Gene therapy access and affordability

Addressing the challenge globally

Here are two articles on this topic …

As a top regulator at the Food and Drug Administration, Peter Marks is worried that some of the drug industry’s most promising medicines may not reach patients with uncommon diseases if companies can’t figure out how to sell them.

Peter Marks

There are an estimated 7,000 rare diseases, many of which affect only small groups of people. Genetic medicines, including RNA-based drugs and gene replacement therapies, could offer a powerful way to treat, and potentially even cure, some of them. But for would-be developers, diseases affecting only a few dozen people might not represent a large enough market to justify the cost of developing and selling a new treatment.

According to Marks, commercial viability for a gene therapy means administering roughly 100 to 200 treatments a year, a threshold that could be difficult to clear in a single country for rare conditions.

Marks noted instances when gene therapies were taken off the market or returned by their developers to the original academic researchers.

In Europe, for example, first GSK and then Orchard Therapeutics abandoned one of the first gene therapies approved there, a treatment called Strimvelis for a condition known as ADA-SCID. Only a few dozen patients were ever treated, and Orchard has also handed back rights to a successor treatment. More recently, Bluebird bio withdrew two gene therapies from the EU market after running into difficulties securing reimbursement in several European countries.

Bluebird recently won FDA approval for both of those therapies in the U.S. One, to be sold as Skysona at a cost of $3 million, is for an inherited condition known as CALD that affects about 50 boys each year. Bluebird has said it expects to treat around 10 each year.

In his remarks to the conference, known as the Meeting on the Mesa and attended by many in the cell and gene therapy field, Marks highlighted a few areas where the FDA could help ease hurdles for ultra-rare disease treatments.

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SOURCE: BIOPHARMA DIVE Published Oct. 13, 2022

We have to find a way: FDA seeks solutions to aid bespoke gene therapy

Ned Pagliarulo, Lead Editor

A new consortium tackling the growing economic hurdles that prevent life-saving gene therapies from reaching children who need them the most has been co-founded by a UCL researcher.

Claire Booth

AGORA (Access to Gene Therapies for Rare Disease) launched 22 September at London’s Great Ormond Street Hospital for Children (GOSH) brings together stakeholders from across the UK and Europe who represent academic groups, regulators, funders, patient advocacy groups and drug developers.

It aims to create a sustainable solution for children with rare and ultra-rare diseases to access novel gene therapies that are shown to be effective in clinical trials.

Although there are currently over 140 clinical trials for ultra-rare diseases taking place, only a tiny percentage have resulted in developed, marketed or registered treatment currently available for patients.

Co-founding member of AGORA, Professor Claire Booth (UCL Great Ormond Street Institute of Child Health & GOSH), said “It is vital that we continue to offer these treatments to patients in need and not let decades of research and huge financial investment go to waste. We can’t just accept that it’s too costly to treat these children”.

The first successful gene therapy was administered in 1990 Since then, around 150 gene therapy clinical trials have been undertaken for an estimated 500 patients with rare and ultra-rare diseases. Many of these therapies have enabled children who would have been expected to die in infancy to go on to live a normal, healthy life. But the small numbers and high costs prevent huge therapies being available through commercial routes and getting them to the patients that need them.

In the last two years, six gene therapy products have been dropped by commercial manufacturers for these non-medical reasons, leaving no way for patients to access life-saving treatments. As the majority of early-stage research into these therapies is funded through national research programmes, these dropped treatments also represent a huge waste of public money.

AGORA will focus initially on ultra-rare diseases which mainly include inborn errors of immunity (genetic diseases causing immune deficiency) and inborn errors of metabolism – for which no equally effective alternative curative treatments are available.

Ultimately, the aim is to explore the creation of an independent, sustainable, not-for-profit entity that can support marketing authorisation, delivery and access to therapies which are not commercially sustainable and therefore would be unavailable to patients.

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SOURCE:  UCL News.  23 September 2022

New consortium to ensure access to gene therapies for children with rare diseases

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