These are some of the issues I think need to be addressed regarding pharma and patient involvement. All of these observations are based on my interactions/or attempted interactions with Vertex and contact with the FDA.
I’m guessing Vertex isn't involved in this project you mention. This is a shame, because they certainly have a lot to learn about being “Patient Centric”!
Transparency of Expanded Access/ Compassionate Use programs: The whole purpose of compassionate use /Expanded Access programs is to provide access to a drug that is in development to patients that are in great or dire need of this drug that is thought to possibly benefit them. These patients are not eligible for a clinical trial of the drug. (in this particular example that I will refer to, they are excluded because they have an FEV1 of less than 40% or unstable disease). Vertex has had an expanded access/compassionate use program for Kalydeco in existence for quite some time for certain residual function mutations. I can’t say for certain for how long, but I have good reason to believe that it’s been available for years- as early as 2013 or 2014. This program was not known about by many CF doctors until recently.
Companies should choose transparency and voluntarily put these expanded access programs on the clinicaltrials.gov site.
Because these programs aren’t clinical trials, they are not required to be listed on the clinicaltrials.gov site, although according to the FDA, they may be registered voluntarily.
They also have the responsibility to increase awareness of the program by identifying the drug as being available for expanded access when registering for other clinical trials that include the drug. (In this case, Ivacaftor.)
The FDA guidelines for industry state: “When registering a clinical trial that includes a drug or device that is also available via an expanded access program, the question Has Expanded Access? should be answered Yes (see Expanded Access and Has Expanded Access? on ClinicalTrials.gov). Also provide the ClinicalTrials.gov Identifier (NCT Number) for the expanded access record.”
This was not done by Vertex as far as I can tell. There is no mention of Expanded Access on the Clinicaltrials.gov for studies that include Ivacaftor (Kalydeco) such as the recently closed VX-661-108 trial. (There was an arm of study where Ivacaftor was used alone, as a monotherapy, without the 661 for those with residual function)
Also from FDA’s industry guidelines for expanded access programs: “Physicians or patients may inquire with the sponsor (or the manufacturer of the investigational drug if different from the sponsor) about possible availability. Because expanded access to an investigational drug is provided under an IND and information pertaining to an IND is generally not publically available, the best source other than Clinicaltrials.gov for information on expanded access to a particular investigational drug, such as whether expanded access is available or who to contact to obtain access, is the sponsor of the IND or the manufacturer of the investigational drug. Sometimes the sponsor or the manufacturer will include such information on their Website.
The FDA encourages companies that are developing drugs for the treatment of serious diseases, especially those for which the clinical development program has been granted fast-track of breakthrough therapy designation, to make contact information and information about their company’s expanded access programs and policies available to the public.”
https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/ucm429687.htm
This has not been done by Vertex. Not only isn’t this available on their website or clinicaltrials.gov, I was not given information about it when I called Vertex specifically asking for information for expanded access programs for those with residual function. I was told to have my doctor contact them. This is a perfect example of disreguard for the concept of participatory medicine where patients should be seen as equal partners in their healthcare. If there is some valid reason that these FDA guidelines weren’t followed, then Vertex should have made that known to me when I questioned all of this. I sincerely hope that there is a reason because it sickens me to think that people have suffered unnecessarily or perhaps even lost their life because of lack of awareness of this program.
These examples can be passed on to your group as a patient’s perspective on how pharma should NOT be acting.
I have some other suggestions and will post them when I have more time.
