Peter Marks, a top official at the Food and Drug Administration, is not tasked with determining if the expense of the medicines his teams are reviewing is justifiable. However, he is concerned that if businesses can’t figure out how to market them, some of the drug industry’s most promising medications will not reach patients with rare diseases.
There are thought to be about 7,000 uncommon diseases, many of which only strike very tiny populations. A potent method of treating some of them, and perhaps even curing them, could be provided by genetic medicines, such as RNA-based medications and gene replacement therapies. However, for aspiring entrepreneurs, diseases that only affect a handful of people could not represent a sizable enough market to warrant the expense of creating and distributing a new cure.
At a conference organised by the Alliance for Regenerative Medicine on Wednesday, Marks, director of the FDA’s Center for Biologics Evaluation and Research, said they were not going to find sufficient charitable groups to cover the cost of gene therapies for the hundreds of thousands of different diseases that need to be addressed.
The speaker said that they need to figure out how to make this economically feasible so that industry can move toward this. Marks claims that in order for gene therapy to be commercially viable, 100 to 200 treatments must be given annually. For rare diseases like severe combination immunodeficiencies or adrenoleukodystrophies, this barrier may be challenging to meet in a single nation.
It has not slipped their notice at FDA that there have been some concerns about the future in gene therapy, said Marks, citing cases in which gene medicines were pulled from the market or given back to their creators by the academic researchers who first developed them.
For instance, two of the first gene therapies licenced in Europe—Strimvelis for the illness known as ADA-SCID—were abandoned by GSK and then Orchard Therapeutics. Only a small number of patients have ever received treatment, and Orchard has also returned the rights to the replacement therapy. Bluebird Bio recently withdrew two gene therapies from the EU market after having trouble getting reimbursement in a number of European nations.
Both of those treatments have just received FDA approval in the United States. The first, Skysona, which will be sold for $3 million, is for the genetic disorder known as CALD, which affects approximately 50 boys annually. According to Bluebird, it plans to treat about 10 patients annually.
Marks outlined a few areas where the FDA may assist in lowering barriers for therapies for ultra-rare diseases in his remarks at the event, known as the Meeting on the Mesa, which was well-attended by professionals in the field of cell and gene therapy.
The government is currently creating a cookbook for designing and producing customised gene therapies, which might make it simpler for academic research teams to transition their cures from the lab to the marketplace. Additionally, it is investigating how to speed up the approval of other applications that make use of the same technology by using manufacturing and non-clinical data from one application. Because they are utilised repeatedly, some components of gene therapies are not like your usual small-molecule medicine, according to Marks.
Another way to reduce production costs, which are much higher for gene and cell therapies than for other more widely used drug classes, is through automated manufacturing.
In order to provide developers with more assurance that a product they successfully obtain approval for in one country would also have a good chance of succeeding in other countries, the FDA is now attempting to align itself with other authorities. Some of these issues might be connected to how one can expand access to gene therapy for limited populations, according to Marks. What may be a small population in the United States becomes a sizeable number when one moves globally, the author says.