Lenalidomide maintenance – the journey to a “yes”

29th January 2021 // Shelagh McKinlay

At about six o’clock, in the morning of 21 June 2019, I sat bleary eyed in the departure lounge of Edinburgh airport, waiting to board a plane south where a short taxi ride would deliver me to the UK HQ of the pharmaceutical company, Celgene.

Celgene (since bought by Bristol Myers Squibb) were the manufacturers of lenalidomide (Revlimid®), and Dr Simon Ridley, our then Director of Research, and I were there to discuss plans for lenalidomide maintenance post stem cell transplant (HDT-SCT) to be made available to myeloma patients in the UK.

It wasn’t the first meeting I’d had about lenalidomide maintenance, and it certainly wouldn’t be the last, but it was a pivotal moment – just a few weeks after that June meeting we finally got confirmation that an appraisal for lenalidomide maintenance would get underway.

Fast forward to 27 January 2021 and the approval of lenalidomide maintenance was announced by NICE, around three months after it had been approved in Scotland.

For the first time, newly diagnosed myeloma transplant patients throughout the UK will have access to a maintenance treatment, in line with international best practice.

All treatment approvals in myeloma are important but lenalidomide maintenance stands apart; both in the number of patients who stand to benefit and the improved effectiveness it delivers.

Each year, around 1,300 myeloma patients in the UK receive a stem cell transplant, and lenalidomide maintenance offers the potential to double remission times and improve survival for every one of those patients. When you think of the days, months, and years of extra quality of life that will deliver it is quite overwhelming. It’s a major step forward in the treatment of myeloma in the UK.

They say that success has many fathers and in this instance that is certainly true. The approval of lenalidomide maintenance has been the result of partnership and collaboration, including the partnership between patients and clinicians who participated in the Myeloma XI trial under the leadership of Professor Graham Jackson. This data was crucial to the treatment’s approval, enabling the benefits of maintenance to be fully demonstrated.

Myeloma UK supported the trial and helped to recruit patients. Once the data had been delivered, the advocacy team stepped in, working with patients, clinicians, Bristol Myers Squibb (BMS), NICE, the Scottish Medicines Consortium and NHSE England to get it over the line.

This involved too many meetings, emails, and letters to count, but some of our key actions were:

  • Developing, with input from patients, a survey which generated over 460 responses – the largest treatment survey we have ever done – where 98% of respondents wanted maintenance approved and 90% of patients who had received the treatment rated their experience as very positive or positive
  • Submitting written evidence to NICE and the Scottish Medicines Consortium (SMC) on the scope of the appraisal, the case for approval, and comments on technical issues
  • Attending the SMC full committee and Patient and Clinician Engagement (PACE) meetings and two NICE Committee meetings
  • Working with our wonderful Patient Expert, Steve Bilcliffe, who brought his knowledge and lived experience to bear on the process
  • Meeting with Clive Betts MP, himself a myeloma patient, who was hugely helpful and supportive in keeping things moving when progress threatened to stall
  • After NICE initially recommended against approval we worked with clinicians and with the company to understand the challenges and to make sure that the patient voice was heard every step of the way, turning the “no” into a “yes”

This list of activities is by no means exhaustive but it hopefully gives a sense of what goes on behind the scenes when we are working to get new treatments approved. It is resource intensive and complex work and it does take time – in this instance, unfortunately, more time than we would have wanted.

Lenalidomide has been approved as a treatment in the UK for over a decade. It received its licence for maintenance post HDT-SCT in 2017, the results of the Myeloma XI study were published in late 2018 – and yet it was not till late 2019 that the process for approval really got underway, and not until 2021 that we got a positive decision.

There are lots of reasons for this: interpreting the data, negotiating a commercial agreement, these things all take time and each player in the system can explain why things are as they are – but meanwhile patients are waiting and, sadly for some, their opportunity to access this treatment has been missed.

And so mixed in with the relief and gladness that we now have a “yes” there is also a sense of sadness about those patients who have missed out. That’s why we are committed to not only doing our best to get a positive result from every individual appraisal, but to keep working to make the whole system better.

We have the busiest drug approval pipeline we have ever had and will shortly begin work on our first appraisal of innovative new CAR-T therapies. We are constantly looking to the future and keeping pace with progress, but today it is also good to look back at how far we have come and to savour the knowledge that the approval of lenalidomide maintenance will help thousands of patients in the future to live longer and with a better quality of life.