Is cell therapy failing commercialization in Europe?

by Alexey Bersenev on August 19, 2016 · 2 comments

in business, regulation

Christopher Bravery – a consultant on advanced biologicals, has opened Cell Therapy Stream of the annual Bioprocessing Summit with worrisome note: “Half of approved cell and gene therapy products on European market eventually failed commercialization and were withdrawn“. Let’s look at it in details.

Since introduction of regulation cell/ gene therapy as Advanced Therapy Medicinal Products (ATMP) in 2007, seven (+1 to be) cell and gene therapy products were approved by EMA:

  1. Chondrocelect by Tigenix in 2009
  2. Glybera by uniQure in 2012
  3. MACI by Genzyme/ Sanofi/ Vericel in 2013
  4. Provenge by Dendreon in 2013
  5. Holoclar by Chiesi in 2015
  6. Imlygic by Amgen in 2015
  7. Strimvelis by GSK in 2016
  8. Zalmoxis by MolMed in 2016 (not finally approved yet as of today)

Now, let’s look at withdrawal history:

  1. MACI’s authorization was suspended in 2014 (1 year on a market)
  2. Provenge was withdrawn in 2015 (2 years on a market)
  3. Chondrocelect withdrawal is starting in 2016 (7 years on a market)

So, 3/4 (75%) purely cell-based ATMPs or 3/7 (43%) of all approved ATMPs were withdrawn in the last 3 years! We can exclude Zalmoxis, which yet to be approved, and statistics will be even worse. We can also exclude Glybera, which was administered only once in 3 post-approval years and considered as commercial flop, and statistics will be much worse. So, is this the solid case of failed ATMPs commercialization? Yes, at this point it is. I’m emphasizing “at this point”, because (i) 7 approved products is not big number for statistical analysis and (ii) 4 products were approved in the last one year and half – not long enough for analysis of market performance.

The question is WHY? Why commercialization of cell-based products is failing? Christopher Bravery asked the audience: “Do we focus too much on regulatory approval, but not enough on commercialization?” Yet another question he asked: “Are we choosing right indications?”. Of course, it is important to analyze each case of failed commercialization and post-marketing adoption. As far as I know, all 3 mentioned withdrawals were initiated by companies-manufacturers, but not by EMA. So, nothing wrong was with safety of these ATMPs. Also, at time of approval these ATMPs were deemed as “efficacious” in intended applications. However, the market is harsh. Sales were not as good as expected and companies were making business decisions. Were developers over-optimistic at the time of approval? I think so. Didn’t they account for competitors and reimbursement issues? I think so. In any case, as of today seem like most of cell and gene therapies in Europe are not commercially viable. We have a lot of lessons to learn from these failures.

So, as of today regulatory approval in cell/ gene therapy doesn’t mean commercial viability. I’d like to encourage you to discuss reasons for commercial failures. Also, please post all links to failure analysis, posted somewhere on a web. We all have to learn.

PS 1: At the time of writing this post, by some reasons, all information, related to Chondrocelect was absent on EMA web-site.

{ 2 comments… read them below or add one }

van Seek August 31, 2016 at 6:44 am

Well, It could be that companies and scientist driving the introduction of new cell therapies on the market are very enthusiastic during the development and see the approval as the “final goal”. However, they start to think too late about optimaliztion of their cell manufacturing processes so they are commercially viable. Or it could be, that the optimization of the proces is simply too risky (the product would chang which unacceptable during late phase trials) and therefore the product/proces is not fully optimized before reaching the market.
Other thing could be that the real life thereputic efficacy is badly reflected in cell therapy clinical trials which than translate in therapetic failures…. maybe doing clinical trials with personalized medicine is different from conventional
drugs…

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james richardson February 3, 2017 at 7:20 pm

Dear Alexey,
This is very important information to our public, and their policy-makers.

Perhaps the approach is wrong. We would not expect blood product supplies to be provided at a profit.
Academic units have relatively high success rates in providing cells and running clinical trials. Perhaps this is the future, and can be supported with more appropriate regulatory field as they do not have the commercial drive to cut corners and make a profit. Also the academic units have reputations to maintain and continue their ability to provide care in the regenerative medicine field.

Do you have information on the relative success of academic cell providers?

The public are hoping that new treatments can be delivered, especially through autologous cells. companies hoping for profit tend to look at allogenic cells. I see the future in autologous, especially as so many Chinese have cord cells stored from birth. In due course there will be a demand for proven uses of these cells. Why not develop services for these cells through academic and medical aspirations to help people , rather than depend on the profit motive?

It will be hard work, with personal attention to safety and individual patient needs, but this is what surgery does.

James B Richardson

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