Market Access for Cell and Gene Therapies

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Publication Date:
February 2020
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Pharma challenged to rethink its market access strategies

While the promise of cell and gene therapy (C&GT) to deliver enduring or even curative treatments is welcomed there is no long-term market experience and early lessons with these complex therapies suggest that pharma must ramp up its market access game. Ensuring availability of product, supporting clinical delivery, educating HCPs and harnessing the patient voice all play a critical role in building stakeholder confidence and supporting pharma's pricing and reimbursement ambitions.

With over 200 C&GT products in the pipeline, we are just at the beginning of the sector's evolution with these revolutionary therapies. Pharma must learn from real world experience and that is why, in February 2020, we interviewed C&GT experts in Market Access for Cell and Gene Therapies. They reveal the trends and issues that are shaping this fast developing sector and the unique suite of market access obstacles that pharma must overcome.

C&GT experts explore key questions...

  • It's not just about the price—what pill-plus services will gain payer support?
  • What market access lessons can be learnt from products already launched?
  • How can pharma benefit from early patient engagement to manage expectations, build registries and identify appropriate clinical outcomes in the long-term?
  • What role can pharma play in ensuring the establishment of specialist treatment centres for the safe administration of C&GT products?
  • How might technologies for improving shelf life and the logistical management of therapies play out in your market access strategy?
  • Pay for performance and amortised contracting are great ideas but can they ever be a practical payment mechanism in the C&GT space?

What C&GT experts say

"The world of medicine is changing, requiring an increased number of interactions with payers that must include education, for example on how to demonstrate benefit and justify costs. Once approvable endpoints change, the discussion with payers will have to change; that at times can be a good thing but sometimes it makes the conversation more difficult. A traditional market access person will always ask their development teams to provide data and lots of data. One challenge is that novel treatments will get approved when much of the data still has to be generated in years of long-term monitoring. Products can get approved on short term readouts, but reimbursement needs to be based on long-term benefit. That scenario can make the life of a market access person quite challenging."
Vered Caplan
Chairman & CEO, Orgenesis

"Typically, therapy producers will have manufacturing centres located in key geographical locations e.g. U.S. and Europe. So if a U.S. based centre is closed for some reason, there is an opportunity to divert production through to Europe. There are certain challenges associated with this because you have to move product through customs clearance twice. A further challenge associated with this strategy is the additional time it adds to the supply chain. For example, if a deep clean of cleanrooms is scheduled, those days will not be available for manufacture of the product and clinical centres can see this in TrakCel's scheduling tool. Potentially, if they have a very sick patient, there is an option to move manufacturing outside of the geographical location. But there are certain risks associated with any supply cycle changes; any change would be addressed with a risk based approach."
Matthew Lakelin
VP Scientific Affairs and Business Development, TrakCel

"At the ISCT, I've restructured to have market access and we call it Market Access and Patient Association, MAPA. I think that the market access ends in the patient. But consequently should start on the patient as well. We should think how this is going to be used in the patient, and educate the patient and bring the patients in. If we look at Duchenne, for instance, the regulators and physicians and developers were at one point very locked on the six minute walk test but patients when they're asked say, well I'd like to walk, but even before I'd walk I'd like to be able to use my arms. If I don't walk I'm sitting down, my arm function is even more important than walking. It's important early on to understand what the patients want and need. And then tell them that we can deliver that or not, and what we deliver instead. That dialogue with the patient must start very early on, in the development and clearly on setting the expectations, not in a paternalistic way, but through information to protect their expectations regarding what these products can and cannot do. This is an absolute must."
Miguel Forte
CEO, Bone Therapeutics

What to expect

A detailed report exploring the unique market access issues that pharma must address when bringing C&GT products to market

  • An examination of the 4 key C&GT issues that impact market access
  • 20 targeted questions to C&GT experts
  • Their responses which provided 23 insights supported by 125 directly quoted comments

Expert contributors

The contributors to this report are C&GT experts that have experience with development, manufacturing, pricing and reimbursement and providing access to these innovative treatments.

  • Alengo Nyamay'Antu, PhD, Scientific Communication Specialist at Polyplus-transfection S.A. Alengo is a scientific advisor in the biotechnology industry and highly-skilled in protein biochemistry, cell biology and biomedicine.
  • Claudia Zylberberg, PhD, is a leader in regenerative medicine. She is the founder and CEO of Akron Biotechnology, a manufacturer of cGMP-grade ancillary materials for the tissue, cell and gene therapy industry. She also co-founded AssureImmune, an adult stem cell bank. Dr. Zylberberg holds numerous patents and has developed several patent-pending platform technologies in cryopreservation, novel formulations and others. She has authored and co-authored several peer-reviewed publications and has received grants from the NIH and Department of Defense, among others.
  • Debra Susarchick, Chief Product Officer, TrakCel, is a senior business and technology leader with broad experience delivering commercial and supply chain programs in the pharmaceutical and biotechnology industries. Prior to joining TrakCel, Debra worked at Kite Pharma, where she was on the global launch team for YESCARTA. She has worked in the pharmaceutical industry since 1996, initially at Merck and Co and then moved into the oncology space where she worked at Celgene within their oncology division.
  • Matthew Lakelin, PhD., VP Scientific Affairs and Business Development at TrakCel, has worked at the company since it was founded in 2012. Prior to TrakCel Matthew worked for a special distribution company that specialised in pharma products, clinical trials, and ATMP products. He is a pharmacologist by training and has been working with sales and pharma products throughout his career.
  • Miguel Forte, PhD., is currently Chief Executive Officer at Bone Therapeutics, effective 1 January 2020. Dr. Forte has significant experience in regenerative medicine and in the cell therapy industry, most recently as Chief Executive Officer of Zelluna Immunotherapy. He is currently also serving as Chief Commercialization Officer and Chair of the Commercialization Committee of the International Society of Cellular Therapy. Dr. Forte held in the past a senior position at the European Medicines Agency, was Vice-President Global Medical Affairs Inflammation at UCB, Chief Medical Officer at TxCell.
  • Simon Ellison, Cell and Gene Therapy Service Director, World Courier. His role is to develop a portfolio of global logistic and supporting service strategies that take the advanced therapy industry forwards. Whilst at the Cell and Gene Therapy Catapult he identified the need for, and built the Seamless Freight portfolio. Prior to this Simon was the Head of Commercial at NHS Blood and Transplant (NHSBT) where he merged the needs of the industry with NHSBTs manufacturing and supply chain capability.
  • Vered Caplan, Chairman & Chief Executive Officer for Orgenesis, Inc.,Chairman of ATVIO Biotech Ltd and Chairman of the Board of MaSTherCell Global, Inc. (both are subsidiaries of Orgenesis, Inc. Previously she held the position of Chief Executive Officer at Kamedis and Gammacan International, Inc. Ms. Caplan received a graduate degree from Tel-Aviv University and an undergraduate degree from Technion-Israel Institute of Technology.
  • Anonymous. A CDO, leading North America, cell therapy company with more than 25 years of experience in the biopharmaceutical industry developing products to treat rare disease and for multiple disease areas, including haematology and ophthalmology.

Why choose FirstWord ExpertViews

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  • Are based on primary research with experts whose knowledge and current experience is proven
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