On 17 May 2017, I attended the first day of the World Advanced Therapies and Regenerative Medicine Congress in London. It was an exciting day with an eclectic audience from large pharma, growing biotechs, tools and reagents companies, regulators, service providers, and academia and government. Everyone represented is united in their quest to make regenerative medicine a reality.
For those who are not familiar with this sector, regenerative medicine is believed, by many, to be the future of medicine and healthcare. It has the potential to cure indications from cancers to rare diseases. There are over 730 companies currently active in this sector, and over 800 clinical trials are taking place around the world. Big pharma have been keeping a watchful eye on the developments in the sector and slowly but surely showing interest in getting in on the action. Sven Kili, Vice President, Head of Cell and Gene Therapy Development at GlaxoSmithKline (GSK), spoke about where GSK is with its gene therapy pipeline and what they’ve learned from taking the gene cell therapy Strimvelis to market. Hopefully, these learnings will pave the path for more therapies reaching the market and more indications being targeted.
Throughout the day, I kept thinking back in 2012-2013 when I started getting more involved in this sector through delivering a regenerative medicine strategy project for a PA client. Then, as now, I heard people asking the following questions:
- Can we advance manufacturing enough to achieve automation and other innovations that enable scale up (allogeneic therapies) and scale out (autologous therapies)?
- How do we reduce the Cost of Goods Sold (COGS)?
- How do we work with regulators better to get these therapies through to market and patients? And how do we navigate the different regulatory pathways in the various geographies?
- Will these therapies be reimbursed? Who will pay for them?
Thankfully, I was reassured by a number of conversations at the Congress that we have been making progress in this sector and we ARE learning and putting those learnings to good use:
- The right discussions and collaborations are happening to advance the sector. It’s no secret that no one single organisation is going to solve these challenges on their own. There will always be gaps to be filled by bringing in the right expertise from outside the organisation, be they academia, consultants, and/or other pharma, biotech, and tools companies. Thermo-Fisher Scientific, for example, is investing in an innovation programme with a network consisting of academia and industry with a goal to address the challenges facing cell therapy
- Some companies, such as GSK, are sharing their learnings, which will hopefully benefit all those following the same path of trying to take these therapies to market. And it’s good to see that the difficulties facing one product’s journey towards the patient are not experienced in vain
- A clear message emerged around ensuring there is a solid case for these therapies, medically and economically. This is important in order to make sure patient safety is always at the front and society is on board with paying for these therapies. I particularly liked William Burns’s reassuring reference that ‘good medicine is always good business’
- The regulators are talking to each other. That’s a good start. The lack of harmonisation across regulators is a key problem and talking is a starting point towards achieving more consistency and clearer messaging across markets
- Some regulators are even leading the way by providing a myriad of ways to support companies in this sector. In his talk, Dr. Ian Hudson, CEO of the Medicines and Healthcare Products Regulatory Agency (MHRA) detailed all of the support mechanisms available for companies seeking access to the UK market (eg The ‘one stop shop regulatory advice service for regenerative medicine’). Participants also discussed Japan’s innovative approach to regulation of advanced therapies. For example, conditional approvals in Japan allow therapies to be used on patients with life threatening, debilitating conditions with no available alternative treatment. This conditional approval also allows companies to gather the real world evidence they need to prove the effectiveness of their products and obtain full approval. Europe has a similar conditional approval system in place. However, Japan goes one step further in that conditionally approved therapies are eligible for reimbursement
- Many are working towards reducing COGs which in the medium to long term will hopefully make the economic case clearer and reimbursement less challenging.
For me, the key take home message is that collaboration and consolidated effort is key to making regenerative medicine a reality. As ever, this is an exciting sector and the amount of activity from a wide range of players is encouraging. I just hope that – in addition to talking about the challenges that are stopping the sector from advancing as quickly and safely as we want it to – the various stakeholders work ever closer together, share more and continue to learn from each other.