Drugs which might seem very effective in clinical trials, sometimes fail in the market due to non compliance and acceptability issues from the patients. On the other hand, a drug with known and serious side effects is sometimes well accepted by the population, despite the risk involved.
An eye opener in this regard is the case of the Tyasarbi, the drug against Multiple Sclerosis. It was approved in 2004 by the US FDA, and withdrawn hardly 3 months post its launch, after being associated with cases of PML. However; the response of Tysabri in non responsive MS patients was so radical, that in spite of knowledge of life threatening and serious side effect as PML the patients were willing to take a risk with the drug, as opposed to allowing progression of MS. This was evident as representatives of MS patients and their relatives testified to the drastic improvement shown by the drug in patients of MS. There was a strong opposition to the withdrawal of the drug by the patients, who due to the non availability of the drug were being sentenced to progression of MS and ultimately a vegetative life. Thus in 2006 FDA ultimately decided that the side effects of Tysabri though serious, were acceptable considering the benefit accrued to the patient and the risk of non availability of the drug. Subsequently the drug was also approved for treatment of non responding patients of Crohn’s Disease.
Another case was the role played by patients is the case of patient led advocacy group for Duchenne’s Muscular Dystrophy (DMD). A group of more than 80 representatives from the Duchennes community held discussions on their expectation from drugs, the risk they were willing to take for clinical trials and at what risk of non curative retardation of disease was acceptable to them. A guidance document intended for the industry and regulatory authorities was then submitted to the FDA to give a direction to drug development in the field. FDA in turn got actively working with the pharmaceutical company Serepta on the development of the drug Eteplirsen for DMD.
These incidences led US FDA to come up with a unique initiative that allows patients to voice their opinions regarding their disease, how it impacts their life, their opinions on the current treatments available and what they expect from new drugs and therapies in the future. This program is aptly called as Patient Focused Drug Development (PFDD) program. The reports of these meetings are released to the public and industry, being aptly named as ‘Voice of the Patient’ reports.
PFDD have the following positive impact in the pharmaceutical world:
- It helps to give the patients’ viewpoint to the type of drugs needed in the market.
- The interaction provides the stakeholders, that is the patients & disease advocacy groups, the clinicians and researchers, and the payers, and the biopharmaceutical industry, with an opportunity to utilize this information in bringing about a positive change in the drug development process.
- Involving patients with drug development in the early stages would enable the researchers to get a better insight to the problems and possibly, newer avenues for drug discovery.
- Patient’s experiences would help in development of Patient Reported Outcomes (PRO) and help in Clinical Outcome Assessment(COA) in clinical trials.
- Since payers are increasingly depending on Real World Evidence to support their decisions, having a feedback from the end users of the drug well before the launch, gives payers an insight into how the drug will be accepted post its launch.
- For the success of any pharmaceutical product there has to be a market need, scientific opportunity to produce a drug and reimbursement or insurance coverage. PFDD ensures that the industry is made aware of the needs of the people or what they expect from the drug.
- It would help the industry to cater to the needs of the patients and also reduce the time taken for them to gain access to new drugs.
I believe that with the processes for systematic representation of patient’s voices, the regulatory authorities will now have a holistic outlook to how the drugs affect the patient’s life, the need for newer therapeutic options for diseases helping faster access of patients to drugs and clinical trials.
Become a Certified HEOR Professional – Enrol yourself here!