Joint Clinical Assessments: a new chapter in European pharmaceutical policy

The EU Health Technology Assessment Regulation (HTAR) was developed in response to unequal patient access to new drugs and delays in their availability across many European countries. Representing a significant shift in European pharmaceutical policy, the focus of the discussion about access is now on comparisons of benefit of new drugs against the health care standards in EU Member States.

While the European Medicines Agency’s (EMA) marketing authorization process assesses the benefit-risk profile of individual drugs, HTA informs decisions on reimbursement, pricing and treatment guidelines within the respective health care systems. The Regulation aims to reduce duplication of effort across EU Member States and accelerate patient access to new treatments through a joint assessment of the available clinical evidence on which decisions can be based across Europe.

JCAs are produced in parallel with the EMA’s marketing authorization process. The Regulation thus brings HTA forward to an earlier stage in the life cycle of a new drug. This is because national decision-making can only be supported without delay by the European HTA process if a JCA is available shortly after European marketing authorization. This presents challenges. Above all, evidence suitable for such assessments must now be available at an earlier stage. Ideally, this will encourage pharmaceutical companies to initiate the collection of comparative data earlier in their clinical development programme.

However, providing the evidence base is only the first step on a drug’s path into the health care system: patient access will continue to depend on additional factors. For instance, in countries where the ability and willingness to pay vary significantly, a national price must be negotiated in each case. It remains to be seen what contribution JCAs can make towards overcoming these additional hurdles.

The impact of JCAs may extend beyond supporting decision-making in EU Member States. By providing a systematic, independent and transparent assessment of the added benefit of a new drug compared to a range of alternative treatment options, JCAs could be used outside Europe too. This also applies to the US, where there is currently no transparent process linking clinical benefit to reimbursement and pricing decisions. Private payers already rely on assessments by independent bodies such as the Institute for Clinical and Economic Review (ICER). JCAs could therefore become an important basis for price negotiations in the US.

However, some voices, particularly from the pharmaceutical industry, question whether JCAs will actually facilitate market access in Europe. This will depend on various factors, including the extent to which EU Member States converge in their requirements, specifically for the comparators relevant for the assessment. If the Regulation succeeds in making new drugs with a proven added benefit more consistently available to patients across Europe, the answer is likely to be positive.