In a novel approach to being heard and relaying a message to the US Food and Drug Administration (FDA) Commissioner, Dr Steve Hahn, MND advocates are taking to Twitter to impress upon Dr Hahn the urgency required when it comes to early access to existing lifesaving treatments currently in final phase 3 clinical trials.
This week Matt Hancock, Secretary of State for Health and Social Care announced a trial of Inclisiran, a cholesterol lowering drug, for 40,000 patients in the UK prior to approval by the European Medicines Agency (EMA) or Medicines and Healthcare products Regulatory Agency (MHRA) or National Institute for Health and Care Excellence (NICE). The inclisiran trial is a collaboration between the National Health Service (NHS) and the drug manufacturer Novartis.
Two MND platform trials were also in the news this week. The first is the trial at Sean M. Healey & AMG Centre at Massachusetts General Hospital (US). ALS Association made an unexpected $3 million donation over three years. Enrolment is expected in early 2020 at 54 sites in the US. The drugs being trialled are expected to be Verdiperstat, CNM-Au8, Zilucoplan, IC14 and Pridopidine. The second platform trial is MND-SMART (Scotland), a charitably funded university and NHS collaboration. The trial is for 750 MND sufferers and includes all the estimated 500 MND sufferers in Scotland. The two drugs being trialled are to be announced shortly.
The above is welcome news to MND sufferers seeking to highlight to the FDA the need for urgent access to treatments such as NurOwn, CuATSM, Tregs and others in late phase FDA clinical trials. Early approval of a treatment currently in an ongoing clinical trial implies that the drug company submits an application for such approval (Priority Review, Breakthrough Therapy, Accelerated Approval and Fast Track). Nevertheless, there is a role for the FDA to play in facilitating the process and potentially signalling a favourable stance to the company. In many cases such signals may prove sufficient for the drug manufacturer to submit an application for early approval.
With the above in mind it is hoped that further collaborations between government, NHS, drug companies and MND Association will result in a model for early access to treatments in the form of continuous mass clinical trials for 5,000 MND sufferers using lifesaving treatments. Additional routes to achieving early access to treatment may include the introduction of new legislation granting such rights as well as government and MHRA/NICE inviting drug companies to apply for an Accelerated Assessment or government providing incentives to achieve the same.
If you would like to participate in the Saturday Tweet-a-thons, details can be found below.
- Saturdays, 12 noon to 6pm EST (5pm to 11pm GMT)
- 1 or more hourly tweet
- Use no more than two hashtags including #tweet2defeatALS
- The other # may be #NurOwn or other hashtag (#MND)
- A personal message to Dr Steve Hahn, FDA Commissioner
- Other recipients include @BrainstormCell
- No retweets or duplicate messages
- No insults or swearing
Example Tweet:
---
@SteveFDA @SteveHahnMD @BrainstormCell
Please work with the drug companies towards providing early access to ALS drugs/treatments. [ or any personal message ]
#tweet2defeatALS #NurOwn
---
For a UK version of the Tweet-a-thon on a different day of the week recipients could include the below listed.
@BorisJohnson @MattHancock @sallylight17 @mndassoc @DarbyRimmerMND @NICEComms @MHRAgovuk @neuroshef @kingsmnd @BrainstormCell @EuansCentre @AnneRowlClinic