Health Technology Assessment (HTA) agencies across the globe are witnessing a paradigm shift in their drug assessment processes. From formulating guidelines on evaluating cell and gene therapies, incorporating patient input, real-world data and other unconventional metrics to implementing managed entry agreements, the changes are essential to keep up with the dynamics of the ever-changing medicines market.

In 2016, following an independent review of access to new medicines, the Scottish government announced its plan to improve access to new end-of-life, orphan and ultra-orphan medicines. The appraisal included recommendations on reforming treatment approval processes in the country. One of the provisions was to introduce an interim decision option for medicines addressing unmet needs using real-world data, patient-reported outcomes, and other qualitative measures. Consequently, the Scottish Medicines Consortium (SMC), the HTA agency of Scotland, introduced interim decisions in 2018. Drugs with a European Medicines Agency (EMA)’s conditional marketing authorization (MA) are eligible for listing within NHS Scotland on an interim basis. The process is comparable to that of England’s Cancer Drugs Fund (CDF), but the government has not designated a separate budget to fund provisional acceptances.

EMA conditional MA is granted to medicines with restricted evidence exhibiting a high benefit/risk ratio with an assurance of comprehensive data in the future. Drugs fighting against life-threatening or orphan diseases where immediate availability outweighs risks associated with immature data can gain from faster access. The conditional MA is valid for one year and renewed annually. At the end of the license validity, the EMA converts the conditional MA to a standard one. At this stage, the holder of the MA presents additional evidence which is subject to reevaluation. Under the interim decision option, SMC would consider a drug if the committee expects that the new evidence will address the uncertainties highlighted in the initial submission.

Drugs that stand to benefit from SMC interim decisions are ones that have immature data with uncertainty on long-term risks and benefits but demonstrate an efficacy potential. The aim is to promote accelerated access while limiting the strain on NHS Scotland resources. Banking on the acknowledged EMA pathways, the committee intends to mitigate risks associated with insufficient evidence. Bound by the set-up of the conditional MA, pharmaceutical companies are responsible for collecting additional clinical data for reassessment and providing real-world evidence, if any. As of July 2019, SMC has received its first interim option submission, but the details remain confidential. The final decision has also not yet been published.

The implementation of interim decisions in association with EMA conditional MA promotes reuse of clinical information and expertise, reduces duplication, brings about collaboration at the European level, and optimizes the use of resources. The collective aim of the industry stakeholders is to provide equitable and faster access to life-saving therapies. It is noted that SMC interim decision option offers an opportunity for accelerated access but is also restrictive since the selection criteria solely depends on the EMA conditional MA terms. According to a report published by the EMA, only 30 medicines received a conditional MA from 2006 to 2016. More recently, drugs fighting diseases such as non-small cell lung cancer, beta-thalassemia, hyperlipoproteinemia type 1, or squamous cell carcinoma have been granted conditional licenses.

Interim acceptances are expected to boost access for medicines for which therapeutic alternatives are not available. Even though the idea is to shorten time to market, timelines associated with interim submissions have not been defined. The Scottish government is also investing in improving the process for Individual Patient Treatment Requests (IPTRs), promoting patient engagement, and developing national electronic health records. Pharmaceutical companies with ultra-orphan, end-of-life drugs in the pipeline can expect relaxed regulations, streamlined operations, and faster access within NHS Scotland. The impact, likely positive, will be evident soon.

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