost effective and a good use of taxpayer money in the long term, reducing delays and boosting patient outcomes in the interim.
Examples of previous medicines which patients have accessed in a similar way through managed access agreements include a treatment for children with spinal muscular atrophy and a treatment to slow the progression of a life-limiting metabolic disorder.
It builds on the success of the reformed Cancer Drugs Fund which, in the past 5 years, has provided more than 80,000 people access to life-extending or potentially life-saving drugs which might otherwise not have been available for years.
Health and Social Care Secretary, Sajid Javid, said: I want NHS patients to be the first in the world to access the most promising and revolutionary treatments that could extend or save their lives. The launch of the Innovative Medicines Fund delivers another manifesto pledge and will fast-track cutting-edge medicines to adults and children to give people renewed hope for a better future.
A total of £680 million has been ringfenced for the Innovative Medicines Fund and Cancer Drugs Fund – £340 million each – to fast-track medicines to NHS patients.
An estimated one in 17 people in England will be affected by a rare disease in their lifetime. The Innovative Medicines Fund will provide quick access to novel treatments, including potentially lifesaving gene therapies for serious conditions with few treatment options. It often takes longer for pharmaceutical companies to collect data on a medicine’s clinical and cost effectiveness for rare diseases due to the smaller patient cohort. Rather than making patients wait until this date is available, this new scheme will allow access while this important process takes place, with support from NHS England and NICE.
NHS Commercial Medicines Director, Blake Dark, said: The NHS continues to be a pioneer in striking deals and rolling out the latest cutting-edge drugs and treatments. This new Innovative Medicines Fund will build on the success of the Cancer Drugs Fund, enabling more patients to benefit from early access to the most promising cancer and non-cancer medicines.
The NHS Long Term Plan shows we are committed to adopting NICE-approved treatments at the earliest opportunity and £680 million of ringfenced funding will help provide faster access to promising new drugs and ensure the NHS remains at the forefront of securing the best revolutionary treatments for patients.
It is hoped improving access to treatment for those patients most in need will help alleviate pressure on the NHS, supporting wider efforts to tackle the COVID-19 backlog.
All medicines deployed through the Innovative Medicines Fund and Cancer Drugs Fund will have been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) after meeting high standards of safety and quality and will have been recommended as suitable for the fund by NICE.
Chief executive of NICE, Dr Samantha Roberts, said: I am delighted that NICE has been able to play a key role, alongside NHS England and NHS Improvement, to create this important new initiative to give people earlier and faster access to promising new innovations in treatment.This fund, like the Cancer Drugs Fund, will help us more quickly identify and make available transformational new treatments that will bring real benefits to thousands of people and offer high value to the NHS.
The Innovative Medicines Fund supports the government’s Rare Diseases Action Plan to ensure people living with rare conditions benefit from faster diagnosis, treatments and support to manage their conditions.
Following a public call for evidence, the government is also developing a 10-year cancer plan to make England a world-leader in cancer care, with renewed attention paid to innovative treatment and early diagnosis to improve outcomes for patients.
Background As part of the NHS Long Term Plan, NHS England’s Commercial Medicines Directorate has used its commercial capabilities to secure access to several innovative medicines for NHS patients, including many ‘world-first’ or ‘first in Europe’ drug deals.
Some of the most successful treatments which have been rolled out through the Cancer Drugs Fund include: * CAR-T therapy – children and adults in the UK were among the first in Europe to benefit from this innovative gene therapy. It has now been approved by NICE and is available at 13 NHS hospitals across England * Larotrectinib (Vitrakvi) – a cancer treatment for children, young people and some adults which targets tumours according to their genetic make-up, rather than where they originated from in the body. * Kisqali (ribociclib) – a breast cancer drug that was available through the Cancer Drugs Fund and is now routinely funded, potentially benefiting up to 3,300 women
