Ultragenyx

Rüdiger Schulze

Pharmaceutical Innovation Authority

On 10 July 2026, the federal parliament in Germany endorsed the GKV Contribution Rate Stabilization Act, which had been proposed by the Ministry of Health several months ago. This reform package aims to stabilize contributions while maintaining high-quality health care services through a wide range of cost-containment measures that affect many different stakeholders. It contains a mix of welcome measures and new uncertainties for both biotech innovators and patients living with rare and ultra-rare diseases. While the reform preserves the core orphan drug framework and removes some burdensome AMNOG cost-containment measures, it also introduces new financial pressures that could influence future investment and launch decisions.

Even more important is the potential impact that these measures could have on patients living with rare diseases and their families and caregivers, who often depend on long-term access to interdisciplinary secondary care and innovative medication. These communities are particularly vulnerable to cost containment because they already bear a high economical, psychological and social burden.

Content of the Proposed Reform for Rare Diseases

The new legislation seeks to limit expenditure and generate additional revenues for statutory health insurances ( Gesetzliche Krankenversicherung, GKV) by a multitude of measures that come with mixed implications for patients living with rare diseases.  Positive signals are sent by not imposing further restrictions to the reimbursement of orphan drugs. The abolition of the pricing guardrails and the combination rebate also removes some measures that had been criticized for creating administrative burden and reducing predictability for innovative medicines.

At the same time, the pharmaceutical sector is expected to contribute to savings through an additional rebate on patent-protected drugs. Unlike previously proposed, this rebate will not be dynamic, meaning it will not depend on the actual expenditure for these drugs, but will instead be fixed at 15.5% of their net price, which means the rebate will more than double from its current level of 7%.

Price increases remain prohibited until 2030 by which time the price moratorium will have been in place for 20 years. Price-volume elements that were introduced to reimbursement agreements in 2022 will now be handled more consistently by introducing an additional fixed rebate for exceeding certain annual revenue thresholds.

Meaningful Reforms for Rare Disease communities

For patients living with rare diseases, access to innovative medication is critical as it often determines whether a disease can be treated effectively at all. Germany should be proud of its historically “fast and broad” access to orphan drugs. The latest analysis by the European Industry Association of the Pharmaceutical Industry (EFPIA) found an availability rate of 97% in Germany for all orphan drugs that have received marketing authorization between 2021 and 2024.

While no further restrictions on the reimbursement of Orphan Drugs have been introduced, the higher co-payments place an additional burden on patients with chronic diseases, particularly those with conditions that require frequent hospitalizations. Co-payments remain capped at 1% of annual income for patients with chronic diseases but come with the burden of proof on patients, who must navigate additional bureaucracy. If patients with rare diseases choose not to seek medical help because of economic considerations, this could lead to a deterioration of their health and quality of life.

Patients living with rare diseases are affected by bureaucracy throughout the healthcare system, often requiring multiple applications to access medical supplies or services, navigating unstructured referral pathways from primary to specialty care and facing the poorly organized transition from pediatric to adult medicine. None of these challenges is addressed by the current reforms.

The challenge of navigating the complex healthcare system is becoming increasingly relevant as a new generation of advanced therapies and gene therapies reaches patients with rare diseases. Unlike many traditional medicines, these treatments often involve highly specialized treatment centers and long-term follow-up to fully understand their clinical benefit. For health systems, the challenge is therefore not only how to finance innovation, but also how to assess and deliver therapies whose value may unfold over many years.

As scientific progress accelerates, the challenge is no longer simply whether innovative treatments can be developed, but whether patients can access them in time to benefit. This is particularly relevant for advanced therapies and gene therapies, where patient populations are small, treatment pathways are highly specialized and delays in diagnosis or referral can have irreversible consequences. For these therapies, timely access depends not only on reimbursement decisions but also on the ability of health systems to identify eligible patients and connect them to expert centers.

Innovation Must Reach Patients

The GKV Contribution Rate Stabilization Act represents the most ambitious change in healthcare financing Germany has seen in decades. But uncertainty for biotech innovators is increasing, making the future availability of these drugs hard to predict. From the perspective of patients with rare diseases, the proposed reform does not address the challenges of delayed diagnosis, referral and care transitions.

At a time of unprecedented scientific progress in rare disease research, including the emergence of advanced therapies and gene therapies, the true measure of success will not simply be whether innovation reaches the market, but whether it reaches patients. Timely diagnosis, referral to expert centers and efficient treatment pathways may ultimately prove just as important as reimbursement policy in ensuring scientific breakthroughs translate into better outcomes for people living with rare diseases.