Generating comprehensive data in rare diseases or chronic progressive conditions, can be challenging often requiring lengthy study durations and very high investment. The European Medicines Agency (EMA) therefore considers granting early market authorisation of a product for seriously debilitating, life-threatening, or rare diseases. Given the current global climate from challenges posed by COVID-19, even more interest has been directed towards fast-tracking regulatory approval. Conditional Marketing Authorisation (CMA) is a regulatory pathway for such diseases that enables timely access to therapies.
Our question is: what are the HTA implications of launching with a CMA?
1. Among the many attributes of these products, our initial exploration was to look at the HTA outcomes for drugs with orphan or for oncology indications.
2. We wanted to take a closer look at those 40 drugs. We categorised them by their orphan designation and clinical evidence packages to see if categorisations commonly sought a CMA.
3. We did a deep-dive into the HTA outcomes of the 40 drugs that launched via a CMA. The purpose of our analysis was to explore HTA outcomes of CMA products in indicative HTA markets.
Among the many attributes of these products, our initial exploration was to look at the HTA outcomes for drugs with orphan or for oncology indications.
HTA Outcomes achieved by oncology and non-oncology indications that launched with a CMA in FR, DE, and UK
Majority of the products not assessed by respective HTA body are very recent entrants and likely to be assessed in the near future.
In Germany, a clear majority of drugs that were assessed were given ‘not quantifiable’ or ‘added benefit not proven’ as outcomes. The majority of the outcomes are not quantifiable, which may be due to the additional clinical benefit rating in certain circumstances for orphan drugs under AMNOG.
In the UK, more favourable HTA outcomes were observed from the 40 drugs that launched via a CMA in comparison to both France and Germany. Like France and Germany, orphan drugs also achieved more favourable HTA outcomes in the UK as opposed to non-orphan drugs.
Interestingly, the most common HTA outcome in the UK was recommended via CDF, which shows that oncology drugs may be the main driver for CMA products obtaining a favourable HTA outcome. Moreover, the future prospect of an Innovative Medicines Fund for the UK may act in a similar way to the CDF for oncology drugs, opening the opportunity for non-oncology products to pursue a conditional reimbursement route.
HTA Outcomes achieved by Oncology and Non-Oncology indications that launched with a CMA in FR, DE, and UK
Building on our previous analysis of orphan drugs, we decided to take a closer look at non-oncology vs. oncology drugs. The country that exhibited a noticeable difference in non-oncology vs. oncology HTA outcomes is the UK with most drugs recommended via CDF. This proves that oncology drugs launching via a CMA achieve favourable HTA outcomes because of the special considerations of the CDF.
4. We collated the obvious key observations, and we are now prepared to ask the bigger questions.
Now that we have established our observations, we are now in the position to ask deeper questions. Through our observations, we are able to pinpoint criticalities that will reshape our understanding of the CMA pathway and its implications on HTA and price.
If you are considering launching your drug via a CMA or if you have an orphan drug, reach out and find out more about what we can do for you.
About the authors
Dr. Jesvin Samuel