Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American
Posted: August 9, 2017 at 4:48 am
By Ben Hirschler
LONDON (Reuters) - The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe.
Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States.
Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases.
After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years.
In the past five years, European regulators have approved two gene therapies - the first of their kind in the world, outside China - but only three patients have so far been treated commercially.
UniQure's Glybera, for a very rare blood disorder, is now being taken off the market given lack of demand.
The future of GlaxoSmithKline's Strimvelis for ADA-SCID - or "bubble boy" disease, where sufferers are highly vulnerable to infections - is uncertain after the company decided to review and possibly sell its rare diseases unit.
Glybera, costing around $1 million per patient, has been used just once since approval in 2012. Strimvelis, at about $700,000, has seen two sales since its approval in May 2016, with two more patients due to be treated later this year.
"It's disappointing that so few patients have received gene therapy in Europe," said KPMG chief medical adviser Hilary Thomas. "It shows the business challenges and the problems faced by publicly-funded healthcare systems in dealing with a very expensive one-off treatment."
These first two therapies are for exceptionally rare conditions - GSK estimates there are only 15 new cases of ADA-SCID in Europe each year - but both drugs are expected to pave the way for bigger products.
The idea of using engineered viruses to deliver healthy genes has fuelled experiments since the 1990s. Progress was derailed by a patient death and cancer cases, but now scientists have learnt how to make viral delivery safer and more efficient.
Spark Therapeutics hopes to win U.S. approval in January 2018 for a gene therapy to cure a rare inherited form of blindness, while Novartis could get a U.S. go-ahead as early as next month for its gene-modified cell therapy against leukaemia - a variation on standard gene therapy.
At the same time, academic research is advancing by leaps and bounds, with last week's successful use of CRISPR-Cas9 gene editing to correct a defect in a human embryo pointing to more innovative therapies down the line.
Spark Chief Executive Jeffrey Marrazzo thinks there are specific reasons why Europe's first gene therapies have sold poorly, reflecting complex reimbursement systems, Glybera's patchy clinical trials record and the fact Strimvelis is given at only one clinic in Italy.
He expects Spark will do better. It plans to have treatment centers in each country to address a type of blindness affecting about 6,000 people around the world.
Marrazzo admits, however, there are many questions about how his firm should be rewarded for the $400 million it has spent developing the drug, given that healthcare systems are geared to paying for drugs monthly rather than facing a huge upfront bill.
A one-time cure, even at $1 million, could still save money over the long term by reducing the need for expensive care, in much the same way that a kidney transplant can save hundreds of thousands of dollars in dialysis costs.
But gene therapy companies - which also include Bluebird Bio, BioMarin, Sangamo and GenSight - may need new business models.
One option would be a pay-for-performance system, where governments or insurers would make payments to companies that could be halted if the drug stopped working.
"In an area like haemophilia I think that approach is going to make a ton of sense, since the budget impact there starts to get more significant," Marrazzo said.
Haemophilia, a hereditary condition affecting more than 100,000 people in markets where specialty drugmakers typically operate, promises to be the first really big commercial opportunity. It offers to free patients from regular infusions of blood-clotting factors that can cost up to $400,000 a year.
Significantly, despite its move away from ultra-rare diseases, GSK is still looking to use its gene therapy platform to develop treatments for more common diseases, including cancer and beta-thalassaemia, another inherited blood disorder.
Rivals such as Pfizer and Sanofi are also investing, and overall financing for gene and gene-modified cell therapies reached $1 billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.
Shire CEO Flemming Ornskov - who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder - sees both the opportunities and the difficulties of gene therapy.
"Is it something that I think will take market share mid- to long-term if the data continues to be encouraging? Yes. But I think everybody will have to figure out a business model."
More here:
Gene Therapy Is Now Available, but Who Will Pay for It? - Scientific American
- This Pitt program is leveling up the gene therapy workforce in Pittsburgh - University of Pittsburgh - March 12th, 2024
- Opinion | Banning Gain-of-Function Research Would Do Far More Harm Than Good - Medpage Today - March 12th, 2024
- The Biggest Challenges Facing Broader Adoption of Novel Cell and Gene Therapies - Pharmaceutical Executive - March 12th, 2024
- Research and development areas of focus - Bristol Myers Squibb - March 4th, 2024
- Overcoming the challenges of gene therapy - Drug Discovery News - March 4th, 2024
- Breakthrough Gene Therapy for LHON: Vision Restoration in Focus at Medical Conference - BNN Breaking - March 4th, 2024
- Revolutionizing Healthcare: Global Gene Therapy Market Surges to US$ 3.44 Billion by 2022 As Revealed In Ne... - WhaTech - March 4th, 2024
- Looking to the future of clinical trials: Gene therapy, precision medicine, and the ongoing quest for rare disease solutions - pharmaphorum - March 4th, 2024
- Complement system response to AAV vector gene therapy - EurekAlert - February 23rd, 2024
- 10 gene therapy companies you should know about - Labiotech.eu - February 23rd, 2024
- Fetal surgery expert Tippi MacKenzie shifts to in-utero gene therapy - STAT - February 23rd, 2024
- Rejuvenate Bio Announces Gene Therapy-Mediated Partial Reprogramming Extends Lifespan and Reverses Age ... - Business Wire - February 23rd, 2024
- Combatting HIV using gene therapy - Drug Target Review - February 23rd, 2024
- Expanding the Horizons of Cell and Gene Therapy - RegMedNet - February 23rd, 2024
- Global Advanced Therapy Medicinal Products CDMO Market Analysis Report 2024-2030: : Increasing Investment and ... - Yahoo Finance UK - February 23rd, 2024
- 2023 gene therapy research STAR Grant winners announced - Pacific Biosciences - February 13th, 2024
- Lifetime Cost of DEB Gene Therapy Could Be $15-$22 Million - Medscape - February 13th, 2024
- 5 Things Youll Learn in Frederick Community Colleges New Cell and Gene Therapy Essentials Certificate ... - BioBuzz - February 13th, 2024
- Regenxbios investigational gene therapy shows promise in Hunter syndrome - PMLiVE - February 13th, 2024
- Topical Gene Therapy Effective in Treating Debilitating Eye Condition - InventUM - University of Miami - February 13th, 2024
- Bayer Says First Patient Randomized In Askbio Phase II Gene Therapy Trial - XM - February 13th, 2024
- Top 3 grants in regenerative medicine: January 2024 - RegMedNet - February 13th, 2024
- Realistic Expectations, Limitations With Gene Therapy for Parkinson Disease: Ignacio Mata, PhD - Neurology Live - February 5th, 2024
- Gene-Based Therapy May Slow Development of Life-Threatening Heart Condition - NYU Langone Health - February 5th, 2024
- After Promising Early Efficacy, Eli Lilly Eager to Study Hearing Loss Gene Therapy in More Children - precisionmedicineonline.com - February 5th, 2024
- Gene therapy hailed as medical magic wand for hereditary swelling disorder - The Guardian - February 5th, 2024
- France Approves Gene Therapy Clinical Trial to Treat Hearing Loss in Children - The Hearing Review - February 5th, 2024
- Early Trial Success for Inherited Deafness Gene-Therapy - RegMedNet - February 5th, 2024
- Can gene therapy be the key to restoring hearing loss? - Labiotech.eu - February 5th, 2024
- Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum ... - Business Wire - February 5th, 2024
- FDA inspection clears Abeona's gene therapy BLA By Investing.com - Investing.com - February 5th, 2024
- Hearing Restored in Deaf Children in Gene Therapy Clinical Trial - Securities.io - February 5th, 2024
- Experimental gene therapy allows kids with inherited deafness to hear - The Associated Press - January 28th, 2024
- Children with genetic deafness have hearing restored with gene therapy: Study - ABC News - January 28th, 2024
- Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at ... - Investors | Eli Lilly and... - January 28th, 2024
- UChicago Medicine among the first in the country to offer newly approved sickle cell gene therapies - UChicago Medicine - January 28th, 2024
- Gene Therapy Allows an 11-Year-Old Boy to Hear for the First Time - The New York Times - January 28th, 2024
- Gene therapy restores hearing in children with hereditary deafness - EurekAlert - January 28th, 2024
- Experimental gene therapy helps treat kids with inherited hearing loss - CBS News - January 28th, 2024
- Children's Hospital of Philadelphia Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss - PR Newswire - January 28th, 2024
- Gene Therapy Brings Hearing to Kids With Congenital Deafness - U.S. News & World Report - January 28th, 2024
- Gene Therapy Restores Hearing in Small Trial for Kids With Hereditary Deafness - Technology Networks - January 28th, 2024
- Cell Therapies on Ice: Seven Trends in Cryopreservation To Enable Cell and Gene Therapy Research - Technology Networks - January 28th, 2024
- Gene Therapy Brings Hearing to Kids With Congenital Deafness - HealthDay - January 28th, 2024
- Gene therapies restore hearing in several kids with inherited deafness - Livescience.com - January 28th, 2024
- 11-Year-Old Boy Hears For First Time Ever Thanks To Gene Therapy Breakthrough - IFLScience - January 28th, 2024
- Innovative Gene Therapy Restores Hearing In Deaf Child, Offering Potential Cure - Study Finds - January 28th, 2024
- Sound of Success, Gene Therapy Breakthrough Grants Hearing to Deaf Children in China-Harvard Study - Hoodline - January 28th, 2024
- Perspectives in FTD Research Webinar: Gene Therapy for FTD -- What Do I Need to Know? | AFTD - AFTD - January 20th, 2024
- UK leads the way in cell and gene therapy - Research Professional News - January 20th, 2024
- Charles River's (CRL) New Launch to Boost Gene Therapy Programs - Yahoo Finance - January 20th, 2024
- UK maintains status as an attractive destination for advanced therapy clinical trials - BioPharma-Reporter.com - January 20th, 2024
- Understanding the impact of AI on the advancement of gene therapy development - pharmaphorum - January 20th, 2024
- Gene therapy TSHA-102 delivered to 1st child in REVEAL trial - Rett Syndrome News - January 20th, 2024
- AAV Vectors in Gene Therapy Market is Predicted to Observe Skyrocketed Growth During the Study Period (2019-2032 ... - PR Newswire - January 20th, 2024
- Family pins hopes on gene therapy - The Recorder and Times - January 20th, 2024
- Arbor, 4DMT partner on new gene therapies in ALS, other diseases - ALS News Today - January 11th, 2024
- The year of gene therapy? FDA's Marks calls for solutions to regulatory, manufacturing challenges - BioWorld Online - January 11th, 2024
- Gene Therapy Approvals Expected to Ramp Up in 2024 Amid Manufacturing, Cost Challenges - BioSpace - January 11th, 2024
- Turning On Gene Therapy with the pA Regulator - RegMedNet - January 11th, 2024
- Revolutionizing Healthcare: Gene Therapy Market Projected to Reach USD 46.5 billion by 2030 at a 22. - PharmiWeb.com - January 11th, 2024
- Regeneron CEO Reveals the Game-Changing Potential of Gene Therapy in Biotech! - BioTecNika - January 11th, 2024
- Bayer's AskBio checks off another early win for gene therapy platform with Parkinson's safety data - FierceBiotech - January 11th, 2024
- Are Rare Brain Diseases the Next Commercial Frontier for Gene Therapy? - MedCity News - January 3rd, 2024
- Novartis, Voyager Ink Up-to-$1.3B Expansion of Neuro Gene Therapy Collaboration - Genetic Engineering & Biotechnology News - January 3rd, 2024
- Novel switch turns genes on/off on cue, a promising step toward safer gene therapy - Phys.org - January 3rd, 2024
- Which Therapeutic Areas Experts Think Will Gain Traction In 2024 - Clinical Leader - January 3rd, 2024
- Voyager Therapeutics and Novartis Announce Major Licensing Deal in Gene Therapy Field - Medriva - January 3rd, 2024
- We Are About to Enter the Golden Age of Gene Therapy - Inverse - January 3rd, 2024
- Revolutionizing Gene Therapy: Baylor Researchers Develop RNA-Based Switch for Therapeutic Protein Control - BNN Breaking - January 3rd, 2024
- Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease - Yale Medicine - December 25th, 2023
- Top story: Family connects with researchers behind Canavan gene therapy - UMass Medical School - December 25th, 2023
- Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy - AJMC.com Managed Markets Network - December 25th, 2023
- Hemgenix Gene Therapy Shows Long-Term Efficacy, Safety in Hemophilia B Patients - Managed Healthcare Executive - December 25th, 2023
- The missing piece of the puzzle - EUobserver - December 25th, 2023
- Brace yourselves for these trends in clinical research in 2024 - Labiotech.eu - December 25th, 2023
- RegMedNet's 2023 remix: top content of the year - RegMedNet - December 25th, 2023
- BU Researchers Helped Develop the First FDA-Approved Gene Therapies to Treat Sickle Cell Disease - Boston University - December 16th, 2023
- Research discovers that mice possess a natural gene therapy system - Phys.org - December 16th, 2023
- Cell- and gene-therapy hub bound for Lake Success; New York to invest up to $150 million - Newsday - December 16th, 2023