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On 15th July 2020, Findacure was joined by Naomi Litchfield of Bionical Emas, Josie Godfrey of Metabolic Support UK and Liz Ryburn of Spinal Muscular Atrophy UK for a collaborative webinar that focused on Early Access Programmes (EAPs) and how patient groups could participate in them.

What are Early Access Programmes (EAPs)?

EAPs are the pathway to investigational medicine: more specifically, they are schemes developed by companies to allow patients to gain free access to drugs that are still under investigation, but have shown promise in their condition. EAPs are the only alternative for patients who aren’t able to participate in clinical trials due to eligibility, location, cost or other external factors, because there is no approved treatment available to them.

The downside of drug research and development is that it takes time to develop and produce a drug safely, efficiently and ethically; a commodity that isn’t on the rare disease community’s side. Safety must never be compromised in an effort to get a drug out to patients faster, which led our webinar to focus on the unique role EAPs have to play within the rare disease community.

If you happened to miss our insightful webinar, here is our recap of the major points and key takeaways from all three speakers!

Speaker 1: Naomi Litchfield from Bionical Emas

Naomi Litchfield was the first to speak on our “What does early access actually mean?” webinar. Naomi works for Bionical Emas, which is a Clinical Research Organisation (CRO) that’s headquartered in Willington, Derbyshire, UK. Naomi specialises in Early Access Programmes (EAPs), but comes from a clinical research nursing background.

Naomi first worked as a nurse at Greater Ormond Street Hospital (GOSH) and cared for rare disease patients and their families. She not only saw the devastating impact that rare diseases have on her patients, but she also saw those same devastating impacts on her sister who battled and survived four rare types of cancer. Early Access Programmes, or Compassionate Use Therapy as they’re sometimes called, are what saved her sister’s life.

The biggest takeaways from her speech?


  • When part of an EAP, it is the doctor who is requesting the drug on your behalf. Regardless, patient groups should speak with companies and regulators about their need for early access. After all, patient groups know their community’s needs and wants. Don’t underestimate your power – have your voice heard!
  • Do your research on available EAPs for your rare disease and bring that information to your doctor. Talk through your EAP options together so your doctor is aware that you’re interested in partaking in one should the chance arise. It’s important that your doctor knows your stance on EAPs and your willingness to participate!

Speaker 2: Josie Godfrey from Metabolic Support UK

Next up was Josie Godfrey, who is a Trustee at Metabolic Support UK. Josie has over a decade of experience working in the rare disease space, and has set up a highly specialised technology program at The National Institute for Health and Care Excellence (NICE), which is a subset of the Department of Health & Social Care in England. She has worked with patient group organisations, industry and NHS trader bodies to address policy and access for rare diseases.

Josie stated that EAPs represent the following three adjectives to patient groups: hope, life-saving and life-changing. When no treatment, or very ineffective or limited treatments, are available to patients, EAPs become their only hope. She believes that EAPs make a real difference, especially to those with progressive conditions, because EAPs are the only chance these patients have for receiving the promising new medicines that could change and/or save their life.

Josie’s key takeaways?


  • Like Naomi stated, patient groups know their community’s needs, wants and hopes for the future. This information is power. Focus on collecting your patient group’s real-world data so that you can bring solutions, not problems to the table. Your patient group data can help inform NICE processes and discussions with the NHS about providing faster access to medicines in England, Scotland and Wales.
  • It is never too early to talk to companies who are working in your specific rare disease area about their interest in EAPs. Set in place a formal agreement before talking with these companies, so you can protect the confidentiality of your discussions and provide comfort to all parties involved. This will allow all parties to speak freely and openly, so real progress can be made. Don’t wait for the company or NHS to come to you!
  • Communication is key! Ensure that you clearly communicate what EAPs are, what they look to achieve, what they can offer patients and any limitations there might be to partaking in an EAP, such as eligibility criteria, costs and location. You’ll also want to outline if an EAP will run for a finite period of time, and if so, how long. Patients must understand how long they’ll be guaranteed treatment through the EAP before NICE and the NHS reach a reimbursement decision. Talk early and often!

Speaker 3: Liz Ryburn from Spinal Muscular Atrophy UK

The final speaker on our webinar was Liz from Spinal Muscular Atrophy UK (SMAUK). Liz is a Support Services Team member at SMAUK and comes from a social work background. Liz spoke of her experience advocating for an EAP for the rare disease Spinal Muscular Atrophy Type 1, or SMA Type 1, which has a life expectancy of fewer than two years. She, alongside Spinal Muscular Atrophy UK, TreatSMA and Muscular Dystrophy UK (MDUK), played a pivotal role in bringing the first available treatment for SMA Type 1 to patients through an Expanded Access Programme. Although the treatment wasn’t a cure, it proved to prolong the lifespan of SMA Type 1 patients.

Her tips to patient group organisations that are trying to advocate for an EAP for their rare disease?

  • Get familiar with the UK regulatory system and terminology as fast as possible.
  • Knowing your patient group community and what it needs and wants is key. Keep your community informed!
  • Work with clinicians to align your advocacy efforts. NICE, the NHS and pharmaceutical companies all listen to them. Clinicians are also the people who tried and tested your drug and will be administering it.
  • Know what’s happening globally – understand the global context!
  • Work with pharmaceutical companies, but keep your distance and advice. There are two sides to negotiations.
  • Work with other patient groups and recognise their strengths. Don’t let it become a competition!
  • Don’t underestimate your power, but don’t overestimate your influence. Fight for your drug and community, but remember that there is a wider context and multiple players.
  • Recognize that emotions run high as patients desperately need and want a treatment. Manage expectations.
  • Understand clinical outcomes and economic models of cost-effectiveness.
  • Recognise that each EAP and drug follows its own, unique access route.

To get the full story straight from these experts, watch our “What does early access actually mean?” webinar recording now!

“What does early access actually mean?” webinar recap

by Blayne Baker time to read: 7 min