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Table 4 Special HTA and reimbursement considerations for orphan drugs

From: The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe

Country Special HTA considerations for orphan drugs Special reimbursement considerations for orphan drugs
Germany • Certain special HTA criteria are applied to orphan drugs:
- Higher p-values for small sample sizes
- Use of surrogate endpoints
- Additional benefit is considered proven at marketing authorization (MA) if the budget impact is less than €50 million per year for a particular indicationa
• Higher therapeutic benefit is automatically recognized for orphan drugs (Section 35a, para. 1 clause 10 of the German Social Code Book V), since these drugs had to prove significant additional therapeutic benefit compared to other possibly already approved drugs as part of the European marketing authorization procedure
• The ascertainment of an additional benefit, which is automatic for orphan drugs, is also binding for subsequent administrative acts, which includes reimbursement decisions by the G-BA (body issuing reimbursement decisions)
• IQWiG (body issuing HTA recommendations) only assesses target population size and drug budget impact to all population, and the G-BA decides only on the extent of additional benefit (this applies to all drugs, not only orphan drugs)
• While there are no specific pricing considerations for orphan drugs, the latter are often characterized as having no therapeutic alternatives (by G-Ba and IQWiG) - this makes comparison with existing therapies impossible and means free pricing in practice
France • Certain special HTA criteria are applied to orphan drugs:
- Additional benefit is considered proven at MA if the budget impact is less than €30 million per year for a particular indication
- Accelerated HTA procedure is available for all innovative drugs (not only for orphan drugs)
• The Ministry of Health decides on the reimbursement of the drug, taking into the SMR and ASMR considerations
• The Agency for the Sanitary Security of Health Products (Agence Française de Sécurité Sanitaire des Produits de Santé) can issue authorization for temporary use in case of life-threatening conditions or/and when there is no therapeutic alternative (this is not specific to orphan drugs but can be applied to them)
Netherlands None • Hospitals may apply for full additional funding for orphan drugs that are prescribed within their institution. The additional temporally funding considers therapeutic value, cost prognosis and outcomes research – treatment of all patients need to be documented in a patient registry
• In case of orphan drugs the therapeutic value, the severity of the disease and the efficient prescription will be important for the decision on definitive listing/ funding
Poland None None
Sweden None • TLV (body issuing reimbursement decisions) usually accepts a higher willingness-to-pay threshold for treatment of severe conditions; the human value principle implies equality of all people, while the principles of need and solidarity imply that conditions for which there is a greater need take precedence over others; in practice this means a higher cost-effectiveness threshold may be considered for orphan drugs
England None None
Scotland • Certain special HTA criteria are applied to orphan drugs:
- Lower levels of evidence are accepted for clinical trials (e.g., on efficacy and safety) and in economic evaluations
- Additional data may be required (e.g., surrogate markers and quality-of-life data)
Wales None None
  1. Sources: [210]
  2. Notes: aWith the exception of orphan drugs, the new Pharmaceutical Market Reorganisation Act of 2010 made the early evaluation of the additional benefit of a pharmaceutical product by the G-BA mandatory after MA; nevertheless, manufacturers of orphan drugs need to submit a dossier so that the G-BA can assess the level of additional benefit and use this in price negotiations, if needed [6]. MA marketing authorization