Author: Dr. Chitra Lele, Sciformix Corporation
The challenge for pharmaceutical commercialization no longer ends with regulatory approval; it intensifies after the approval. Today there is greater awareness about the benefit and risk of products, not only among healthcare professionals (HCPs), but also among consumers and payers. All interested parties are demanding more evidence than mere safety and efficacy data from clinical trials. Effectiveness of treatments and comparative effectiveness relative to other therapies are important to measure. This requires real-world data based on longer follow-up and a more representative sample of the patient population than what is typically used in clinical trials. Patient reported outcomes (PROs) can help fill this gap, yet there are challenges that must be addressed.
Sources of data can be found in claims databases, disease registries and electronic health records (EHRs), but the quality of readily available real-world data may be poor, due to missing data and inadequate validation. Hence, more outcomes data has to be generated to provide comprehensive evidence of the value of the drug. In addition, patient perspective has become more important to the payers, further supporting the need for outcomes data.
PROs and other similar tools are used to capture quality of life (QoL) data along the continuum of clinical development, post-approval studies and patient registries, which are more compelling than, for example, survival or even progression-free survival data. QoL data rose to prominence a couple of decades back with the increase in incidence of oncology indications, high drug prices and small incremental gains in survival rates offered by the new drugs. QoL data is also equally relevant for other lifestyle diseases such as diabetes and asthma. The fundamental premise now is that health related QoL and well being of patients are core co-primary endpoints in clinical research and clinical care. Many companies use PRO data to measure the impact and effectiveness of their drugs, even during Phase II and Phase III clinical trials. In fact, some companies are also using PROs at the very start of the drug development process. Early observational and epidemiology studies can identify unmet clinical needs and potential profitable drug markets. For example, PRO health surveys can be used to quantify the physical health burden of a particular type of disease.