The role of PROs in approval, reimbursement and prescribing decisions in Europe
By: Montse Casamayor | November 10, 2015
Patient reported outcomes (PRO) have become an increasingly important part of the oncology drug approval process. End of 2009, the US Food and Drug Administration (FDA) issued a formal guidance to describe how PRO instruments will be reviewed and evaluated with respect to claims in approved medical product labeling; and four years later EMA produced a reflection paper on the use of PRO measures in oncology studies.
Regulators clearly value this data as part of their drug assessment process, but what about after the drug is approved? PRO data provides valuable insight into the quality and efficacy of these drugs, but there has been little research to determine whether payers and providers use this information in the context of decision-making for cancer treatments. To provide some insight into this area, Quintiles conducted a study exploring the impact PRO label claims have on prescribing and reimbursement of these drugs.
Our research, while preliminary, delivered some interesting results that we are presenting in a poster at the 2015 ISPOR European Congress in Milan.
What we found
To conduct the research we looked at submissions made to Health Technology Assessment (HTA) agencies in Germany, the UK and France for oncology treatments to see what impact PRO data had on approval, reimbursement and prescribing. Overall, we reviewed 85 HTA submissions covering 31 oncology drugs. Manufacturers presented PROs in 67 percent of HTA submissions; PROs were also presented in the HTA submissions of eight drugs with no EMA PRO data. Data relating to quality of life and pain were the most common.
Our findings suggest at a high level that if the PRO claims were statistically significant it improved the chance that a drug would be recommended and reimbursed.
But we also found that not all PROs are equal. Our research shows the PROs that have the greatest impact on reimbursement align directly with the competitive advantage that the drug brings to the market. That advantage is different for all of them, so there is no one consistent type of PRO that works for everyone. For one drug it might be the significant impact greater efficacy has on quality of life, whereas for others the relevant PRO data might be tied to the reduction of adverse events, greater tolerance, or less pain or fatigue.
The key lesson that drug manufacturers can take from this research is that gathering PRO data can add value beyond the approval process. If the manufacturers take the time to collect specific PRO results that demonstrates statistically significant benefits, physicians will be more likely to prescribe the drug and payers will be more likely to cover the cost. However, it will only work if manufacturers are thoughtful about the PRO data they collect and use to differentiate their product. Many manufacturers today rely on generic PRO tools that gather the same information as their competitors about efficacy and safety. While this data is valuable to regulators, it may not have much appeal for payers and providers. To get the most benefit from these data collection efforts, manufacturers need to identify or develop custom tools that will garner the specific relevant PRO data to make their products stand out. It will likely add time, money and complexity to the clinical trial process, but if it causes more payers and providers to support the drug, than it is well worth the effort.