How does industry better navigate the challenges in getting innovation into routine practice
by Nicola Redfern, NJ Redfern Ltd
Writing this following Rare disease day, having reflected (I am putting pen to paper mid March to meet the deadline) should be easy. LinkedIn was filled with Medics For Rare Diseases socks and amazing heart-wrenching posts, art from Beyond the Diagnosis, video montages from around the globe and events in parliament, the Francis Crick, Swansea hospital—the list was so much longer than in the past—national and local institutions picking up the challenge and shining a light. And the light on rare disease shone brightly in the last week of February and into early March. The RARE Revolution team too did an amazing job as always. We even saw new government commitments made in some countries to help advance the rare disease experience and journey.
So why do I risk entering April unenthused? I know its not just me—others are commenting on the ongoing struggle many face. Perhaps it’s the inevitable slump after all the energy everyone invested in the above, like you have the day after a party or when exams finish. Energy spent. So many of the same challenges and hurdles exist and we have to work harder to see and hear the progress amongst all the other noise in our world right now. Day-to-day living costs are high, waiting times are long, the NHS in the UK (and other countries) is struggling from staff shortages, no beds etc and people struggle every day. The news reminds us of all the bad things happening in our world for so many countries and populations. I can’t write an article at the moment without acknowledging that things are hard and unsettling. And when caring for someone with ongoing health challenges that luxury of down time and slump just doesn’t exist. The day-to-day attempt at normal has to continue and battles resume to maximise the quality of life and experience of the individuals diagnosed and all those around them.
As pharma and biotech we too need to find ways to rise above the noise and challenges to advance access to new innovations and make them a choice in routine conversations for the individuals who might benefit. New processes and regulations are coming into play this year which we are learning to navigate, without all the stars and islands plotted on the map yet. Those who think they have navigated the regulatory and reimbursement hurdles still face significant challenges getting things to patients. Sadly some of the support that was there is also being unpicked and questioned by new governments as other things take priority.
So what can we do positively to help things move forward…..
- We need to pull even closer together as an industry and share – we have lots of industry associations and groups to help facilitate this! Many of the things facing one rare condition are similar to others. The diagnosis may relate to only a handful of people across the globe or hundreds living with a prevalent condition but there are common threads—if not necessarily within the science but definitely with others having navigated access. Let’s make sure we really explore the synergies and learn the lessons across disease areas and not recreate the wheel. Yes protect your asset, be cognisant on what you can and can’t share from a competition law perspective, but do publish on how things are achieved and write case studies on your hurdles as well as successes. So often I hear teams only looking at their area of interest and being protective or silent in meetings. We have more in common than less. Lets use that strength. The changes with European HTA would be a great place to start as the first companies go through this.
- Standardisation – how a product rolls out into routine clinical practice starts at phase II clinical trials (if not before). We need to work stronger cross functionally internally to consider the consequences of vial size, freezer temperatures, cartridge sizes, formulation etc for the pharmacists and nurses who have to administer them locally at provider/hospital level. The more we can ensure what is being done in the clinical trial setting is practical for day to day use the better. I’d highly encourage engagement directly with pharmacists and nurses early and if it’s a treatment that will be given by a friend or family member at home please involve the patient community in discussions. Then having done that can we somehow look at what other companies are doing so there isn’t unnecessary variation. A 2-degree difference could result in a whole new fridge or freezer being required.
- Plan for commercialisation early – its hard as every step in the development process is expensive and time consuming and neither pharma or providers want to invest time or money planning for something that may not happen. That said there are clear milestones through the licensing process and strong indicators of how reimbursement discussions are evolving to gatekeep against. Commercialisation is complex and goes way beyond getting a yes from NICE or their equivalents around the globe. There are some companies and therefore patients out there currently facing real delays in access.
Informing all stakeholders early, in the UK completing Pharmascan as a starting point, and then collaborating on an ongoing basis are all so important. The national commissioning teams and the commercial unit all offer surgeries to help build relationships and answer questions.
Think …..
- Are service agreements drafted and aligned so they can be signed quickly and rolled out?
- Has the hospital insurance been revisited to ensure it covers the new situation?
- How long will it take to get the product through ethics / drugs and therapeutics committees in each hospital?
- How often do patients need to actually visit the hospital or can shared care protocols and more local outreach clinics help reduce the burden of travel?
- Is the network between clinical teams across hospital trusts (or even between departments within the same trust) well established and referral processes defined and rolled out?
- If the staff aren’t budgeted for, recruited and in place at the point of reimbursement who will actually discuss the product with families, consent patients and administer treatment?
- Will it impact fertility services?
- Does pharmacy have capacity to make up your product if it needs aseptic conditions?
- Has the relevant department initiating treatment informed and if necessary contracted with their pharmacy, pathology, phlebotomy service for extra work, tests, etc?
It may be unrealistic to have all this in place on day one (or, as legal expectations suggest, day 60/90 if in Wales/England) but do we know during negotiations what reality looks like. Are we starting early enough? As a country team are you managing expectations internally up the ladder accordingly? Don’t forecast sales to start 90 days post NICE if it’s going to take another six months for the service or you miss your targets and potentially waste money on manufacturing/production costs.
More importantly does the patient community know when practically treatment might be available? And jointly with providers, NHS decision makers and the patient groups can we do anything to speed this up? Today there are examples out there where its taking too long. One would hope if a provider organisation pitches to provide or advertises in some countries to provide a new innovation that they are driving this equally alongside the company and the community.
- Working with patient organisations –we can still do better. There is never a valid ‘too early’ response. In an ideal world we design trials that meet the needs of those living with a particular condition and ensure that we not only meet the regulator and reimbursement needs but that the product we deliver is both wanted and fit for purpose. Thankfully the regulators are now starting to drive this more overtly, with them consulting the community to a greater degree than in the past. Understanding who will want, how quickly, when it will fit alongside other treatment options are all as important as the why they might want it. If the story doesn’t hang together well or has holes in it people get distracted, disorientated or loose interest!
Our patient information also needs to be validated and written so its easily understood, translated into multiple languages and easily accessible. Who better to help prepare it than the groups themselves and yet I hear of companies still doing in isolation. The patient groups are our strongest allies and support if we truly collaborate, listen and involve. At the end of the day they want these new treatment options more than any of us who work for the organisations.
- Raise our voices – in many countries we can not promote to the public and I personally believe that is a good thing as it adds a level of protection for the individuals, especially at a time when people can be vulnerable, and ensures conversations happen between families and the clinical team. We can however speak more overtly in my opinion on what good the industry does for society from both an emotional, practical and financial perspective. Do politicians, decision makers and the public really appreciate the positive impact of the industry on the health of a nation. Most individuals I know utilise a medicine that the industry has developed—the pain killer for a headache, an antihistamine for hay fever, Savlon for a cut…. All things used in my household this weekend, alongside my mums Alzheimer’s medication, her thyroid tablets ,without which, she is a zombie, my dads blood pressure tablets…. The list goes on. We play a positive contribution in society that is often under represented. In rare diseases especially we are at the brink of some amazing life changing innovations and personalised medicine approaches which are ground breaking. You may be working on one specific rare condition but together we are many and we need to harness that voice.
In writing this I’ve made myself more energised. When we pause to catch breath and think there are still so many areas we can collaborate on and improve. Yes I’d like to think the new government will roll out an amazing plan this spring to address all the failings in our health system and that the amazing staff who work there will get the support, resources and recognition to do what they aspire to. Even more so I would love to see rare disease highlighted as a priority and recognised as important in that plan. I’m sure we can jointly raise our voices—what ever hat you are wearing as you read this – and continue to shine a light on the challenges the rare community faces as we did on Feb 28th. It’s the day-to-day improvements and choices though that can equally positively impact how life is for so many thousands. As you read this please stop and consider just one change you can embrace and champion that might change the direction of travel.
About Nicola
Nicola set up NJ Redfern Ltd in 2022 to enable her to consult, coach and collaborate with other organisations, following her experience at bluebird bio. She has a long history in rare disease and oncology within the pharmaceutical and biotech industry and has worked closely with the Cell and Gene Therapy Catapult and the ATTC network in the UK. She is a member of the International Society of Cell and Gene therapy (ISCT) ethics sub group, contributes to the UK ATMP Engage community and projects, and speaks at various congresses and webinars about the challenges of bringing cell and gene therapies to market.
Nicola is committed to and motivated by ensuring people living with significant health challenges have a choice and are able to access transformative treatment options quickly once science and innovation reaches a point to positively impact their lives. She also believes the UK will be stronger if these new treatments are supported and embraced holistically across our ecosystem.
She can be contacted at nickiredfern@gmail.com or connect with her on LinkedIn.
