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Blog Carnival: Because we all want to see life-saving cell and gene therapies saving lives

by | Aug 21, 2019

Image by Keith Johnston from Pixabay

Cell and gene therapies are a game changer for patients. As academics and industry have been saying for years now, these therapies offer the promise of cures for previously incurable diseases and disorders.

There were over 1,000 clinical trials underway worldwide at the end of the first quarter of 2019.[1] The U.S. Food and Drug Administration (FDA) has approved 17 products, making it the “leader” in this area so far. (For more on the “why” of this, read Siofradh McMahon’s post below.) The FDA approvals include Kymriah and Yescarta to treat specific cancers, Luxturna to treat retinal dystrophy and Zolgensma for pediatric patients with spinal muscular atrophy. We are also seeing approvals happening in Canada and elsewhere, but not to this degree.

Approvals are a significant hurdle to overcome, but they are not the final barrier to patient access. As you will read in the following posts, from groups in Canada, the U.S., the U.K. and Australia, the challenges can be complicated and reflect the nuances of their regulatory and health-care systems, geographies, cost, clinical trials, the role of private insurance, and more.

If you’ve “participated” in previous blog carnivals with Signals, you’re already familiar with the format. (Or catch up here.) There is a theme – in this case it’s “What are the hurdles in your country impacting patient access to cell and gene therapies?” – and all of the participating experts answer that question from their own lens and experience. You are encouraged to read all the posts – this year there are five – to get the full picture and appreciate the different approaches to the topic.

For example, OIRM’s Krista Lamb writes about what her organization is hearing directly from patients in Losing Patience. CIRM’s Kevin McCormack also addresses patient issues in his post Time and money and advancing stem cell research.

Then we have Siofradh McMahon of CCRM, Chihwei Teng of CCRM Australia and Jacqueline Barry of Cell and Gene Therapy Catapult providing very comprehensive overviews of the regulatory and reimbursement complications in Canada, Australia and the U.K. respectively.

Please read:

Bringing innovative cell and gene therapies to Canadians

(Australia:) Adding CAR-T to Cart: What are the hurdles impacting patient access to cell and gene therapies?

What are the hurdles impacting patient access to cell and gene therapies in the UK?

After reading all these posts, you’ll likely come to the conclusion that we still have a long way to go. Nevertheless, patients should be excited about new therapies coming to market and take comfort in knowing industry, regulators, public and private payors, and advocacy groups are all working together to solve these problems so life-saving treatments can save lives.

If you have your own thoughts on this topic, please leave your comment below.

 

[1] Alliance for Regenerative Medicine Q1 2019 Data Report

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Stacey Johnson

Stacey Johnson is the editor of Signals and a contributor. For 25 years, Stacey has been providing strategic communications counsel to government, corporate, technology and health organizations. She began her career at the CTV Television Network and then moved to Hill & Knowlton Canada where she advised clients in a variety of industries and sectors. Stacey is the Vice President, Communications and Marketing for CCRM, a leader in developing and commercializing regenerative medicine-based technologies and cell and gene therapies. She has a Master's degree in Public Relations. You can follow her on Twitter @msstaceyerin.

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