The actual report (not linked to by CNBC of course) says
The potential to deliver “one shot cures” is one of the most attractive aspects of gene
therapy, genetically-engineered cell therapy and gene editing. However, such treatments
offer a very different outlook with regard to recurring revenue versus chronic therapies,
particularly in certain diseases where it is possible to cure a large proportion of the
prevalent patient pool (or at least prevent an additional dose from being required for an
extended period). While this proposition carries tremendous value for patients and
society, it could represent a challenge for genome medicine developers looking for
sustained cash flow. GILD is a case in point, where the success of its hepatitis C
franchise has gradually exhausted the available pool of treatable patients.
We highlight several potential solutions for these genome medicine companies to
sustain an attractive profile.
Solution 1: Address large markets: Hemophilia is a $9-10bn WW market (hemophilia A,
B), growing at ~6-7% annually. We estimate that the hemophilia A market currently
represents ~$6.5bn, while hemophilia B is ~$1.2bn.
Solution 2: Address disorders with high incidence: Spinal muscular atrophy (SMA)
affects the cells (neurons) in the spinal cord, impacting the ability to walk, eat, or
breathe. It is the leading genetic cause of death in infants. SMA affects ~1 in 11k
babies, and 1 in 50 individuals in the US is a genetic carrier. Cancer is also a sustainable
market given the patient population is almost entirely incident driven.
Solution 3: Constant innovation and portfolio expansion: There are hundreds of
inherited retinal diseases (genetics forms of blindness). Once a gene therapy is
approved for a genetic eye disease, the validated platform could be used to quickly
develop many more eye-based gene therapies. Pace of innovation will also play a role as
future programs can offset the declining revenue trajectory of prior assets.
or innovators dilemma upsets or forces the incumbents. the transformer (attention is all you need) model paper did release in 2017 and Google did nothing about it until OpenAI forced their hand
awaiting this happening to pharma as well as new technology reduces the barrier to entry for innovators
No, it’s saying there is a limited part of the drug market where curing may not be as profitable as maintenance. So they’re trying to find alternate ways of financing that development. For most of pharma, curing is a payday.
I think it is good and honest for companies to acknowledge that the current incentives work against trying to achieve [SOME_GOOD_RESULT], as in that case the first step towards getting that thing done would be figuring out how can we change the money flow so that there would actually be motivation for companies to direct their R&D efforts towards that.
If the society says to manufacturer "if you do this, you'll get less money from us" that is effectively the society saying "please don't do this". People and governments vote with their wallets about what they want, and if they're "voting wrong" or ineffectively, then they need to change what they pay for, instead of being surprised that they don't get what they disincentivize.
The potential to deliver “one shot cures” is one of the most attractive aspects of gene therapy, genetically-engineered cell therapy and gene editing. However, such treatments offer a very different outlook with regard to recurring revenue versus chronic therapies, particularly in certain diseases where it is possible to cure a large proportion of the prevalent patient pool (or at least prevent an additional dose from being required for an extended period). While this proposition carries tremendous value for patients and society, it could represent a challenge for genome medicine developers looking for sustained cash flow. GILD is a case in point, where the success of its hepatitis C franchise has gradually exhausted the available pool of treatable patients. We highlight several potential solutions for these genome medicine companies to sustain an attractive profile. Solution 1: Address large markets: Hemophilia is a $9-10bn WW market (hemophilia A, B), growing at ~6-7% annually. We estimate that the hemophilia A market currently represents ~$6.5bn, while hemophilia B is ~$1.2bn. Solution 2: Address disorders with high incidence: Spinal muscular atrophy (SMA) affects the cells (neurons) in the spinal cord, impacting the ability to walk, eat, or breathe. It is the leading genetic cause of death in infants. SMA affects ~1 in 11k babies, and 1 in 50 individuals in the US is a genetic carrier. Cancer is also a sustainable market given the patient population is almost entirely incident driven. Solution 3: Constant innovation and portfolio expansion: There are hundreds of inherited retinal diseases (genetics forms of blindness). Once a gene therapy is approved for a genetic eye disease, the validated platform could be used to quickly develop many more eye-based gene therapies. Pace of innovation will also play a role as future programs can offset the declining revenue trajectory of prior assets.