The Ministry of Health of the Nation announced this Friday that it reached an agreement with the Novartis laboratory to start providing a
high-cost gene therapy
for the treatment of
Spinal Muscular Atrophy (SMA) type 1
.
It is about
Zolgensma
, a drug that is administered only once and has a
list value of 2 million dollars
.
What is the agreement reached by the Ministry of Health and who will be able to receive it?
This drug is the one in the campaign with which the influencer Santiago Maratea raised the money for Emma Gamarra at the beginning of 2021. Emmita --as the baby became known in networks-- had the most severe degree of Spinal Muscular Atrophy (SMA). .
This disease is a
condition of genetic origin
characterized by symmetric, progressive muscle weakness and atrophy of variable severity, the result of degeneration and loss of lower motor neurons in the spinal cord and brainstem nuclei.
This mainly affects the lower limbs and respiratory muscles.
The joint venture agreement
The dialogue with the laboratory started in 2020 and an agreement was recently reached that includes the
risk sharing strategy
.
“This means that the State is responsible for the value of the drug as long as the drug works.
In the event that certain milestones or objectives are not met,
the cost will have to be absorbed by the laboratory
, ” Natalia Messina, director of Special Medicines and High Price of the National Ministry of Health
, explains to
Clarín .
A 2021 photo of Emma Gamarra, the baby for whom Santiago Maratea raised the money for her medication.
The drug Onasemnogene Abeparvovec (Zolgensma), with which gene therapy is performed, has a commercial sale value of more than 2 million dollars and is the highest priced drug in the region and
the second most expensive worldwide
.
As part of the agreement, the State will acquire it for
1.3 million dollars (plus VAT)
.
“As soon as the patient receives the medicine,
the State will pay 20%
.
The payment will be made in pesos at the value of the official dollar on that date," Messina specified when asked by this newspaper.
Every 12 months, the laboratory may claim
another 20% to be paid in the same way
.
At that time, the patient will be evaluated again and it will be determined if this payment corresponds or not, according to the evolution of his health.
“There are
certain milestones
that the patient has to reach, depending on their age, related to their respiratory and motor situation.
If he does not reach them or worsens, the State will not be responsible for the payment and the laboratory will have to absorb the cost ”, he maintains.
The Ministry of Health refers to an investment of
more than 15 million dollars because an
open purchase order for that amount
is going to be approved
, with the idea of being able to cover the treatment of 12 patients, which is an estimate of potential children with AME 1 who might need medication.
This is the available budget, but it does not mean that it will be used in its entirety and the payment will also be made in a fragmented manner.
“There is a
registry of patients with SMA
for the administration of another medication to treat Spinal Muscular Atrophy, which is called Spinraza.
We are going to add the patients who require Zolgensma to that same registry”, says Messina.
There are four subtypes of SMA based on the age of onset and the severity of the disease.
The SMA type 1 form is the most common and severe (58%)
and the symptoms are of childhood onset.
Candidates to receive this medication must have SMA type 1 and may not be older than 9 months.
Zolgensma, the first high-cost drug for which a joint risk agreement is signed.
The doctor must register the patient and the
National Commission for Patients with SMA (CONAME)
-which already exists to analyze the cases that require Spinraza- will evaluate if the administration of Zolgensma corresponds to it.
This commission will also review whether the patient meets those milestones mentioned above.
The State will take charge in all cases,
also when the person has social or prepaid work
.
“This means that the only way to access Zolgensma will be through the State.
If the patient is a candidate to receive the medication, from now on the family will avoid the difficult journey associated with access, which often involved going
to court
, ”says the reference from the Health portfolio.
This
will also benefit the prepaid and obras sociales
that, in some cases, have been taking over a percentage (generally, 10% or 20%) of the cost of the medication, almost always after filing an appeal for protection.
A possible precedent for other drugs
Regarding the possibility of this strategy being extended to other drugs, Messina assured: “This agreement
it could be
a precedent for addressing access to high-cost medicines
.
We are going to start evaluating other therapies.
The dialogue with Novartis began in 2020, it took a while to reach this arrangement, which contemplates a reduction in value, the risk-sharing strategy and payment every 12 months and according to the evolution of the patient.
The commitment was sealed with the signing of Resolution 21/2023 by
Minister Carla Vizzotti
.
After this purchase order is opened, the laboratory has to formally submit the proposal and accept the conditions.
"We believe that
three months from now we could start buying the medicine
," confirms Messina.
He explains that they plan for the CONAME approval to be carried out in a single meeting, as is the case today with the majority of cases in which Spinraza is authorized, taking into account that they are severe pathologies for which medications must be supplied in a timely manner. It's fundamental.
"CONAME meets once a month, but when it receives an urgent case, an emergency meeting is requested. We seek to resolve cases within the same day, as long as we have the corresponding documentation," adds Messina.
With this agreement, Argentina becomes
the first country in the region to make an acquisition from the public sector under these criteria
.
What did they say from the lab?
Eugenia Esquerro, medical director of Novartis Argentina, explained that "it is a therapy that consists of
a single dose applied in the patient's lifetime
that acts on the root cause of SMA by replacing the absent SMN1 gene with a healthy copy."
"This will improve the functionality of the motor neurons of
the patient's
nervous system ," he added.
Regarding the negotiation, he highlighted that "an exhaustive analysis of the clinical evidence was carried out and based on that
, a patient profile was built
that would benefit most from the treatment. Then, the bases of the joint risk agreement were consolidated, identifying the milestones of evolution expected in patients after receiving the therapy".
"Finally, the commercial conditions were established and the corresponding internal approvals were processed," he told this newspaper.
Along these lines, he added: "We believe that this achievement
will mark a milestone in the way in which high-cost medicines are incorporated into the health system in
a responsible manner within the framework of collaborative work between the public and private sectors."
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