Early Access to Medicine Schemes

Since the outbreak of a pandemic across the globe, we’ve grown used to governments and healthcare providers resorting to investigational and repurposed drugs in order to treat patients in need of care. Initiatives such as the UK’s RAPID-19 aimed to get treatments for COVID-19 to NHS patients in a drastically accelerated timeframe. Other programmes established by the FDA allowed clinicians in the US to gain access to investigational therapies during the pandemic, and programmes such as the expanded access (EA) programme, and emergency use authorisation (EUA) programme, have allowed for the rapid deployment of potential therapies for investigational treatments with emerging evidence.

COVID-19 highlighted the reality that time is an invaluable resource in healthcare, and the delivery of lifesaving therapies to patients hinges on the timescale they can be delivered in.

Early access schemes also play a vital role in delivering treatment and management for rare diseases. The ABPI has shared that “nearly 40% of the medicines now in development are for rare or very rare conditions, including many cell and gene therapies which are personalised to individual patients.”1

Considering that only 10% of known rare diseases currently have an approved treatment,2it is clear the impact early access schemes may have on patients living with conditions for which no approved treatment, or even management option, currently exist. There may be medicines in the development pipeline for these conditions not yet approved for marketisation, but with clear clinical benefit.

“The Early Access to Medicines Scheme (EAMS), the Innovative Medicines Fund, Accelerated Access Collaborative and Innovative Licensing and Access Pathway all hold the potential to improve the situation for patients with rare and very rare conditions – as well as those for more common conditions,” the ABPI shares.

In the UK, there are a number of initiatives aiming to bring patients early access to lifesaving medicines. The Accelerated Access Collaborative (AAC) is a fast-track route into the NHS for ‘breakthrough’ medicines and technologies. It is a national group made up of partners including

NHS England and NHS Improvement, the Government, NICE, industry associations, research organisations, patient representatives, and other health and care partners.

The Innovative Licensing and Access Pathway (ILAP) aims to streamline patient access to safe, financially sustainable, and innovative medicines, including new chemical entities, biological medicines, new indications, and repurposed medicines. The Accelerated Access Collaborative, EAMS, Innovative Licensing, and Access Pathway are all mechanisms designed to fast-track promising medicines safely through the approval process, so that they get to patients who could benefit as soon as possible. The ABPI believes that “the Early Access to Medicines Scheme (EAMS), the Innovative Medicines Fund, Accelerated Access Collaborative, and Innovative Licensing and Access Pathway all hold the potential to improve the situation for patients with rare and very rare conditions – as well as those for more common conditions,” but maintains, however, that “the opportunity of these initiatives needs to be realised for rare diseases, as well as more common ones.”

Following the outbreak of Ebola virus in West Africa in 2014-16, WHO issued a statement sharing that, in healthcare emergencies, it was ethical to provide patients access to investigational drugs not approved by regulators, in order to save lives.

Johnson & Johnson announced that it would donate Ebola vaccine regimens in support of a WHO early access clinical programme. The programme was launched in response to an outbreak of the virus in Guinea, and aimed at preventing Ebola in West Africa. It began by vaccinating health and other frontline workers, alongside those at increased risk of exposure to the virus in Sierra Leone.

By the end of 2021, over 250,000 patients participating in clinical trials and vaccination initiatives had received at least the first dose of the vaccine regimen for Ebola by J&J. Further, over 200,000 had been fully vaccinated.

There were limitations to the study, however, including the fact that there were more men than women in the adult trial, and the measurement of Ebola antibody concentration levels was only in a subset of participants. Other limitations of the study include the fact that it only focused on safety and immunogenicity, as it was implemented when the West Africa Ebola outbreak had been brought under control. It was therefore not possible to evaluate the efficacy of the regimen.

The Early Access to Medicines Scheme (EAMS) was responsible for the select use of the first COVID-19 medicine, Gilead’s remdesivir. This drug worked to save lives, decrease disease severity and thereby decreasing health issues such as long-COVID-19, and lessening the burden faced by the NHS in a time of crisis. In the UK, the Early Access to Medicines Scheme (EAMS) works to give those living with seriously debilitating or life-threatening diseases, early access to new medicines that have not yet received a marketing authorisation, where there is significant and unmet medical need.

Further, following analysis of the six-week consultation by the MHRA in 2021, the UK regulator has prepared a Government response, outlining key legislative changes, which will provide the UK with an opportunity to maximise the Scheme’s impact. This will be done through accelerating availability of medicines for patients, reducing the burden on manufacturers supplying EAMS medicines, and facilitating the collection of real-world data, which may be used as evidence to support regulatory decision-making for future authorisations.

All this will work to help support more patients benefiting from important EAMS medical products, and the healthcare practitioners delivering them.

WHO recommends antiretroviral treatment (ART) for all HIV-positive patients, regardless of CD4 count or disease stage. This initiative is referred to as “Early Access to ART for All” (EAAA). Early HIV infection is the period up to 6 months after infection with HIV.

HIV is treated with antiretroviral medicines, which stop the virus replicating in the body. ART involves taking a combination of HIV medicines every day, and while it cannot cure HIV, they reduce a person’s viral load to an undetectable level. This suppression of the virus allows the immune system to repair itself, and prevent further damage. A combination of HIV drugs is used, as HIV can quickly adapt and become resistant. For such reasons, it can be extremely important to equip patients with the best and most effective and individualised care for them as possible. Various Early Access schemes can help deliver such healthcare to patients.

Without access to antiretroviral medicines, the virus is able to attack and destroy the infectionfighting CD4 cells (CD4 lymphocyte) of the immune system, the loss of which makes it more difficult for the body to fight off infections, as well as certain HIV-related cancers.

EAAA saw a “substantial effect on retention and a large effect on viral suppression”, according to the Final Report on the study published in December 2018. “During ‘Standard of Care’ and ‘Early Access to ART for All’ respectively, the 12-month retention rates were 80% and 86%, and the viral suppression rates at 12 months post-ART initiation among those we were retained that long were 4% and 79%.”

The report shared: “The MaxART Early Access to ART for All implementation study has been a unique project, testing the real-life implications of providing early antiretroviral treatment (ART) to all people living with HIV through the government managed health system of the Kingdom of Eswatini. The results show that early ART is acceptable, feasible, affordable and effective in preventing new HIV transmissions and improves the health and well-being of people living with HIV. The potential of early ART opens a new era in HIV prevention and treatment, with the possibility of ending HIV and AIDS.”3

In the stepped-wedged, randomised, controlled trial, 14 public sector health facilities in Eswatini were paired, and randomly assigned, to stepwise transition from standard of care (SoC) to EAAA. The MaxArt Consortium trial additionally saw an increase in testing coverage in the community, where compared with baseline data gathered three months prior to the start of the trial, there was a 273% proportional increase in HIV test conducted among adult males, adolescent females, and adult men. The study also found a 722% increase over baseline for adolescent males.

Additionally, because of the MazART trial, the health service in Eswatini was able to identify areas for further investment, demonstrating an often-unconsidered benefit to early access schemes for patients. This benefit included the health service in the country being able to address the system-side barriers to routine viral load monitoring, also designing and implementing innovative community-based approaches, reaching people living with HIV who were not necessarily routinely accessing HIV testing or counselling services.4

Early Access Programmes (EAPS) of a number of forms are being adopted by more institutions and companies every day, delivering vital and lifesaving medicines to patients who have no treatment options otherwise available to them. Different early access programmes might aim to combat the spread or severity of a particular illness, such as EAAA, while others offer pathways for ethical, controlled mechanisms of access for investigational drugs prior to commercial launch, not in a trial setting.

With new issues to global health constantly arising, from monkeypox to this month’s news about tomato flu, it’s possible that we’ll see more early access schemes in the years to come than ever before.