After a long and challenging process, on 28 August 2024, Denmark obtained access to SMA treatment for a large part of the SMA community, including adults

From SMA Europe, the non-profit umbrella organisation of spinal muscular atrophy (SMA) patient organisations from across Europe, we are very excited to showcase the last success of our Danish SMA Community. After a very long fight and many negotiations, the Danish patient organisation, Muskelsvindfonden, announced that the wider SMA Community in Denmark can now access the treatment, including adults living with SMA.

Nicole Gusset, CEO at SMA Europe:

“We as the European SMA community are relieved by the success in Denmark, where after a lengthy journey of negotiations between government and industry, Muskelsvindfonden’s patient advocates secured access to medicines for a large part of the SMA community. This hard-won achievement sets an inspiring example for other countries still striving to ensure all people living with SMA have access to vital treatments. SMA Europe remains committed to working relentlessly to support these efforts across the continent.”

We are proud to present the following interview with Henrik Ib Jørgensen, CEO of Muskelsvindfonden and Danish delegate for SMA Europe and Lisbeth Doktor, a patient representative in the Expert Committee supporting the Medicines Council in Denmark, member of the trustees in Muskelsvindfonden and Danish delegate for SMA Europe.

Henrik Ib Jørgensen, CEO of Muskelsvindfonden and Danish delegate for SMA Europe:

“In an international context, it is interesting to read and reflect on how Denmark twice transitioned from being one of the most restrictive countries regarding access to new treatments for SMA to offering treatment to all who meet the Danish Medicines Council’s start and stop criteria. These two decisions occurred within just over a year.”

Before the two decisions to include those aged six to 25 in 2023 and patients over 25 in 2024, an extensive collection of new real-world data¹ naturally preceded, convincing the Medicines Council of the treatments’ efficacy. Equally important was Denmark being offered a better price.

It is worth noting a small detail when reading publicly available information from Medicines Council meetings. After the first decision in 2023, the Medicines Council highlighted that both new data and a new price were decisive factors in expanding the group of patients offered treatment.

In the second decision in 2024, however, the Medicines Council only mentioned the price. This proved that data was no longer an issue. It was all about the price and the Medicines Council also stated that initially, they were not satisfied with paying the same price for patients aged six-25 and those over 25. Firstly, because the number of patients would increase significantly, and secondly, because the industry had not invested in conducting randomised trials on older individuals. In other words, they calculated that the high development costs alone should be covered by treating people living with SMA under 25.”

Background Story

Henrik:

“To understand the process, one must look at the brief history of the Medicines Council.”

In 2016, the Danish parliament decided to establish a Medicines Council similar to England’s NICE and other similar prioritisation institutes in Europe. The goal was to both curb the exploding prices of hospital medicines which threatened the healthcare system’s economy, and to replace political and commercial lobbying with solid professional assessments and scientific facts. The overwhelming political majority stood behind the decision to create a new Medicines Council.

With the Medicines Council, Danish politicians also made it clear that the state’s most potent strength in price negotiations would always be the courage to say “No” if the price became too high. At the same time, they did not want to abandon patients, so they somewhat simplistically invented a principle that there should be a clear correlation between price and effect.

However, the challenge was that politicians did not demand transparency from the Medicines Council regarding whether the high price or the assessment of the low effect ultimately determined whether they said “No”. The statement was simply that the price was too high relative to the effect. Therefore, the Medicines Council was understandably met with mistrust and suspicion every time they made a controversial decision, as patients asked themselves the question “Can a price be put on my life?” and many professional experts shook their heads when the Medicines Council cast doubt on data they themselves had no reason to question.”

SMA had great principled significance

Henrik:

“Treatment for SMA was the first case encountered by the Danish Medicines Council. It became a matter of principle for both the Medicines Council and it became the case that would never die.

Since the council in 2017 stated the price of Spinraza was too high relative to the effect, over 4500 articles or other media mentions of SMA have been written. There have been debates in parliament and other public gatherings.”

Lisbeth Doktor, patient representative in the Expert Committee supporting the Medicines Council and member of the trustees in Muskelsvindfonden and Danish delegate for SMA Europe:

“Our fight for treatment in Denmark has been incredibly long and hard.”

Initially, the Danish Medicines Council refused to admit anyone to receive any treatment. This was in October 2017, and by that time we didn’t even participate in the Expert Committee as patients. There were no patient representatives onboard, and we questioned this hard in the media. In November 2017 I joined the Expert Committee together with my brother and we began advising and helping collect data. The Expert Committee’s task is to advise the council and provide them with the latest updated information, so it was crucial to be represented this way.

From there we took it step by step, beginning with working for the infants and the youngest children. We had hoped they would allow all children the access, but alas! Not until June 2023 did we manage to include all the Danish children. Up until then, it was only the children under six years that had gained access, and only children diagnosed with SMA1 or SMA2 were included, so a large group of older children were left out for what seemed to be an unfair long wait. The council argued that there wasn’t enough data to approve the treatment when the price asked for was so high. At the same time, they approved both Evrysdi, Spinraza, and Zolgensma for children under six years of age. It felt like they only wanted to help the young children gain more strength, and it was hard for the older people living with SMA to accept this.

In June 2020 we filed a new report from the Expert Committee to the Medicines Council, saying that not only the children should gain access to treatment, but also the adults. This was groundbreaking because, at the time, only very young children received treatment. It was also the first time the Expert Committee had advised treatment to adults. The Medicines Council did not listen, but it did leave an impression on the council, and although they were still skeptical towards the clinical data, they asked the Expert Committee to collect Real-World data. In the media, we questioned why they felt the need to go against their own Expert Committee and the response was total silence. It did put pressure on the Council for the next time they would have to revise the data. Going against the Experts’ guidance is not easy and cannot be done multiple times.

I think it also made an impression that so many countries around the world had reimbursed treatment. We spent a couple of years collecting Real-World data, which supported the initial clinical trials data, and this was enough to finally convince the Council that the treatment worked, and the patients would benefit from it, children as well as adults.

However, the price was still too high. Therefore, the council approved of the Expert Committee’s report stating that they acknowledged the data and that the only barrier left was the price. This was in June 2023, and the statement and acknowledgement of the data were important because they enabled us to back the Medicines Council and turn towards the industry, together with the Council. Before this, we had argued and appealed to both the Medicines Council and the industry to find a compromise, but after the Medicines Council accepted the data and no longer questioned the validation, we were able to fight together with them for a better price.” 

SMA treatment was put out to tender

Henrik:

“In the spring of 2023 prior to the final meeting in June, the council invited all three pharmaceutical companies to submit new price bids. The company offering the lowest price was guaranteed 80% of the market, while the others would share the remaining 20%.

Initially, the tender did not result in any significant discounts, according to Muskelsvindfonden, because the Council had completely mishandled the tender. One thing was that the incentives for the industry to offer lower prices were almost nonexistent because the companies were bound by the price bid, regardless of how many patients the Medicines Council subsequently chose to offer treatment to. Companies could thus risk selling fewer treatments at a lower price. Another, equally significant factor was that pharmaceutical companies could maintain a sky-high price level without considering that the production costs for a treatment are virtually nothing and that the development costs must have long been covered by selling treatments to the many children and young people who had been tested with treatments.

Therefore, Muskelsvindfonden called for the tender to be redone. We wrote that the Medicines Council had shot itself in the foot and appealed to the industry by not allowing for differentiated prices, bulk discounts, or even offering a so-called risk-sharing agreement, where the industry also takes on financial responsibility for the fact that data for treatments for rare diseases are not always as mature and secure as data for more common diseases, where treatments can be tested on more patients and possibly in a shorter time.

The tender was redone“

The post was nothing short of a game-changer

“Not only did the Medicines Council decide that the tender should be redone, but it also decided to invite Muskelsvindfonden and the umbrella organisation Danske Pateitner to an initial dialogue meeting in the price negotiations between the industry and the authorities.

This signaled that patient organisations were now seen as serious partners and allies in pushing for better prices, rather than being seen as a Trojan horse for the industry to achieve the highest possible prices.

It was, of course, a huge dilemma to go hard against the companies that have invested in developing new, groundbreaking treatments for people living with SMA after decades of basic research.

But the analysis was that the three SMA treatments are only the first generation of a long series of Advanced Therapeutic Medicinal Products for rare, neuromuscular diseases. It is also in the patients’ interest that healthcare systems can afford employees in the future. At the same time, advanced medicines also share the characteristic that the long-term effect is often unknown, and the data are incomparable with more frequent diseases. In principle, it is only fair for the industry to take on both an economic and an ethical responsibility to ensure that more people are offered treatment. If we are to ensure faster and better access to new advanced medicines, the risk must be shared between the pharmaceutical industry and the payers.

In the specific case of SMA, there is also a huge difference between children and adults in the sense that most of the clinical trials were designed with patients under 25 years old and that there is only real-world data for patients over 25 years old.

At the dialogue meetings preceding the actual price negotiations between the industry and the authorities, we therefore directly confronted the industry to lower the price, rather than making unrealistic demands on the authorities that they should pay whatever it costs.

“Everyone benefits from the treatment, but price is an important factor we can’t ignore.”

There is no doubt that the three new treatments will revolutionise the lives of future generations with SMA. Especially if they are combined with newborn screening, which Denmark introduced in early 2023. But of course, this does not mean that the treatments do not have value for individuals who are born and may have lived with SMA for many years.

The introduction of newborn screening meant that a record number of babies in Denmark were diagnosed with SMA in 2023 because they now found patients who would otherwise only have been diagnosed later in life when symptoms appeared. Together with the children who were already in treatment, this ensured the industry a solid income for several years.

At the dialogue meetings, Muskelsvindfonden argued that the significant earnings the industry had made on children and young people left all political parties with an even greater responsibility to ensure that no one was left behind and that any doubt or disagreement about data should benefit the patients. It is not the patients who should pay a high price by being denied treatment. Instead, the price should follow the evidence and the effect. If there is no measurable effect, but patients find the treatment effective, the industry has little to lose by lowering the price rather than stopping the treatment. If compassionate use and early access programs can be used in connection with the marketing of the new drugs, they must also be available for the last generations with SMA who are not offered treatment.”

Lisbeth Doktor:

“Throughout the years we have been very fortunate to collaborate with SMA Europe and the exchange of experience and data from around European countries has added to our argumentation and helped us convince the Medicines Council in the end. I believe patient advocacy is very important because it makes us stronger and more convincing when we know more and have access to the information.“

Lessons to learn

Henrik:

“It is not always an effective strategy for patient organisations to focus solely on the value we can gain from a given treatment—without putting just as much effort into discussing the price, which has been the elephant in the room from the beginning of the process we went through in Denmark.

There is little doubt that both the marketing and finance departments of pharmaceutical companies will likely be pleased if patients continue to direct the brunt of their criticism at the prioritization agencies that say no, and the politicians behind those agencies. While the media and commentators target civil servants and elected officials, the company indirectly receives support to maintain a sky-high, and not always reasonable, price level.

Additionally, it was only when Muskelsvindfonden started to work together with the Medicines Council and we helped them to negotiate the price, it didn’t take long to see the number of individuals in treatment multiply.

Since Muskelsvindfonden, like several others, is an umbrella organisation for all neuromuscular diseases, for which we can expect more gene therapies and other ATMP treatments in the future, we as patient organisations need to take a greater interest and responsibility in how we can ensure faster and easier access to effective treatment through so-called risk-sharing agreements, where it is the industry and society – not the patients – that share the risk of immature or weaker evidence.

SMA Europe wishes to express our happiness once more about the outcome of this process which is now the inspiration for other countries and other SMA and rare disease communities. Our organisation will keep working hard to ensure SMA Europe’s member organisations feel supported by us at every moment and that, in the end, the dream of no one being left behind, when it comes to access to treatment, will become true for every person living with SMA.”

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Creating a better world for all those living with SMA

secretariat@sma-europe.eu | www.sma-europe.eu  Im Moos 4, 79112 Freiburg, Germany

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